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Pediatric Study Guide

GI/GU, MSK, Genetics, Nutrition, Endocrine, Neurodevelopmental & Mental Health

FNP PEDS Β· NGR Β· EXAM 3 Β· COMPREHENSIVE STUDY GUIDE

The "Why It Makes Sense" Guide

Every condition walked through: pathophysiology β†’ clinical findings (and why they appear) β†’ diagnostics (and why we pick them) β†’ treatment (and why it works). Built for application-level questions.

⚑ 45+ conditions 🧠 Patho β†’ Signs β†’ Dx β†’ Tx πŸ“‹ Board pearls 🎯 Application-focused
LAST UPDATED Β· APR 23 2026
MODULE 8 Β· CHAPTER 26 & 28 Β· 10 QUESTIONS

Gastrointestinal & Genitourinary

5 GI Β· 5 GU

Pyloric Stenosis

GI Β· STRUCTURAL ANOMALY Β· 2–12 WEEKS
Pathophysiology

Hypertrophy and hyperplasia of the circular muscle of the pylorus narrows the gastric outlet. The thickened muscle acts like a closed valve β€” milk enters the stomach but cannot pass into the duodenum. Over days to weeks, the baby vomits more forcefully, becomes dehydrated, and loses HCl and K⁺ through vomited gastric contents.

Clinical presentation

Classic: firstborn male, 3–6 weeks old, previously feeding well, now with non-bilious projectile vomiting after feeds. Hungry immediately after vomiting ("hungry vomiter"). Weight loss, dehydration, decreased wet diapers.

Exam: olive-shaped mass palpable in RUQ (hypertrophied pylorus). Visible peristaltic waves across epigastrium.

Why the signs make sense Non-bilious because the obstruction is proximal to the ampulla of Vater β€” bile can't mix with the vomitus. Projectile because the stomach must generate massive pressure to push through a closed pylorus. Hungry after vomiting because nothing has reached the duodenum to trigger satiety.
Diagnostics

Abdominal US (first line): pyloric muscle thickness > 3 mm, channel length > 15 mm.

Labs: hypochloremic, hypokalemic metabolic alkalosis (classic).

Why those labs Persistent vomiting loses HCl β†’ alkalosis + low chloride. Volume depletion triggers aldosterone, which retains Na⁺ and dumps K⁺ and H⁺ into urine β†’ paradoxic aciduria + hypokalemia. Fix the fluids before surgery.
Treatment

Pyloromyotomy (Ramstedt procedure) β€” surgeon splits the hypertrophied muscle longitudinally without cutting mucosa. Correct electrolytes and dehydration first β€” anesthesia is dangerous with alkalosis.

🎯 Board pearl If you see a 4–6 week old with progressive projectile vomiting and an olive mass, US first, IVF + electrolyte correction, then surgery. Do not cath for UA, do not work up sepsis first β€” this is mechanical.

Umbilical Hernia

GI Β· STRUCTURAL Β· COMMON IN INFANTS
Pathophysiology

Incomplete closure of the fascial ring at the umbilicus after cord separation allows abdominal contents (usually omentum or bowel) to bulge through. Much more common in African American infants and preemies.

Clinical presentation

Soft, reducible bulge at the umbilicus that protrudes with crying, straining, or standing. Usually painless. Size usually < 1–2 cm.

Why it's usually fine Most (> 90%) close spontaneously by age 4–5 as the rectus abdominis fascia develops and comes together. Incarceration or strangulation is rare in umbilical hernias (unlike inguinal hernias), so watchful waiting is safe.
Treatment

Observation until age 4–5. Surgical repair if: persists past 5 yrs, defect > 1.5–2 cm, symptomatic, or incarcerated. Do not tape/bind β€” no evidence it helps and can cause skin breakdown.

🎯 Board pearl Umbilical hernia = wait. Inguinal hernia = refer to surgery (high incarceration risk, especially < 6 mo).

Appendicitis

GI Β· ACUTE Β· MOST COMMON PEDS SURGICAL EMERGENCY
Pathophysiology

The appendiceal lumen gets obstructed (fecalith, lymphoid hyperplasia after viral illness, rarely tumor). Mucus and bacteria accumulate behind the obstruction β†’ pressure rises β†’ venous and then arterial compromise β†’ wall ischemia β†’ bacterial invasion β†’ perforation within 24–72 hrs if untreated.

Clinical presentation
  • Classic sequence: anorexia β†’ periumbilical pain β†’ nausea/vomiting β†’ pain migrates to RLQ β†’ low-grade fever
  • McBurney's point tenderness (1/3 way from ASIS to umbilicus)
  • Rovsing sign (LLQ palpation β†’ RLQ pain), psoas sign, obturator sign
  • Children under 5 often present atypically with diffuse pain and perforate early (delayed dx)
Why pain moves Periumbilical first because visceral (stretch) pain from the appendix refers to the T10 dermatome β€” the umbilicus. Then RLQ when inflammation reaches the parietal peritoneum, which gives sharp, localized somatic pain. Once pain localizes, perforation is closer.
Diagnostics
  • CBC: WBC 10–18K with left shift; > 20K suggests perforation
  • CRP elevated
  • US first in kids (no radiation); CT if US equivocal
  • UA to rule out UTI / pyelonephritis (can mimic)
Treatment

Appendectomy + IV antibiotics. NPO, fluids, pain management. Perforated appendicitis may need drainage and longer abx course.

🎯 Board pearl Sudden relief of pain before surgery = perforation, not improvement. Pressure releases when the appendix bursts β€” then diffuse peritonitis follows.

Constipation & Encopresis

GI Β· CHRONIC Β· FUNCTIONAL IN > 95%
Pathophysiology

A painful stool (illness, dehydration, toilet training, dietary change) β†’ child withholds β†’ stool sits in the rectum β†’ water is reabsorbed β†’ stool becomes harder β†’ next BM hurts more β†’ withholding cycle. Over time, the rectum distends and loses sensation; liquid stool leaks around the impaction = encopresis (involuntary soiling).

Clinical presentation
  • Infrequent, painful, hard stools; straining; blood-streaked stool (anal fissure)
  • Posturing (heel-standing, stiffening) that parents often misread as trying to poop β€” actually withholding
  • Abdominal pain, decreased appetite
  • Encopresis in older children (after age 4)
  • Palpable stool mass in LLQ or suprapubic area
Why encopresis is not "bad behavior" The distended rectum physically loses its ability to sense fullness. Liquid stool slides around the impaction without warning. Shaming the child makes withholding worse and treatment harder.
Red flags (r/o organic cause)
  • Onset in neonate or delayed passage of meconium > 48 hrs β†’ Hirschsprung
  • FTT, bilious vomiting, ribbon stools, tight empty rectum on exam
  • Spine abnormalities (tuft of hair, dimple) β†’ tethered cord
  • No response to aggressive therapy
Treatment

Step 1 β€” Disimpaction: PEG 3350 (Miralax) 1–1.5 g/kg/day Γ— 3–6 days, or enemas.

Step 2 β€” Maintenance: PEG 0.4–0.8 g/kg/day for months. Goal: 1–2 soft stools daily until the rectum regains tone.

Step 3 β€” Behavior: toilet sits 5–10 min after meals (takes advantage of gastrocolic reflex), reward the sitting not the producing, fiber + fluids.

Why PEG works (and why it takes months) PEG is an osmotic laxative β€” it pulls water into the colon, keeping stool soft so the child relearns that pooping doesn't hurt. The stretched rectum needs months to shrink back and regain normal sensation. Stopping PEG too early = relapse.

GERD (vs. Physiologic Reflux)

GI Β· CHRONIC
Pathophysiology

Lower esophageal sphincter (LES) tone is immature in infants; transient LES relaxations allow gastric contents to reflux. Physiologic reflux ("happy spitter") = normal; resolves by 12–18 months as LES matures. GERD = reflux causing complications β€” esophagitis, poor growth, respiratory symptoms.

Clinical presentation
  • Physiologic: effortless spit-up, thriving, happy
  • GERD: poor weight gain, feeding refusal, arching/irritability with feeds, recurrent aspiration/pneumonia, apnea, Sandifer syndrome (back arching)
  • Older kids: heartburn, regurgitation, chest/epigastric pain, nocturnal cough
Why it self-resolves in most infants As the infant grows, the esophagus lengthens, the LES matures and tightens, they spend more time upright, and solid foods replace liquids. By 12–18 months, the mechanical and developmental issues resolve.
Diagnostics

Clinical. Consider upper GI (r/o anatomic β€” malrotation, pyloric stenosis), pH probe or impedance study for atypical presentations, endoscopy if esophagitis suspected.

Treatment

Conservative first: thickened feeds (rice cereal), smaller more frequent feeds, upright 20–30 min after, left-side positioning, elevate head of bed (older kids), avoid tobacco smoke exposure.

Pharm (only if true GERD): H2 blocker (famotidine) or PPI (omeprazole). Avoid empiric PPIs in happy spitters β€” no benefit, and they increase risk of infection and fracture.

Functional Abdominal Pain

GI Β· CHRONIC Β· ROME IV
Pathophysiology

Visceral hypersensitivity β€” the gut-brain axis is dysregulated. Normal gut distention or motility is perceived as painful. Often worsened by stress, anxiety, and poor sleep. Not psychosomatic, not "faking" β€” the pain is real but without structural disease.

Clinical presentation
  • Recurrent abdominal pain, often periumbilical, > 2 months
  • No weight loss, no nocturnal pain, no blood, normal growth
  • Normal physical exam, normal labs
  • Often in 5–12 year olds; school avoidance pattern
🚨 Red flags that make it NOT functional Pain that wakes from sleep · blood in stool · weight loss · FTT · fever · joint pain · perianal disease · family hx IBD · vomiting (esp. bilious) · dysphagia · pain far from umbilicus
Diagnostics

Limited workup: CBC, ESR/CRP, CMP, UA, celiac screen, stool studies if diarrhea. Extensive workup is usually low-yield and reinforces illness behavior.

Treatment

Validation + school attendance + CBT + regular meals, sleep, exercise. Treat anxiety/depression if present. Peppermint oil helpful for IBS-type. Avoid extensive workup β€” it reinforces the sick role.

Why "the pain is real" matters Dismissing the pain ("there's nothing wrong") makes it worse. Families need to hear: "This pain is real. Your gut is hypersensitive. We're going to help you function with it, not search for a disease that isn't there." That reframe is therapeutic.

Urinary Tract Infection

GU Β· INFECTION Β· E. COLI IN > 80%
Pathophysiology

Ascending bacterial colonization from the perineum β†’ urethra β†’ bladder (cystitis) β†’ ureters/kidneys (pyelonephritis). E. coli is the dominant pathogen; others include Klebsiella, Proteus, Enterococcus. Girls are at higher risk (short urethra, proximity to anus); uncircumcised boys in the first year.

Clinical presentation
  • Infants: fever without a source, poor feeding, irritability, vomiting, jaundice (newborns)
  • Toddlers/preschool: fever, abdominal pain, new-onset enuresis
  • School-age: classic dysuria, urgency, frequency, suprapubic pain
  • Pyelonephritis: high fever, flank/CVA tenderness, vomiting, ill-appearing
Why infants present with just fever Infants can't localize or verbalize pain. Fever without a source in any child < 2 years should trigger UTI workup β€” a "normal" exam doesn't rule it out. AAP: obtain UA + Cx in febrile infants 2–24 mo without a clear source.
Diagnostics
  • Cath UA + Cx in pre-continent children (bag specimens = 85% false positive β€” skin contamination)
  • Midstream clean catch if toilet-trained
  • Positive UA: leukocyte esterase, nitrites, WBC > 5/hpf
  • Positive Cx: β‰₯ 50,000 CFU/mL from cath, β‰₯ 100,000 from clean catch
  • After first febrile UTI in < 24 mo: renal/bladder ultrasound
  • VCUG: only if recurrent febrile UTIs or abnormal US
Why nitrites matter Gram-negative enteric bacteria (like E. coli) reduce urinary nitrates to nitrites. A positive nitrite is very specific for UTI but not sensitive β€” needs bacteria to sit in the bladder for 4+ hrs, so frequent voiders can have false negatives. LE is more sensitive but less specific.
Treatment

Outpatient: cephalexin, cefixime, TMP-SMX, or nitrofurantoin Γ— 7–14 days (longer for febrile/pyelo). Admit if: < 2 mo old, toxic, vomiting, dehydrated, immunocompromised, or failing outpatient therapy.

Vesicoureteral Reflux (VUR)

GU Β· STRUCTURAL
Pathophysiology

Normally, ureters enter the bladder at an oblique angle β€” bladder pressure during voiding closes the ureteral orifice. In VUR, the ureter-bladder junction is abnormal (short tunnel) and urine flows backward up the ureter during voiding. Primary VUR = congenital anatomic defect. Retrograde urine carries bacteria to kidneys β†’ pyelonephritis β†’ renal scarring β†’ HTN and CKD long-term.

Clinical presentation

Often silent β€” diagnosed after a child presents with febrile UTI. Suspect in any child with recurrent febrile UTIs or pyelonephritis.

Grading (I–V)
  • I: reflux into distal ureter only
  • II: into renal pelvis, no dilation
  • III: mild dilation
  • IV: moderate dilation + blunting of calyces
  • V: severe dilation, tortuous ureter, intrarenal reflux
Diagnostics

VCUG = gold standard; performed after recurrent febrile UTI or abnormal renal US.

Treatment

Low grade (I–III): often spontaneously resolves as the child grows. Observation Β± prophylactic antibiotics (amoxicillin or TMP-SMX).

High grade (IV–V): surgical correction (ureteral reimplantation, Deflux injection).

Why we care Repeated pyelonephritis in a reflux kidney causes permanent renal scarring β†’ HTN and CKD in adulthood. Preventing febrile UTIs in the first 5 years matters for lifetime renal health.

Nocturnal Enuresis

GU Β· BEHAVIORAL / DEVELOPMENTAL
Pathophysiology

Three mechanisms β€” often combined:

  • Delayed arousal: child doesn't wake to a full bladder
  • Nocturnal polyuria: insufficient nighttime ADH release β†’ large urine volumes
  • Small functional bladder capacity

Primary enuresis: child was never dry (most common). Secondary: dry β‰₯ 6 mo, now wetting β€” look for trigger (UTI, new stressor, DM, constipation, OSA, abuse).

Clinical presentation

Involuntary nighttime wetting in a child β‰₯ 5 years, at least 2Γ—/week for 3 months, not explained by a medical condition. Strong family history (if both parents were bedwetters, child has ~75% risk).

Diagnostics

UA to rule out UTI, DM, DI. History focused on daytime symptoms (if daytime wetting or dysfunctional voiding β†’ different workup). Screen for constipation (hugely underdiagnosed contributor) and OSA.

Treatment

Age < 6–7: reassure, avoid treatment β€” most outgrow it.

Age β‰₯ 6–7 when child is bothered:

  • First-line: bedwetting alarm (60–70% cure; relapse low)
  • DDAVP (desmopressin): for short-term use (sleepovers, camp). Works by mimicking ADH β€” reduces nighttime urine production
  • Treat any constipation or OSA first β€” often fixes it
  • Motivation + bladder-stretching exercises
Why the alarm works (and takes time) The alarm conditions the brain to associate bladder fullness with arousal. It takes 8–12 weeks of consistent use β€” parents must wake with the child every time. Stopping after a few weeks = failure. The alarm teaches the brain, which is why the effect is durable after it's discontinued.

Labial Adhesions

GU Β· FEMALE Β· 3 MO – 6 YRS
Pathophysiology

In the hypoestrogenic state between infancy and puberty, inflammation of the vulvar skin (from chronic moisture, diapers, mild irritation) causes the labia minora to stick together midline. The fusion is epithelial, not fibrous.

Clinical presentation

Midline raphe where labia minora fuse. Usually asymptomatic and incidental. Occasional complications: post-void dribbling, UTI, vulvitis.

Treatment
  • Asymptomatic: observation. Resolves at puberty when estrogen rises.
  • Symptomatic: topical estrogen cream to the raphe nightly Γ— 2–6 weeks. Betamethasone cream is an alternative.
  • Never forcibly separate β€” traumatic and will re-adhere.
Why estrogen works Estrogen thickens the vulvar epithelium and loosens the epithelial adhesions. It's topical, locally acting, and brief use is safe. Watch for transient breast budding or vulvar pigmentation with prolonged use.

Vulvovaginitis (Prepubertal)

GU Β· FEMALE Β· MOST COMMON GYN COMPLAINT
Pathophysiology

The prepubertal vagina is hypoestrogenic (thin, alkaline epithelium, no protective lactobacilli), making it vulnerable to irritants and infections. Most cases are nonspecific β€” from poor perineal hygiene, wiping back-to-front, bubble baths, tight clothing, soaps.

Clinical presentation
  • Vulvar erythema, itching, burning, dysuria
  • Discharge (usually minimal in nonspecific; thick/purulent suggests infection)
  • Consider foreign body (toilet paper) if foul, bloody discharge
  • Consider group A strep if beefy erythema (esp. after strep pharyngitis)
  • Consider pinworms if nocturnal itching
  • Consider sexual abuse if STI pathogen isolated
Treatment

Hygiene education (front-to-back wiping, cotton underwear, no bubble baths, loose clothing, sitz baths). Treat specific infections as identified. Candida is uncommon prepubertally β€” don't reach for fluconazole reflexively.

Cryptorchidism (Undescended Testis)

GU Β· MALE Β· MOST COMMON MALE GU DISORDER
Pathophysiology

The testes normally descend from the abdominal cavity through the inguinal canal into the scrotum between 7–9 months gestation. Descent can arrest anywhere along this path. ~3% of term and 30% of preterm males have undescended testes at birth; 75% descend spontaneously by 3–6 months.

Clinical presentation

Empty or small hemiscrotum; testis may be palpable in the inguinal canal or not palpable at all. Bilateral non-palpable testes β†’ workup for DSD.

Retractile testis (cremaster pulls testis up but it returns to scrotum) is normal β€” differentiate carefully.

Treatment

Watchful waiting until 6 months (most descend by then). Refer to urology at 6 months if still undescended. Orchiopexy by 12–18 months.

Why the 12–18 month window matters The testis needs the cooler scrotal environment for spermatogenesis. Body-temperature testes cause irreversible germ cell loss after about 12–18 months. Even after surgical correction, fertility can be impaired β€” so early surgery matters. Also: undescended testes have 4–10Γ— lifetime risk of germ cell tumors, and scrotal positioning allows self-exam for early detection.
🎯 Board pearl Never tell a parent "it'll descend eventually" beyond 6 months β€” that window closes. Refer by 6 months, operate by 12–18 months.
MODULE 9 Β· CHAPTERS 31 & 32 Β· 12 QUESTIONS

Musculoskeletal & Genetic Conditions

9 MSK Β· 3 Genetics

Developmental Dysplasia of the Hip (DDH)

MSK Β· PACKAGING DEFORMITY Β· NEWBORN SCREENING
Pathophysiology

A spectrum of abnormal hip development β€” from mild acetabular dysplasia (shallow socket) to frank dislocation. The femoral head and acetabulum are a complementary pair; each shapes the other during development. If the femoral head sits outside the socket, the acetabulum doesn't form properly β†’ progressive dysplasia β†’ early osteoarthritis if untreated.

Risk factors ("6 Fs")
  • Female (4–6Γ— more common β€” maternal estrogens loosen ligaments more in female fetuses)
  • First-born (tight uterus)
  • Family history
  • Frank breech (most significant β€” hip held flexed + externally rotated in utero)
  • Fluid (oligohydramnios)
  • "Footling" and other non-vertex positions
Clinical exam
  • Barlow (dislocates a dislocatable hip): adduct + push posteriorly β€” feel "clunk" OUT
  • Ortolani (relocates a dislocated hip): abduct + lift anteriorly β€” feel "clunk" IN
  • Older infant (> 3 mo): Barlow/Ortolani become negative; look for asymmetric thigh/gluteal folds, limited abduction, Galeazzi sign (knees at unequal heights when hips flexed β€” short femur on affected side)
  • Walking child: Trendelenburg gait, waddling (bilateral)
Why the exam changes with age In the newborn, the hip is reducible β€” you can pop it in and out. By 3 months, soft tissues tighten and the hip becomes fixed β€” Barlow/Ortolani disappear. That's why early detection matters: a hip that's reducible responds beautifully to a Pavlik harness; a fixed hip needs surgery.
Diagnostics
  • < 4–6 months: ultrasound (femoral head is mostly cartilage β€” not yet ossified β†’ invisible on X-ray)
  • > 4–6 months: X-ray (femoral head ossification center appears)
  • Universal US screening in breech girls and high-risk infants at 4–6 weeks
Treatment
  • < 6 months: Pavlik harness β€” holds hips flexed and abducted (the "frog-leg" position that encourages proper socket formation)
  • 6–18 months: closed reduction + spica cast
  • > 18 months: open reduction Β± femoral/pelvic osteotomy
Why Pavlik works The harness holds the femoral head centered in the acetabulum. Because each half of the joint shapes the other, proper positioning during growth remodels both the head and the socket into normal anatomy. No surgery needed if caught early.

Talipes Equinovarus (Clubfoot)

MSK Β· PACKAGING Β· "TALI-PES EQUINO-VARUS" = "TOE-FOOT HORSE-INWARD"
Pathophysiology

A complex congenital foot deformity with four components (remember CAVE):

  • Cavus (high arch)
  • Adductus (forefoot turned inward)
  • Varus (hindfoot inverted)
  • Equinus (ankle pointed down β€” "horse-like")

Soft tissues (tendons, ligaments) are short on the medial and posterior side; bones are malpositioned. Can be isolated (idiopathic) or syndromic (spina bifida, arthrogryposis).

Clinical presentation

Obvious at birth β€” foot looks like a golf club. Rigid (true clubfoot) vs flexible (positional β€” corrects passively; benign).

Treatment

Ponseti method β€” serial casting starting in the first 1–2 weeks of life. Weekly manipulation + casting for 5–8 weeks β†’ percutaneous Achilles tenotomy β†’ bracing (boots-and-bar) for several years to prevent recurrence.

Why start so early Infant soft tissues are highly plastic and remodel easily. The Ponseti method has > 90% success without major surgery if started early. Delay = rigid deformity requiring surgical release.

Osgood-Schlatter Disease

MSK Β· OVERUSE Β· ADOLESCENT ATHLETE
Pathophysiology

Traction apophysitis of the tibial tubercle. Repetitive quadriceps contraction pulls on the patellar tendon, which inserts on the tibial tubercle β€” an active growth plate in adolescents. Repeated traction causes microavulsion, inflammation, and bony prominence.

Clinical presentation
  • 10–15 yo, active boy (or girl) in jumping/running sports (basketball, soccer, gymnastics)
  • Anterior knee pain, worse with activity
  • Tender, enlarged tibial tubercle
  • Pain reproduced with resisted knee extension
Why adolescents specifically The tibial tubercle apophysis is cartilaginous in pre-teens. During the growth spurt, the quadriceps muscle grows faster than the tendon/bone, creating increased traction right where the tendon inserts on a fragile growing apophysis. After skeletal maturity (apophysis fuses), Osgood-Schlatter can't happen.
Treatment

Self-limited β€” resolves when growth plate closes. Ice, NSAIDs, quad stretching, activity modification (not full rest β€” partial is fine). Patellar strap may help. Residual bony prominence may persist.

🎯 Board pearl Osgood-Schlatter = tibial tubercle. Sinding-Larsen-Johansson = inferior pole of patella (same mechanism, different site). Sever disease = calcaneal apophysitis in younger child (heel pain in runners age 8–12).

Nursemaid's Elbow (Radial Head Subluxation)

MSK Β· DISLOCATION Β· TODDLER
Pathophysiology

Sudden axial traction on a pronated, extended arm (adult pulls child by the hand) slips the annular ligament partially off the radial head. The ligament gets trapped in the radiohumeral joint. Peak age 1–4 yrs β€” after age 5 the annular ligament thickens and it becomes rare.

Clinical presentation
  • Toddler who refuses to use one arm, holds it slightly flexed and pronated at side
  • Cries only when the arm is moved; otherwise seems fine
  • No swelling, no deformity, no bruising
  • Classic history: "I pulled him up by the hand" or "he slipped off the couch while I was holding his hand"
Diagnostics

Clinical. X-ray not needed if history is classic β€” just reduce. Get X-rays if history inconsistent, swelling, or deformity (r/o fracture, abuse).

Treatment

Reduction: supinate and flex (or hyperpronate) β€” feel a click. Child uses the arm within 10–15 min. No immobilization needed.

Why this matters for board stems Classic scenario: toddler + "not using arm" + mechanism involving a pull. Do not X-ray first β€” reduce first. Also: counsel parents to lift under the armpits, not by the hand.

Idiopathic Toe Walking

MSK Β· LOWER EXTREMITY Β· AGE < 3 NORMAL
Pathophysiology & workup

Walking on the balls of the feet without heel strike. Common and benign in children under 3 as part of gait development. Persistent toe walking > 3 yrs needs evaluation to rule out:

  • Cerebral palsy (hyperreflexia, spasticity)
  • Muscular dystrophy (Gower sign, calf hypertrophy, CK elevation)
  • Tethered cord (spine findings)
  • Autism (associated sensory/behavioral findings)
  • Short Achilles tendon (limited passive dorsiflexion)
Treatment

Idiopathic: observation, stretching, heel-cord exercises. Serial casting or botulinum toxin if persistent/painful. Surgery (tendon lengthening) rare.

Legg-CalvΓ©-Perthes Disease (LCPD)

MSK Β· HIP Β· IDIOPATHIC AVASCULAR NECROSIS
Pathophysiology

Idiopathic avascular necrosis of the femoral head. Blood supply to the ossification center is interrupted β†’ bone dies β†’ over months, bone is resorbed β†’ new bone remodels (reossification). The softened head can deform during this vulnerable phase, leading to a misshapen hip and early osteoarthritis.

Clinical presentation
  • Boys 4–8 years (5:1 M:F), typically short, thin, and very active
  • Painless limp or intermittent pain referred to the knee, thigh, or groin
  • Limited hip abduction and internal rotation
  • Bilateral in ~15%
  • Delayed bone age
Why knee pain = look at the hip The obturator and femoral nerves supply both the hip joint and the skin over the medial thigh/knee. Hip pain refers to the knee via these shared nerve roots. Every child with knee pain needs a hip exam (and vice versa). This is one of the review's most emphasized points.
Diagnostics

X-ray: AP and frog-leg lateral. Findings evolve β€” early: increased femoral head density, joint space widening. Later: fragmentation, flattening. MRI if X-ray normal but suspicion high.

Treatment

Goal: containment β€” keep the femoral head seated in the acetabulum during healing so it remodels round. NSAIDs, activity restriction, PT for range of motion. Bracing or surgery (osteotomy) for more severe cases.

🎯 Board pearl Review said: "Leg-CalvΓ©-Perthes disease typically affects short, active boys with a delay in bone age." Classic test clue is the young active boy with a painless limp or knee pain β€” X-ray the hip.

Slipped Capital Femoral Epiphysis (SCFE)

MSK Β· HIP Β· ADOLESCENT Β· URGENT
Pathophysiology

During the adolescent growth spurt, the femoral head "slips" posteriorly and medially off the femoral neck at the growth plate (physis), like a scoop of ice cream sliding off a cone. Mechanical weakness + hormonal factors during puberty weaken the physis.

Who gets it
  • Obese adolescents, typically 10–15 years old
  • Boys > girls, African American children at higher risk
  • Endocrine associations: hypothyroidism, growth hormone therapy, renal osteodystrophy (think about SCFE in short/young-looking or unusual stature kids)
  • Bilateral in ~40% β€” screen the other side
Clinical presentation
  • Hip, groin, thigh, or KNEE pain (often only knee pain!)
  • Limp, antalgic gait
  • Leg held in external rotation when hip flexed (pathognomonic β€” the displaced head forces external rotation)
  • Limited internal rotation and abduction
  • Stable: able to bear weight. Unstable: cannot bear weight β€” high risk of AVN.
Why external rotation happens The femoral head slips posteriorly, so the femoral neck rotates anteriorly β€” when the hip flexes, the whole leg has to rotate externally to accommodate the displacement. This "obligate external rotation on flexion" is a classic SCFE exam finding.
Diagnostics

Bilateral AP + frog-leg lateral X-rays. Look for widening/lucency of the physis (pre-slip) or the "ice cream slipping off the cone" sign. Draw Klein's line along the superior femoral neck β€” it should cross some of the femoral head. If it doesn't cross, think SCFE.

Treatment

Urgent orthopedic referral. Non-weight-bearing on crutches immediately. In situ screw fixation β€” one screw through the femoral neck into the head prevents further slippage while the physis fuses.

🎯 Board pearl Overweight adolescent with knee or thigh pain and a limp = think SCFE until proven otherwise. Non-weight-bearing, XR both hips, urgent ortho. Delay = avascular necrosis.

Osteomyelitis

MSK Β· INFECTIOUS Β· S. AUREUS DOMINANT
Pathophysiology

Bacterial infection of bone, almost always from hematogenous seeding in children (bacteremia β†’ bone). The metaphysis of long bones has sluggish blood flow and abundant vessels β€” bacteria get trapped there. Infection spreads into bone, forms an abscess, and can track to the joint (especially in infants whose metaphysis is intracapsular at the hip).

S. aureus is the dominant pathogen across all ages. Others: Group A strep, Kingella (< 5 yrs), Salmonella (in sickle cell), Pseudomonas (puncture wound through sneaker).

Clinical presentation
  • Fever + focal bone pain + refusal to use/bear weight on the limb
  • Point tenderness over the bone β€” cardinal feature that differentiates from joint infection
  • Warmth, erythema, swelling over the affected bone
  • Limp if in the lower extremity
Diagnostics
  • CBC with diff, ESR, CRP β€” all elevated. CRP rises within 6–8 hrs and trends with response.
  • Blood culture β€” positive in ~50%
  • X-ray β€” normal in first 7–10 days (bone changes lag behind). Useful to r/o fracture or tumor.
  • MRI β€” study of choice; shows marrow edema within days
  • Bone aspiration/biopsy for definitive organism + sensitivities
Why CRP matters more than ESR CRP rises and falls quickly (6–8 hrs to peak, normalizes within days of treatment). ESR is sluggish β€” takes weeks to normalize. CRP is the better real-time marker of response to treatment.
Treatment

IV antibiotics covering S. aureus (MSSA: cefazolin; MRSA coverage: clindamycin or vancomycin), usually 4–6 weeks total (can transition to PO after clinical response). Surgical drainage if abscess, failure to respond, or joint involvement.

Transient Synovitis (Toxic Synovitis)

MSK Β· INFLAMMATORY Β· POST-VIRAL
Pathophysiology

Self-limited inflammation of the hip synovium, most often post-viral (URI in the preceding 1–2 weeks) or reactive. Most common cause of hip pain in children 3–10 years. Exactly the scenario from Case Study #1.

Clinical presentation
  • 3–10 yo, recent URI or viral illness
  • Acute hip pain, limp, or refusal to bear weight
  • Hip held flexed, abducted, externally rotated β€” position of lowest intracapsular pressure
  • Afebrile or low-grade fever (high fever suggests septic arthritis)
  • Not systemically ill β€” this is the key clinical distinction from septic arthritis
Why that hip position Flexion + abduction + external rotation maximizes the intracapsular volume β€” giving the inflamed, swollen joint the most room. Any forced motion that reduces that volume (extension, adduction, internal rotation) compresses the inflamed tissue and hurts. The position itself is a physiologic pressure-relief valve.
Kocher criteria β€” septic arthritis vs. transient synovitis

Four predictors of septic arthritis:

  • History of fever (> 38.5Β°C)
  • Non-weight-bearing
  • ESR β‰₯ 40
  • WBC > 12,000

All 4 present β†’ ~99% probability of septic arthritis. 0 present β†’ < 0.2%. Add CRP > 2.0 mg/dL for improved sensitivity (rises within 6–8 hrs of inflammation).

Diagnostics & workup
  • X-ray hip (AP + frog-leg) β€” usually normal; r/o other causes
  • CBC with diff, ESR, CRP, blood Cx
  • Hip US Β± aspiration if septic arthritis suspected β€” effusion > 2 mm suspicious; aspirate and send fluid for cell count, Gram stain, culture
Treatment

Rest + ibuprofen 7–10 days. Often improves within 24–48 hrs. Follow up in 24 hrs if uncertain β€” septic arthritis worsens while transient synovitis improves. Return immediately for high fever, worsening pain, or inability to move the joint.

🎯 Board pearl (directly from review) "Children with bacterial infection will rapidly worsen, and children with synovitis will often improve over time. In general, children with septic arthritis appear more acutely ill than those with toxic synovitis." If you can watch the child for 12–24 hrs, the trajectory itself makes the diagnosis.

Down Syndrome (Trisomy 21)

GENETIC Β· CHROMOSOMAL Β· MOST COMMON AUTOSOMAL TRISOMY
Pathophysiology

Three copies of chromosome 21 β€” 95% from maternal meiotic nondisjunction (risk rises with maternal age), 4% from translocation, 1% mosaic. Extra dose of chromosome 21 genes causes the multisystem phenotype.

Clinical features
  • Facies: upslanting palpebral fissures, epicanthal folds, flat nasal bridge, small ears, protruding tongue (relative macroglossia), brachycephaly
  • Hands: single transverse palmar crease, short fifth finger with clinodactyly, wide gap between 1st and 2nd toes ("sandal gap")
  • Tone: hypotonia (the "floppy baby")
  • Growth: short stature, use Down-specific growth charts
  • Intellectual disability (mild to moderate usually)
Medical surveillance ("everything is at risk")
  • Cardiac: ~50% have CHD β€” AV canal (AVSD) most characteristic; also VSD, ASD, tetralogy. Echo at birth.
  • GI: duodenal atresia (double-bubble sign), Hirschsprung, celiac disease
  • Thyroid: hypothyroidism (congenital + acquired) β€” screen at birth, 6 mo, 12 mo, then annually
  • Hematologic: 10–20Γ— risk of leukemia (ALL and AML); transient myeloproliferative disorder in newborns
  • Atlantoaxial instability β€” screen before contact sports; avoid cervical manipulation
  • Hearing & vision: OME, cataracts, refractive errors β€” screen regularly
  • OSA β€” polysomnography by age 4
  • Alzheimer disease β€” early onset (chromosome 21 carries APP gene)
Why Alzheimer's? The APP gene (amyloid precursor protein) is on chromosome 21. Three copies = lifelong overproduction of amyloid β†’ nearly universal early-onset Alzheimer's disease by age 50–60. This is a powerful window into sporadic Alzheimer's biology.
Management

Early intervention (PT/OT/ST), educational support, treatment of comorbidities, regular surveillance per AAP guidelines. Genetic counseling for family.

Marfan Syndrome

GENETIC Β· MENDELIAN Β· AUTOSOMAL DOMINANT Β· FBN1
Pathophysiology

Mutation in FBN1 (fibrillin-1), a connective tissue protein that provides structural support in elastic tissues (aorta, lens zonules, periosteum, skin). Defective fibrillin weakens these tissues β†’ aortic dilation, lens subluxation, tall stature with long limbs.

Clinical features
  • Skeletal: tall, long limbs (dolichostenomelia), arachnodactyly (long spider-like fingers), pectus excavatum or carinatum, scoliosis, high-arched palate, joint hypermobility
  • Positive wrist sign (Walker-Murdoch β€” thumb and 5th finger overlap when gripping wrist) and thumb sign (Steinberg β€” thumb protrudes past ulnar border of fist)
  • Cardiovascular: aortic root dilation β†’ aneurysm β†’ dissection (main cause of death). Mitral valve prolapse.
  • Ocular: ectopia lentis (upward lens subluxation), myopia, retinal detachment
Why "up and out" lens subluxation Fibrillin forms the zonular fibers that suspend the lens. When fibrillin is defective, zonular fibers stretch β€” the lens tilts and shifts. In Marfan, zonular failure is typically superior, so the lens drops upward. (In homocystinuria, it drops down and in β€” "HOmocystinuria = HO lens goes down.")
Diagnostics (Revised Ghent criteria)

Echocardiogram (aortic root dimension, Z-score), slit lamp, genetic testing (FBN1). Family history key.

Management
  • Annual echo to monitor aortic root
  • Beta blockers (atenolol) or ARBs (losartan) to slow aortic dilation
  • Avoid contact sports and isometric/max-effort activities (weightlifting) β€” aortic dissection risk
  • Prophylactic aortic root replacement when diameter reaches threshold
  • Annual eye exam; genetic counseling
🎯 Board pearl Tall adolescent with pectus, arachnodactyly, and a murmur β†’ echo before the sports physical. Cleared with beta blocker; excluded from contact/max-effort sports. This is classic PPE content.

Fragile X Syndrome

GENETIC Β· X-LINKED Β· MOST COMMON INHERITED ID
Pathophysiology

CGG trinucleotide repeat expansion in the FMR1 gene on the X chromosome. Normal: < 45 repeats. Premutation: 55–200. Full mutation: > 200 β†’ gene is silenced (methylated) β†’ no FMRP protein β†’ abnormal synaptic plasticity. Anticipation β€” the repeat expands from generation to generation, so symptoms worsen across generations.

Clinical features
  • Intellectual disability (most common inherited cause; Down is most common overall but not inherited)
  • Autism features (Fragile X is one of the most common identifiable genetic causes of autism)
  • Facies: long face, prominent jaw, large ears (often protruding)
  • Macroorchidism β€” large testes, emerges post-pubertally
  • Joint hypermobility, flat feet, mitral valve prolapse
  • Anxiety, hyperactivity, gaze aversion, stereotyped hand movements, tactile defensiveness
Why boys are more affected Fragile X is X-linked. Males have one X β†’ full mutation fully expresses. Females have a second X β†’ often partially compensated; they tend to have milder ID, learning disabilities, and anxiety. Premutation carriers can develop FXTAS (tremor/ataxia) in adulthood and premature ovarian insufficiency.
Diagnostics

DNA testing for CGG repeat number (direct gene testing). Recommended for any child with unexplained ID, developmental delay, or autism.

Management

Early intervention, educational support, SSRI for anxiety, stimulants for ADHD symptoms, treat comorbid epilepsy. Genetic counseling for family β€” mother is at minimum a carrier; sisters, aunts, cousins need testing.

MODULE 10 Β· CHAPTERS 27 & 37 Β· 10 QUESTIONS

Nutritional & Endocrine Conditions

4 Nutritional Β· 6 Endocrine

Food Allergy

NUTRITIONAL Β· IgE-MEDIATED HYPERSENSITIVITY
Pathophysiology

IgE-mediated (immediate): prior sensitization β†’ food-specific IgE on mast cells/basophils β†’ re-exposure β†’ crosslinking β†’ histamine and cytokine release within minutes to 2 hours. Non-IgE-mediated: cell-mediated, delayed (hours to days) β€” e.g., FPIES, eosinophilic esophagitis, milk protein proctocolitis.

Top 9 allergens: milk, egg, peanut, tree nuts, soy, wheat, fish, shellfish, sesame.

Clinical presentation (IgE-mediated)
  • Skin: urticaria, angioedema, flushing, pruritus
  • GI: vomiting, abdominal pain, diarrhea
  • Respiratory: wheeze, stridor, cough
  • Cardiovascular: hypotension, tachycardia (anaphylaxis)
  • Onset within minutes, peaks within 2 hours
Why "two systems = anaphylaxis" Anaphylaxis is defined by involvement of two or more organ systems (skin, GI, resp, cardiovascular) OR hypotension alone after exposure to a known allergen. Epinephrine IM (thigh) immediately β€” do not delay for antihistamines. Antihistamines treat hives; they don't treat airway or blood pressure.
Diagnostics
  • Skin prick test or food-specific IgE (RAST/ImmunoCAP) β€” support diagnosis but don't diagnose alone (false positives common)
  • Oral food challenge = gold standard (in a controlled setting)
  • Detailed history is most important β€” timing, reproducibility, dose
Treatment
  • Avoidance of the trigger
  • Epinephrine auto-injector Γ— 2 (always prescribe two β€” biphasic reactions)
  • Action plan for home, school, caregivers
  • Early introduction of peanut in high-risk infants (LEAP trial) β€” starting 4–6 months reduces peanut allergy
  • Consider oral immunotherapy for selected patients

Celiac Disease

NUTRITIONAL Β· AUTOIMMUNE Β· GLUTEN-TRIGGERED
Pathophysiology

In genetically predisposed children (HLA-DQ2 or HLA-DQ8), ingestion of gluten (wheat, barley, rye) triggers an autoimmune reaction in the small intestine. Gluten peptides are modified by tissue transglutaminase (tTG) β†’ presented to T cells β†’ inflammation β†’ villous atrophy β†’ malabsorption. The damage is entirely reversible with strict gluten avoidance.

Clinical presentation
  • Classic (infants/toddlers after gluten introduction): chronic diarrhea, abdominal distension, FTT, irritability
  • Older children: short stature, delayed puberty, iron-deficiency anemia refractory to iron, dental enamel defects, dermatitis herpetiformis (grouped pruritic vesicles on elbows/knees)
  • Silent celiac: asymptomatic but serology +, damage present
  • Associated conditions: T1DM, Down syndrome, Turner syndrome, autoimmune thyroiditis, IgA deficiency, first-degree relatives
Why all those GI symptoms Villous atrophy reduces the intestinal surface area 10- to 100-fold β†’ malabsorption of fats (steatorrhea), iron, B12, folate, calcium, vitamin D. Short stature and delayed puberty are the growth consequences of chronic malnutrition. Dermatitis herpetiformis is the same autoimmune reaction, just deposited in skin.
Diagnostics
  • tTG-IgA + total IgA (check total IgA because IgA deficiency is common in celiac and would give a false negative tTG-IgA)
  • If IgA deficient: use tTG-IgG or DGP-IgG
  • Gold standard: duodenal biopsy showing villous atrophy + crypt hyperplasia + intraepithelial lymphocytes
  • Must be on gluten-containing diet when testing β€” gluten-free diet normalizes labs and biopsy
Treatment

Strict lifelong gluten-free diet. Referral to dietitian essential. Screen for nutrient deficiencies (iron, vitamin D, folate, B12) and treat. Monitor growth and tTG to confirm adherence. Screen first-degree relatives.

Malnutrition / Failure to Thrive

NUTRITIONAL Β· GROWTH FAILURE
Definition & pathophysiology

Inadequate nutritional intake, absorption, or utilization for the child's growth needs. Defined as:

  • Weight-for-age < 5th percentile
  • Weight-for-length < 5th percentile
  • Downward crossing of β‰₯ 2 major percentile lines

Categorized as: inadequate intake (most common β€” feeding problems, poverty, neglect, food refusal), inadequate absorption (celiac, CF, milk protein allergy), increased metabolic demand (CHD, chronic infection, hyperthyroidism), or defective utilization (metabolic disorders).

Clinical approach
  • Detailed feeding history β€” what, how much, how often, who feeds, mealtime behavior
  • Plot growth on standardized curves β€” pattern often reveals cause (head circumference spared suggests nutritional; all parameters affected suggests genetic/structural)
  • Observe a feeding
  • Psychosocial assessment β€” caregiver mental health, food security, family stress
Why head circumference matters In nutritional (simple) malnutrition, the body conserves the brain β€” HC is preserved while weight drops first, then length. In intrauterine or genetic causes, all three (HC, weight, length) are affected from the start. The pattern of the growth curve alone can narrow your differential.
Treatment

Address underlying cause. Increase caloric density (add fats, oils; concentrate formula carefully). Feeding therapy. Multidisciplinary approach β€” pediatrician, dietitian, social work, behavioral health. Admit if severe, dehydrated, or social concerns. Watch for refeeding syndrome (hypophosphatemia, hypokalemia, hypomagnesemia) in severely malnourished children.

Pediatric Obesity

NUTRITIONAL Β· EPIDEMIC Β· MULTIFACTORIAL
Definition
  • Overweight: BMI 85th–94th percentile for age/sex
  • Obesity (class 1): BMI β‰₯ 95th percentile
  • Class 2 obesity: β‰₯ 120% of 95th percentile (or BMI β‰₯ 35)
  • Class 3 (severe): β‰₯ 140% of 95th percentile (or BMI β‰₯ 40)
Pathophysiology

Energy imbalance β€” intake > expenditure, modulated by genetics, early-life programming, sleep, stress, gut microbiome, food environment, and activity. Adipose tissue is an active endocrine organ β€” excess fat produces inflammatory cytokines and disrupts insulin signaling, leading to insulin resistance and the metabolic consequences.

Comorbidities to screen for
  • Metabolic: T2DM, dyslipidemia, HTN, NAFLD
  • Respiratory: OSA, asthma
  • Orthopedic: SCFE, Blount disease (tibia vara)
  • Dermatologic: acanthosis nigricans (marker of insulin resistance)
  • Reproductive: PCOS in adolescent girls, precocious puberty
  • Mental health: depression, anxiety, disordered eating, bullying
  • GI: GERD, gallstones
Screening (starting at age 9–11)
  • Fasting lipid panel
  • ALT (NAFLD screen β€” most common cause of chronic liver disease in kids now)
  • Fasting glucose or HbA1c if BMI β‰₯ 85th + risk factors
  • BP at every visit
Treatment (2023 AAP guidelines)
  • Intensive health behavior and lifestyle treatment (IHBLT) β€” β‰₯ 26 contact hours over 3–12 months, family-based
  • Pharmacotherapy β‰₯ 12 yrs with obesity: metformin, GLP-1 agonists (liraglutide, semaglutide), orlistat
  • Metabolic and bariatric surgery β‰₯ 13 yrs with severe obesity
  • Do not use "watchful waiting" as a default strategy β€” early intervention works better
Why the 2023 shift The previous "wait and see" approach allowed metabolic damage to compound. 2023 AAP guidelines shifted to early, intensive, evidence-based treatment β€” recognizing obesity as a chronic disease, not a behavior failure.

Hypothyroidism

ENDOCRINE Β· THYROID Β· CONGENITAL OR ACQUIRED
Pathophysiology

Congenital hypothyroidism (CH): thyroid dysgenesis (aplasia, ectopy β€” 85%), dyshormonogenesis, transient (maternal antibodies, iodine exposure). Universal newborn screening catches nearly all cases. Untreated CH causes irreversible intellectual disability and growth failure.

Acquired hypothyroidism: most commonly Hashimoto thyroiditis (autoimmune lymphocytic infiltration destroying thyroid tissue). Associated with T1DM, Down syndrome, Turner syndrome, celiac.

Clinical presentation

Congenital (if screening missed or late): large fontanelles, poor feeding, lethargy, constipation, prolonged jaundice, macroglossia, umbilical hernia, cool/mottled skin, hoarse cry, hypotonia.

Acquired: growth failure + delayed bone age, fatigue, cold intolerance, constipation, weight gain (usually modest β€” not as dramatic as adults), dry skin/hair, bradycardia, delayed puberty or precocious pseudopuberty, goiter.

Why growth failure is the flag in kids Thyroid hormone is essential for linear growth. Kids don't usually describe cold intolerance or fatigue β€” but pediatricians see them dropping percentiles on the growth chart. A child whose weight keeps climbing while height plateaus = hypothyroidism until proven otherwise. (Obesity alone tends to cause tall stature from insulin effects.)
Diagnostics
  • Primary hypothyroidism: ↑ TSH, ↓ free T4
  • Central hypothyroidism: ↓ or inappropriately normal TSH, ↓ free T4
  • Subclinical: ↑ TSH, normal free T4
  • Antibodies: anti-TPO, anti-thyroglobulin (Hashimoto)
  • Bone age X-ray β€” delayed in hypothyroidism
Treatment

Levothyroxine daily, on empty stomach. Start full replacement immediately in CH (10–15 mcg/kg/day); lower doses for acquired. Recheck TSH in 4–6 weeks after dose change. Goal: TSH in normal range. Do not take with calcium, iron, or soy (reduces absorption).

Hyperthyroidism

ENDOCRINE Β· THYROID Β· GRAVES DOMINATES IN PEDS
Pathophysiology

Graves disease β€” autoimmune, TSH receptor antibodies (TRAb) stimulate the thyroid β†’ excess T3/T4 + goiter. Most common cause in children, peaks in adolescence, 5:1 female:male. Can also occur in neonates born to mothers with Graves (antibodies cross the placenta β€” transient).

Clinical presentation
  • Weight loss with increased appetite, hyperphagia
  • Heat intolerance, sweating
  • Tachycardia, palpitations, HTN with widened pulse pressure
  • Tremor, anxiety, emotional lability, poor concentration (often misdiagnosed as ADHD)
  • Declining school performance despite hyperactivity
  • Goiter (diffuse, firm, possible bruit)
  • Ophthalmopathy (exophthalmos, lid lag β€” Graves specific)
  • Growth acceleration, advanced bone age
Why advanced bone age (but not necessarily taller adult) Excess thyroid hormone accelerates both linear growth and skeletal maturation β€” but the growth plates also close earlier. Net effect: tall as a child but often normal-to-short as adults if untreated. Treating promptly preserves growth potential.
Diagnostics

↓ TSH, ↑ free T4/T3. TRAb or TSI confirms Graves. Thyroid US or uptake scan if nodular. Bone age advanced.

Treatment
  • Methimazole = first-line in peds (avoid PTU in kids β€” hepatotoxicity risk; exception: first trimester pregnancy)
  • Beta blocker (propranolol or atenolol) for symptomatic relief of tachycardia/tremor while methimazole works
  • Radioactive iodine or thyroidectomy for definitive treatment in refractory cases or adolescents
  • Monitor CBC (methimazole can cause agranulocytosis) and LFTs

Type 1 Diabetes Mellitus

ENDOCRINE Β· AUTOIMMUNE Β· INSULIN-DEFICIENT
Pathophysiology

Autoimmune destruction of pancreatic Ξ²-cells (T-cell mediated, with autoantibodies as markers: GAD65, IA-2, insulin, ZnT8). > 90% of Ξ²-cells must be destroyed before symptoms appear. Without insulin, glucose can't enter cells β†’ cells starve β†’ the body shifts to fat breakdown for fuel β†’ ketone bodies accumulate β†’ diabetic ketoacidosis (DKA). About 30% of new-onset T1DM presents in DKA.

Clinical presentation
  • Classic triad: polyuria, polydipsia, polyphagia β€” with weight loss
  • New-onset nocturnal enuresis in a previously dry child
  • Fatigue, blurred vision
  • DKA (emergency): nausea, vomiting, abdominal pain, altered mental status, fruity breath (acetone), Kussmaul respirations (deep, rapid β€” compensatory for acidosis), tachycardia, hypotension, dehydration signs (tenting, poor cap refill, tachypnea)
Why every symptom makes sense (Case 2) Polyuria: glucose exceeds the renal threshold (~180 mg/dL) β†’ osmotic diuresis. Polydipsia: response to the fluid loss. Weight loss: cells can't use glucose β†’ fat and muscle are catabolized. Fruity breath: ketones (acetone) exhaled. Kussmaul breathing: blowing off COβ‚‚ to compensate for metabolic acidosis. Abdominal pain: ketone-induced visceral irritation. AMS: cerebral edema, hyperosmolality.
Diagnostics
  • Random glucose > 200 + symptoms, or
  • Fasting glucose > 126, or
  • HbA1c β‰₯ 6.5%, or
  • 2-hr OGTT > 200
  • UA: glucose + ketones
  • Confirmatory / typing: C-peptide (low in T1), islet cell antibodies (GAD65, IA-2)
  • CMP in DKA: metabolic acidosis, hyponatremia (often pseudo β€” glucose pulls water into vessels), normal-or-↑ K (total body K is depleted but acidosis shifts K extracellularly), elevated BUN/Cr from dehydration
  • CBC: WBC often elevated
Treatment

New-onset or DKA: ED/hospital admission. IV fluids (careful β€” too fast = cerebral edema), IV regular insulin drip, potassium replacement as acidosis corrects, gradual glucose correction.

Ongoing: basal-bolus insulin (long-acting glargine/detemir + rapid-acting aspart/lispro with meals), or insulin pump. Carb counting. CGM. Target HbA1c < 7%. Multidisciplinary diabetes team.

🎯 Board pearl β€” Case Study 2 5 y/o with N/V, weight loss, polydipsia, polyuria, new enuresis + tachycardia, hypotension, hypothermia, tachypnea, fruity breath, AMS, tenting, poor cap refill = DKA β†’ ED now. Don't waste time on oral rehydration or clinic workup.

Type 2 Diabetes Mellitus

ENDOCRINE Β· INSULIN RESISTANCE Β· RISING IN ADOLESCENTS
Pathophysiology

Peripheral tissues become resistant to insulin β€” the pancreas initially compensates with hyperinsulinemia, but Ξ²-cells eventually fatigue. Associated with obesity, family history, certain ethnicities (Hispanic, Black, AI/AN, Asian), puberty (normal physiologic insulin resistance worsens), and PCOS.

Clinical presentation
  • Obese adolescent (typically at or after puberty)
  • Asymptomatic on screening, or mild polyuria/polydipsia
  • Acanthosis nigricans (velvety hyperpigmented skin, neck/axilla) β€” marker of insulin resistance
  • Can rarely present with DKA or hyperosmolar hyperglycemic state
  • Family history of T2DM common
Screening (ADA / AAP)

BMI β‰₯ 85% + β‰₯ 1 risk factor (family hx, high-risk race/ethnicity, signs of insulin resistance, maternal DM/GDM). Start at age 10 or onset of puberty. Screen every 3 years.

Diagnostics

Same criteria as T1DM (HbA1c β‰₯ 6.5%, fasting β‰₯ 126, random β‰₯ 200 + symptoms). Differentiation: C-peptide normal or high (Ξ²-cells still working), no autoantibodies, usually obese.

Treatment
  • Lifestyle: nutrition, exercise, weight management
  • First-line pharmacotherapy: metformin (improves insulin sensitivity). If HbA1c > 8.5% or symptomatic β†’ start insulin + metformin.
  • Second-line (if metformin alone fails): GLP-1 agonists (liraglutide) approved β‰₯ 10 yrs; insulin
  • Screen for comorbidities: NAFLD, dyslipidemia, HTN, PCOS, OSA, microalbuminuria, retinopathy

Constitutional Delay of Growth & Puberty (CDGP)

ENDOCRINE Β· GROWTH Β· "LATE BLOOMER"
Pathophysiology

A variation of normal β€” the child is on a delayed tempo. Growth velocity is normal, but the child enters puberty late. Bone age is delayed (matches the delayed growth), and final adult height is usually normal.

Clinical presentation
  • Short stature relative to peers
  • Delayed pubertal onset (no breast budding by 13 in girls, no testicular enlargement by 14 in boys)
  • Family history of "late bloomers" β€” dad shaved late, mom had late menarche
  • Normal growth velocity, delayed bone age
  • Usually self-conscious about being shorter/less developed than peers
Why the bone age tells the story In CDGP, height and bone age are delayed in parallel β€” height for bone age is normal. That means the child has "catch-up time" β€” when puberty eventually fires, they'll reach a normal adult height. In pathologic short stature (e.g., GH deficiency, hypothyroidism, Turner), height is short for bone age too.
Diagnostics

Bone age X-ray (delayed), TSH/free T4, IGF-1, CBC, CMP, ESR, celiac screen, karyotype in girls (r/o Turner). Normal workup + family history + delayed bone age = CDGP.

Treatment

Reassurance is primary. For significant psychosocial distress, a short course of low-dose testosterone (boys) or estrogen (girls) can kick-start puberty without affecting final height. Not usually needed.

Precocious Puberty

ENDOCRINE Β· GROWTH Β· EARLY SEXUAL MATURATION
Definition
  • Girls: secondary sexual characteristics before age 8 (or menarche before 9.5)
  • Boys: secondary sexual characteristics before age 9
Pathophysiology β€” two types

Central (GnRH-dependent, "true"): early activation of the HPG axis β†’ all sex hormones rise together, orderly Tanner progression. In girls, ~90% is idiopathic. In boys, ~50% has a pathologic cause (CNS lesion, hamartoma) β€” always work up boys carefully.

Peripheral (GnRH-independent, "pseudo"): sex hormones from somewhere other than the HPG axis β€” adrenal (CAH, tumor), gonadal (McCune-Albright, ovarian cyst, Leydig cell tumor), exogenous (topical estrogen/testosterone exposure). Progression is often out of order.

Clinical presentation
  • Breast development (thelarche) in girls
  • Testicular enlargement (> 4 mL or 2.5 cm) in boys β€” earliest sign
  • Pubic/axillary hair, body odor, acne
  • Accelerated growth velocity and advanced bone age β†’ tall as a child, short as an adult (early growth plate closure)
  • Psychosocial distress, early menarche
Why "tall as a child, short as an adult" Sex hormones both accelerate growth and close growth plates. Early exposure = early growth spurt and early plate closure β†’ less time in the plates open state β†’ shorter adult height than predicted by genetic potential. Treatment preserves height.
Diagnostics
  • Bone age X-ray of left hand/wrist β€” first step; advanced > chronological
  • LH, FSH (baseline + GnRH-stimulation test β€” pubertal response = central)
  • Estradiol (girls), testosterone (boys)
  • TSH (primary hypothyroidism can cause incomplete precocious puberty β€” Van Wyk-Grumbach)
  • 17-OHP (r/o CAH), DHEAS (adrenal source)
  • Pelvic US (ovarian size, uterine changes)
  • Brain MRI for central precocious puberty in boys, or girls < 6
Treatment
  • Central: GnRH agonist (leuprolide) β€” continuous stimulation desensitizes the pituitary, shutting down LH/FSH pulses β†’ stops progression and preserves adult height
  • Peripheral: treat the underlying cause (tumor removal, hormone replacement for CAH, etc.)
MODULE 11 Β· CHAPTERS 39 & 40 Β· 13 QUESTIONS

Neurodevelopmental & Mental Health Conditions

5 Neurodev Β· 8 Mental Health Β· Biggest chunk of the exam

Specific Learning Disability

NEURODEVELOPMENTAL Β· ACADEMIC DISCREPANCY
Pathophysiology

A heterogeneous group of neurologically-based disorders in which a child's academic achievement is substantially below expected for age and IQ, despite adequate instruction. Involves differences in brain processing of specific academic skills β€” reading (dyslexia β€” most common), math (dyscalculia), written expression (dysgraphia).

Clinical features
  • Normal IQ
  • Persistent difficulty in one or more academic areas despite adequate instruction β‰₯ 6 months
  • Academic skills substantially and measurably below age-expected
  • Begins in school-age years, emerges when demands exceed capacities
  • Not explained by ID, vision/hearing deficit, neurologic disorder, lack of instruction, language barrier
Diagnostics

Psychoeducational evaluation β€” IQ test + achievement testing, demonstrating the discrepancy. Schools are required under IDEA to evaluate if parents request. Audiology/vision screening to rule out sensory contributors.

Treatment

IEP or 504 plan β€” individualized educational interventions, accommodations (extended time, text-to-speech, oral testing). Screen for and treat comorbid ADHD (co-occurs in 30–50%), anxiety, depression. Early intervention = best outcomes.

ADHD (Attention-Deficit/Hyperactivity Disorder)

NEURODEVELOPMENTAL Β· DSM-5 Β· MOST COMMON IN PEDS
Pathophysiology

Neurodevelopmental disorder of executive function β€” dysregulation in prefrontal cortex dopamine and norepinephrine circuits. Prefrontal cortex governs attention, working memory, impulse control, and task initiation. Highly heritable (~75%).

Three presentations: predominantly inattentive, predominantly hyperactive/impulsive, or combined.

DSM-5 criteria

β‰₯ 6 symptoms of inattention AND/OR hyperactivity/impulsivity, persisting β‰₯ 6 months, inconsistent with developmental level. Several symptoms present before age 12. Symptoms in β‰₯ 2 settings (home + school). Interfering with functioning. Not better explained by another disorder.

Adolescents/adults β‰₯ 17: only 5 symptoms needed.

Inattention symptoms

Careless mistakes, trouble sustaining attention, doesn't listen, doesn't follow through, poor organization, avoids sustained mental effort, loses things, easily distracted, forgetful.

Hyperactivity/impulsivity symptoms

Fidgeting, leaves seat, runs/climbs inappropriately, unable to play quietly, "on the go" / "driven by motor," talks excessively, blurts answers, can't wait turn, interrupts.

Diagnostics
  • Vanderbilt Rating Scales β€” from both parent and teacher (symptoms in β‰₯ 2 settings is a diagnostic requirement)
  • Other validated tools: Conners, SNAP-IV
  • Comprehensive history β€” developmental, medical, psychosocial, academic
  • Rule out mimics: sleep deprivation/OSA, hearing/vision problems, absence seizures, anxiety, depression, learning disability, lead poisoning, thyroid dysfunction, trauma
Why parents + teachers both DSM-5 requires symptoms in β‰₯ 2 settings. Classroom-only behavior could be a learning disability, a bad class fit, or a struggling teacher. Home-only behavior could be family dysfunction or anxiety. Rating scales from both reveal the pattern.
Treatment
  • Ages 4–5: behavioral therapy/parent training first. Consider methylphenidate only if behavioral fails and symptoms severe.
  • Ages β‰₯ 6: stimulant medication + behavioral therapy (the combination outperforms either alone β€” MTA study).
  • First-line stimulants: methylphenidate (Ritalin, Concerta) or amphetamines (Adderall, Vyvanse). Start low, titrate to effect.
  • Non-stimulants: atomoxetine (Strattera β€” SNRI), alpha-2 agonists (guanfacine, clonidine) β€” useful for stimulant side effects, comorbid tics, or sleep issues
  • Monitor: growth (weight, height), BP, HR, sleep, appetite, tics, mood
🎯 Board pearl (from review) "Parents and teachers are more reliable in reporting external symptoms such as those in ADHD or oppositional defiant disorder." Compare that to depression where the adolescent is the most reliable reporter β€” external behavior vs. internal state.

Autism Spectrum Disorder

NEURODEVELOPMENTAL Β· EARLY ONSET Β· SPECTRUM
Pathophysiology

A neurodevelopmental disorder with strong genetic contribution (twin concordance ~90% MZ) and altered brain connectivity β€” differences in social cognition networks, sensory processing, and executive function. Onset before age 3 (though diagnosis may come later). Not caused by vaccines, parenting, or anything the family did.

DSM-5 diagnostic criteria β€” 2 domains

Domain A β€” Persistent deficits in social communication and interaction (all 3 required):

  • Deficits in social-emotional reciprocity (back-and-forth conversation, sharing interests/emotions)
  • Deficits in nonverbal communication (eye contact, gestures, facial expressions)
  • Deficits in developing, maintaining, understanding relationships

Domain B β€” Restricted, repetitive patterns of behavior/interests (β‰₯ 2 of 4):

  • Stereotyped or repetitive motor movements (hand flapping), use of objects, or speech (echolalia)
  • Insistence on sameness, rigid routines, ritualized behavior
  • Highly restricted, fixated interests of abnormal intensity
  • Hyper- or hyporeactivity to sensory input (fascination with lights, aversion to textures/sounds)

Symptoms in early developmental period; cause functional impairment; not better explained by ID.

Red flags by age
  • 6 mo: no joyful expressions, limited eye contact
  • 9 mo: no back-and-forth sounds or facial expressions
  • 12 mo: no babbling, no pointing/gestures, no response to name
  • 16 mo: no words
  • 24 mo: no 2-word phrases
  • Any age: loss of skills (regression)
Screening
  • M-CHAT-R/F at 18 and 24 months β€” universal screening
  • Any concern at any age β†’ refer for formal evaluation and early intervention simultaneously β€” don't wait
Why "early intervention simultaneously" Diagnostic evaluation takes months. Neuroplasticity is greatest in the first 3 years. Starting Early Intervention services right away while the full evaluation is being completed gives the child weeks to months of therapy they'd otherwise miss. Don't wait for the diagnosis to act.
Treatment
  • Early intensive behavioral intervention (ABA, NDBI), speech-language therapy, OT, special education
  • No medications for core features
  • Treat comorbidities: irritability/aggression (risperidone, aripiprazole β€” only FDA-approved meds for autism, for irritability), ADHD symptoms, anxiety, sleep disturbance (melatonin), GI issues
  • Family support, parent training, respite care

Anorexia Nervosa

EATING DISORDER Β· RESTRICTION Β· HIGHEST MORTALITY OF ANY PSYCH DISORDER
Pathophysiology

Multifactorial β€” genetic, neurobiologic, psychological, sociocultural. Dysregulation of hunger/satiety circuits, reward processing, and body image perception. Restriction becomes reinforcing (both psychologically and neurobiologically β€” starvation itself alters serotonin and dopamine signaling).

DSM-5 criteria
  • Restriction of energy intake β†’ significantly low body weight
  • Intense fear of weight gain or behavior that interferes with weight gain
  • Disturbance in body image, self-evaluation unduly influenced by weight/shape, or denial of seriousness
  • Subtypes: restricting vs. binge-eating/purging
Clinical signs (consequences of starvation)
  • Vitals: bradycardia, hypotension, orthostatic changes, hypothermia
  • Skin: dry, lanugo (fine downy hair β€” body insulation response), yellowish (hypercarotenemia), acrocyanosis
  • Cardiac: prolonged QTc, arrhythmias, mitral valve prolapse, pericardial effusion
  • Endocrine: amenorrhea, low T3 (sick euthyroid), delayed puberty, osteoporosis
  • GI: delayed gastric emptying, constipation
  • Labs: ↓ WBC, ↓ platelets, low electrolytes, low albumin, transaminitis
Why lanugo and bradycardia The body enters a conservation state. Lanugo grows because subcutaneous fat is gone and core temp can't be maintained β€” fine hair provides insulation. Bradycardia (HR 40s–50s) is parasympathetic dominance + reduced metabolic demand β€” the body slows everything down to conserve energy.
Medical admission criteria (any one)
  • < 75% median BMI or rapid weight loss
  • HR < 50 daytime or < 45 nighttime
  • BP < 90/45
  • Temp < 35.6Β°C
  • Orthostatic HR increase > 20 or BP drop > 20/10
  • Electrolyte abnormalities, arrhythmia, syncope, dehydration, acute food refusal
Treatment
  • Family-Based Therapy (FBT / Maudsley) = first-line for adolescents β€” parents take charge of refeeding
  • Nutritional rehabilitation with slow caloric advancement
  • Watch for refeeding syndrome: after prolonged starvation, sudden refeeding causes insulin surge β†’ cells take up phosphate, potassium, magnesium β†’ severe hypophosphatemia, hypokalemia, hypomagnesemia β†’ cardiac arrhythmias, delirium, seizures. Prevent with slow advancement + electrolyte monitoring/supplementation.
  • SSRIs don't work for anorexia until weight is restored
  • Multidisciplinary team: pediatrics, psychiatry, dietitian, therapy

Bulimia Nervosa

EATING DISORDER Β· BINGE + COMPENSATORY
DSM-5 criteria
  • Recurrent binge eating (large amount + loss of control)
  • Recurrent compensatory behavior β€” vomiting, laxatives, diuretics, fasting, excessive exercise
  • β‰₯ 1Γ—/week for β‰₯ 3 months
  • Self-evaluation unduly influenced by weight/shape
  • Does not occur exclusively during anorexia
Clinical signs
  • Weight often normal or slightly elevated β€” doesn't exclude diagnosis
  • Russell's sign β€” calluses/scars on knuckles from self-induced vomiting
  • Parotid gland enlargement ("chipmunk cheeks") β€” from chronic purging
  • Dental erosion β€” lingual surfaces of upper teeth (perimolysis) from gastric acid
  • Esophagitis, Mallory-Weiss tears, aspiration
  • Electrolyte derangements: hypokalemia, metabolic alkalosis (from vomiting), hypochloremia
  • Menstrual irregularity but usually not amenorrhea
Why lingual dental erosion (not buccal) Vomit acid pools against the lingual (tongue-side) surfaces of the upper incisors. The buccal surfaces are bathed in saliva and spared. Lingual erosion + Russell's sign + normal weight + menstrual issues = bulimia until proven otherwise.
Treatment
  • CBT-E (Enhanced CBT for eating disorders) = first-line
  • Fluoxetine (FDA approved; only SSRI with indication) β€” works for bulimia even at normal weight
  • Nutritional counseling, regular meal structure
  • Treat electrolyte abnormalities, dental referrals

Binge Eating Disorder

EATING DISORDER Β· BINGE WITHOUT COMPENSATION
DSM-5 criteria
  • Recurrent binge eating episodes (large amount + loss of control)
  • Associated with β‰₯ 3 of: eating rapidly, eating until uncomfortably full, eating when not hungry, eating alone (embarrassment), feeling disgusted/guilty after
  • Marked distress
  • β‰₯ 1Γ—/week for β‰₯ 3 months
  • No compensatory behaviors (distinguishes from bulimia)
Clinical presentation & treatment

Often associated with obesity, depression, shame. Most common eating disorder in adults; also in adolescents. CBT = first-line. Lisdexamfetamine (Vyvanse) is FDA-approved for BED in adults (not first-line in adolescents). SSRIs may help mood symptoms.

ARFID (Avoidant/Restrictive Food Intake Disorder)

EATING DISORDER Β· NOT ABOUT BODY IMAGE
DSM-5 criteria
  • Eating disturbance β†’ significant weight loss, nutritional deficiency, dependence on supplements, or marked psychosocial impairment
  • Not about body image or weight concerns (this is the key distinction from anorexia)
  • Not explained by food scarcity or a medical/psych condition
Three common drivers
  • Sensory aversion β€” extreme picky eating (texture, smell, color)
  • Lack of interest in food β€” low appetite drive
  • Fear of consequences β€” choking, vomiting, allergic reaction (often after a traumatic episode)

Strongly associated with autism spectrum and anxiety disorders.

Treatment

CBT for ARFID, feeding therapy, OT for sensory issues, nutritional rehabilitation, treat comorbid anxiety. Very different approach than anorexia β€” weight restoration alone isn't the goal; the underlying driver must be addressed.

Separation Anxiety Disorder

ANXIETY Β· DEVELOPMENTALLY INAPPROPRIATE
Pathophysiology

Separation anxiety is normal from 8 months to ~3 years (stranger/separation anxiety develops with object permanence). It becomes a disorder when it's developmentally inappropriate (persisting past expected age or resurfacing), excessive, and functionally impairing.

DSM-5 criteria

β‰₯ 3 of the following, for β‰₯ 4 weeks in children:

  • Excessive distress when separation occurs or is anticipated
  • Persistent worry about losing attachment figures (accident, illness, death)
  • Persistent worry that an event will cause separation (getting lost, kidnapped)
  • Reluctance or refusal to go to school or elsewhere alone
  • Reluctance to sleep away from home or without attachment figure nearby
  • Nightmares about separation
  • Physical symptoms (headaches, stomachaches) when separation occurs or is anticipated
Why the stomachaches Anxiety activates the autonomic nervous system β†’ GI hypersensitivity, nausea, and true visceral distress. The child isn't "faking" a stomachache to avoid school β€” the stomachache is real, and the school-related anxiety causes it. This reframe helps with family communication.
Treatment
  • CBT with graduated exposure = first-line
  • SSRI (fluoxetine, sertraline) if moderate-severe or CBT inadequate
  • School re-entry plan; don't allow prolonged school avoidance (deepens the pattern)
  • Parent training β€” avoid reinforcing avoidance with excessive accommodation

Generalized Anxiety Disorder

ANXIETY Β· PERVASIVE WORRY
DSM-5 criteria
  • Excessive anxiety/worry about multiple events, more days than not, for β‰₯ 6 months
  • Difficult to control the worry
  • β‰₯ 1 (in children) of: restlessness, fatigue, difficulty concentrating, irritability, muscle tension, sleep disturbance (adults need 3)
  • Clinically significant distress/impairment
Clinical presentation
  • The "worrier" β€” worries about school, family, future, world events
  • Perfectionism, reassurance-seeking
  • Somatic: headaches, abdominal pain, fatigue, sleep disturbance
  • Often misattributed to "stress" for years before diagnosis
Treatment
  • CBT = first-line
  • SSRIs (fluoxetine, sertraline, escitalopram) β€” all first-line for moderate-severe
  • Combination CBT + SSRI = best outcomes (CAMS study)
  • Avoid benzodiazepines in pediatrics β€” dependence risk, disinhibition

Major Depressive Disorder

MOOD Β· CASE STUDY #3 REFERENCE
Pathophysiology

Complex interaction of genetic vulnerability, neurotransmitter dysregulation (serotonin, norepinephrine, dopamine), stress/HPA axis, and psychosocial factors. In adolescents, hormonal changes, social/academic stress, and developing identity all contribute.

DSM-5 criteria

β‰₯ 5 of the following, β‰₯ 2 weeks, with at least one being low mood OR anhedonia. Mnemonic: SIGECAPS + mood.

  • Sleep disturbance (insomnia or hypersomnia)
  • Interest loss (anhedonia)
  • Guilt or worthlessness
  • Energy loss / fatigue
  • Concentration impaired
  • Appetite/weight changes
  • Psychomotor agitation or retardation
  • Suicidal ideation
  • Mood (sad/depressed) β€” or irritability in children/adolescents

In kids/teens, irritability can substitute for depressed mood. "Failure to make expected weight gain" can substitute for weight loss.

Why irritability in adolescents (Case 3) The adolescent brain is still developing emotional regulation. Depression often manifests as irritability, anger outbursts, somatic complaints (headaches, abdominal pain), school decline, or withdrawal rather than classic "sad mood." The 14 y/o in Case 3 who "loses his temper easily and punched a hole in the wall" β€” that's depression, not conduct disorder.
Diagnostic workup
  • PHQ-A (modified PHQ-9 for adolescents) β€” 9 symptom questions + 4 follow-ups (duration, impairment, SI in past month, lifetime SI attempt)
  • Interview the adolescent privately β€” essential before anything else (Case 3 step 1)
  • Rule out medical: CBC, TSH, CMP, B12, urine tox, mono if indicated
  • Rule out substance use (marijuana, alcohol, other)
  • Rule out medications β€” steroids, clonidine, Accutane, beta blockers, OCPs, benzos, thyroid supplements
  • Rule out psychiatric comorbidities β€” ADHD, bipolar, anxiety, substance use disorder
  • Screen for suicidality β€” direct, non-judgmental
Treatment
  • Mild: psychoeducation, CBT, supportive care, regular exercise/sleep/nutrition, follow-up in 1–2 weeks
  • Moderate–severe: SSRI + CBT (TADS study showed combination superior)
  • First-line SSRIs in adolescents: fluoxetine (FDA approved β‰₯ 8 yrs) and escitalopram (β‰₯ 12 yrs). (Review said you just need to know SSRIs are first-line.)
  • Dosing rule: start low, go slow. Increase every 5–7 days to target dose; hold dose 4–6 weeks before further increases.
  • Black box warning β€” SSRIs can transiently increase suicidal ideation in youth; weekly contact for first 4 weeks, every 2 weeks for next month, monthly thereafter for the first 12 weeks.
  • Refer to psychiatry if severe, suicidal, multiple diagnoses, or not comfortable managing
⚠️ Serotonin syndrome Hyperthermia + autonomic instability + neuromuscular hyperactivity (clonus, hyperreflexia, rigidity, tremor) + mental status changes. Can progress to seizures, rhabdo, DIC, death. Triggered by SSRI + another serotonergic agent (MAOI, triptan, tramadol, linezolid, dextromethorphan, St John's wort) or SSRI overdose. Treatment: stop offending agents, supportive care, cyproheptadine if severe.

Disruptive Mood Dysregulation Disorder (DMDD)

MOOD Β· NEWER DIAGNOSIS Β· SEPARATES FROM PEDIATRIC BIPOLAR
Why it exists

DMDD was added to DSM-5 specifically to reduce overdiagnosis of pediatric bipolar disorder. Kids with chronic irritability and outbursts were being labeled bipolar (and treated with mood stabilizers), but long-term follow-up showed most develop depression or anxiety, not bipolar. DMDD captures this group correctly.

DSM-5 criteria
  • Severe recurrent temper outbursts (verbal or behavioral) grossly out of proportion to situation
  • β‰₯ 3 times per week
  • Persistently irritable/angry mood between outbursts
  • Present for β‰₯ 12 months, no symptom-free period > 3 months
  • Present in β‰₯ 2 settings
  • Diagnosis only between ages 6 and 18
  • Onset before age 10
Treatment

Limited evidence base. Psychotherapy (parent management training, CBT). Medications target comorbidities β€” SSRIs for depression/anxiety symptoms, stimulants for comorbid ADHD. Atypical antipsychotics only for severe aggression.

Oppositional Defiant Disorder & Conduct Disorder

BEHAVIORAL Β· EXTERNALIZING SPECTRUM
ODD β€” DSM-5 criteria

A pattern of angry/irritable mood, argumentative/defiant behavior, or vindictiveness lasting β‰₯ 6 months, with β‰₯ 4 symptoms from these categories:

  • Angry/irritable mood: loses temper, touchy/annoyed, angry/resentful
  • Argumentative/defiant: argues with authority, actively defies rules, deliberately annoys, blames others
  • Vindictive: spiteful at least twice in 6 months

ODD does NOT include aggression toward people/animals, destruction, theft, or serious rule violations.

Conduct Disorder β€” DSM-5 criteria

A repetitive pattern of violating rights of others or major age-appropriate norms, with β‰₯ 3 symptoms in past 12 months from these categories:

  • Aggression toward people or animals (bullying, fights, weapons, cruelty, forced sex)
  • Destruction of property (fire-setting, vandalism)
  • Deceitfulness / theft (breaking in, lying to obtain, shoplifting)
  • Serious rule violations (staying out despite parental prohibition before age 13, running away, truancy)

Childhood-onset (< 10) = worse prognosis; adolescent-onset = better. CD can progress to antisocial personality disorder in adulthood (but not always).

The ODD β†’ CD distinction matters ODD is defiance. CD is violation of others' rights. An ODD kid refuses to clean their room and argues with mom. A CD kid steals the neighbor's bike and sets fires. Treatment approach differs: ODD responds well to parent management training and family therapy; CD often requires more intensive multisystemic therapy and has worse long-term outcomes. Screen for callous-unemotional traits in CD β€” poorer response to treatment and higher risk of progression.
Treatment
  • Parent Management Training (PMT) = first-line for ODD
  • Multisystemic therapy (MST), functional family therapy for CD
  • School-based interventions, mentoring, skills training
  • Treat comorbid ADHD (high overlap β€” treating ADHD alone can resolve ODD in many cases)
  • Treat comorbid mood/anxiety disorders
  • No medication specifically for ODD or CD β€” target comorbidities
MODULE 12 Β· CHAPTER 36 Β· 5 QUESTIONS

Neurologic Conditions

Non-infectious HA Β· Seizures Β· TBI

Pediatric Headaches (Migraine & Tension)

NEURO Β· NON-INFECTIOUS Β· PRIMARY VS SECONDARY
Pathophysiology

Migraine: cortical spreading depression + activation of the trigeminovascular system β†’ release of CGRP and other neuropeptides β†’ meningeal inflammation + vasodilation + pain. Genetic (~60% have FH). Tension-type: pericranial muscle contraction, stress, poor sleep, posture; mechanism less well defined.

Migraine features (pediatric)
  • Duration in kids: 2–72 hrs (shorter than adults)
  • Location: often bilateral in kids (in adults more typically unilateral)
  • Quality: throbbing/pulsating
  • Severity: moderate-to-severe, interferes with activity
  • Associated: photo- and phonophobia (may be inferred from behavior in young kids β€” seeking dark quiet room), nausea/vomiting
  • Aura (sensory, visual, motor) in ~25% β€” precedes headache by up to 1 hr
Tension features

Bilateral, band-like, pressing/tightening, mild-moderate, not aggravated by activity, no nausea/vomiting, possibly mild photo- or phonophobia but not both.

🚨 Secondary headache red flags β€” SNOOP4 Systemic symptoms (fever, weight loss) Β· Neurologic signs (focal deficits, altered mental status, papilledema) Β· Onset sudden ("thunderclap") Β· Older (not a factor in peds but applies to adults) Β· Pattern change or Progressive Β· Positional (worse lying down β€” ↑ICP) Β· Precipitated by Valsalva Β· Papilledema Β· Parents/partners (family history concerns)
Diagnostics

Clinical diagnosis β€” headache diary. Neuroimaging (MRI preferred over CT for peds) only if red flags, abnormal neuro exam, change in pattern, or recent trauma. Routine neuroimaging for recurrent migraines with normal exam = not indicated.

Treatment
  • Acute/abortive:
    • First-line: ibuprofen 10 mg/kg or acetaminophen 15 mg/kg β€” at onset (don't wait to see if it gets bad)
    • Triptans: almotriptan β‰₯ 12 yrs, rizatriptan β‰₯ 6 yrs, sumatriptan nasal spray β‰₯ 12 yrs
    • Antiemetic (ondansetron) for nausea
  • Lifestyle triggers: regular sleep, hydration, regular meals, stress management, identify food triggers (chocolate, cheese, nitrates, MSG) β€” but individual variation
  • Prophylaxis (β‰₯ 4 headaches/mo or severely disabling): amitriptyline, topiramate, propranolol, cyproheptadine. Riboflavin/magnesium/CoQ10 nutritional supplements
  • Medication overuse headache β€” analgesics > 2–3x/week perpetuate headaches
Why "at onset" Once the migraine cascade (cortical spreading depression + trigeminovascular activation) is fully engaged, abortive meds work poorly. Taking ibuprofen within the first 30 min gives much better relief than waiting to see if the headache will pass. Teach families: "don't wait for it to get bad."

Breath-Holding Spells

NEURO Β· NON-EPILEPTIC Β· 6 MO – 6 YRS
Pathophysiology

Reflexive response, not volitional. Two types:

  • Cyanotic: triggered by anger, frustration, or crying. Child cries forcefully β†’ exhales β†’ breath-holds at end-expiration β†’ cyanosis β†’ brief LOC. More common.
  • Pallid: triggered by pain or sudden fright (minor head bump). Vagally mediated bradycardia/asystole β†’ pallor β†’ LOC. Less common.
Clinical presentation
  • Consistent trigger β†’ single cry (cyanotic) or no cry (pallid) β†’ color change β†’ LOC (lasts seconds) β†’ possible brief tonic posturing or myoclonic jerk β†’ spontaneous recovery β†’ tired but otherwise fine
  • Peak age 1–3 yrs; resolves by 4–6 yrs
  • Family history common
Why this isn't a seizure (important for parents) Breath-holding has a clear provocation (emotional or painful trigger), a predictable sequence (cry β†’ color change β†’ LOC), and immediate recovery. True seizures are usually unprovoked, with post-ictal confusion. The child is not "holding their breath on purpose" β€” it's an involuntary reflex. Reassurance is the primary intervention.
Workup

Check iron β€” iron deficiency anemia is strongly associated with breath-holding; iron supplementation reduces spells in many kids even with only borderline labs. EKG (r/o long QT if pallid/syncopal). EEG only if atypical.

Treatment
  • Reassurance β€” benign, self-limited, no long-term sequelae, outgrown by school age
  • Iron supplementation if deficient or borderline ferritin (< 50)
  • Don't reinforce with overreaction β€” the cyanotic type especially can become a behavior pattern if the child learns it produces a panicked caregiver response
  • Keep the child safe during the spell (cushion fall)

Febrile Seizures

NEURO Β· SEIZURE Β· MOST COMMON PEDS SEIZURE
Pathophysiology

Age-dependent susceptibility β€” immature brain has a lower seizure threshold when body temperature rises rapidly. It's the rate of temperature rise, not the absolute temperature, that's most important. Strong genetic component.

Definition

Seizure in a child 6 months to 5 years with fever, without CNS infection, metabolic disturbance, prior afebrile seizure, or other identifiable cause.

Classification

Simple febrile seizure (all of the following):

  • Generalized (tonic-clonic)
  • Duration < 15 minutes
  • Single event in 24 hours
  • Prior neurologically normal child

Complex febrile seizure (any of the following):

  • Focal features
  • Duration > 15 minutes
  • Recurs within 24 hours
  • Postictal neurologic abnormality (e.g., Todd's paralysis)
Evaluation
  • Simple febrile seizure with identifiable source of fever: no routine labs, no EEG, no neuroimaging. Focus on the source of fever.
  • LP considerations: any child < 12 mo with incomplete or absent Hib/pneumococcal vaccination, any concern for meningitis (irritability, bulging fontanelle, neck stiffness, petechiae), any child on antibiotics (could partially treat meningitis)
  • Complex febrile seizure: consider further workup β€” EEG, labs, imaging depending on features
Treatment
  • Most (> 95%) resolve spontaneously within 5 minutes
  • > 5 min or persistent: benzodiazepine (IV lorazepam, IM/rectal midazolam, rectal diazepam)
  • Treat the fever source
  • Antipyretics for comfort β€” but they do not prevent recurrence; don't oversell
  • Chronic anticonvulsants not indicated for simple febrile seizures
Prognosis (parental counseling)
  • ~30% recur with future febrile illness; higher if first seizure < 18 mo
  • Risk of later epilepsy after simple FS: ~1% (same as general population slightly)
  • Complex febrile seizures carry somewhat higher epilepsy risk (~4–15%)
  • No long-term neurodevelopmental effects from simple febrile seizures
Why antipyretics don't prevent recurrence Scheduled antipyretics during febrile illnesses have been shown in trials not to prevent febrile seizures. The trigger is the rate of temperature rise, which can happen before the parent is aware of the fever. Counsel families to use antipyretics for the child's comfort, not to prevent seizures β€” which avoids false reassurance.

Epilepsy

NEURO Β· RECURRENT UNPROVOKED SEIZURES
Definition (ILAE)

Any of:

  • β‰₯ 2 unprovoked seizures > 24 hrs apart, OR
  • 1 unprovoked seizure + probability of recurrence β‰₯ 60% over next 10 yrs, OR
  • Diagnosis of an epilepsy syndrome
Seizure types (ILAE 2017)
  • Focal onset β€” one brain region; may have motor or nonmotor signs; may impair awareness or not; may spread to bilateral tonic-clonic
  • Generalized onset β€” bilateral networks from the start: tonic-clonic, absence, myoclonic, atonic, tonic, clonic
  • Unknown onset
Common peds syndromes
  • Childhood absence epilepsy: 4–10 yrs, brief staring spells (5–10 sec), multiple daily, subtle eye fluttering; EEG = 3 Hz spike-and-wave; triggered by hyperventilation in clinic. Often remits by adolescence. Tx: ethosuximide first-line.
  • Benign rolandic epilepsy (BECTS): 3–13 yrs, nocturnal facial/oropharyngeal twitching, drooling, speech arrest; EEG = centrotemporal spikes; outgrown by puberty.
  • Juvenile myoclonic epilepsy (JME): adolescent onset, morning myoclonic jerks (drops toothbrush, spills cereal), GTC seizures, sometimes absence; lifelong treatment needed; levetiracetam or valproate.
  • Infantile spasms (West syndrome): sudden flexor/extensor "jackknife" spasms in clusters, 3–12 mo; EEG = hypsarrhythmia; developmental regression; urgent β€” ACTH or vigabatrin.
Workup

EEG (awake + sleep, often with hyperventilation and photic stimulation), MRI brain (preferred over CT; CT only for acute setting β€” bleed, mass). Labs if metabolic cause suspected. Genetic testing for specific syndromes.

Treatment
  • AED selection based on seizure type + syndrome
  • Monotherapy preferred; 2/3 seizure-free on first or second agent
  • Goal: seizure freedom with minimal side effects
  • Refractory (2 drugs failed) β†’ refer to epileptologist; consider ketogenic diet, VNS, epilepsy surgery
  • Counsel about driving, bathing safety, seizure action plan, medication adherence

Concussion / Mild Traumatic Brain Injury

NEURO Β· INJURY Β· FUNCTIONAL NOT STRUCTURAL
Pathophysiology

Traumatic force (direct or inertial) β†’ axonal stretch β†’ neurometabolic cascade: K⁺ efflux, glutamate release, ionic imbalance, mitochondrial dysfunction, vasoreactivity changes β†’ energy crisis. Imaging is normal β€” this is a functional disturbance, not a structural injury.

Clinical presentation

Any combination of:

  • Physical: headache, nausea/vomiting, dizziness, light/noise sensitivity, balance problems, fatigue, blurred vision
  • Cognitive: slowed thinking, difficulty concentrating, memory problems, feeling "foggy"
  • Emotional: irritability, sadness, anxiety, emotional lability
  • Sleep: sleeping more or less, trouble falling asleep

Loss of consciousness is not required for diagnosis β€” most concussions have no LOC.

When to image (PECARN rules apply)

Age < 2 years β€” CT if any of:

  • AMS or signs of AMS
  • Palpable skull fracture
  • Scalp hematoma (non-frontal, in infants)
  • LOC > 5 seconds
  • Severe mechanism (MVC, fall > 3 ft, high-impact object)
  • Not acting normally per parent

Age β‰₯ 2 years β€” CT if any of:

  • AMS or signs of AMS
  • Signs of basilar skull fracture (hemotympanum, Battle's sign, raccoon eyes, CSF otorrhea/rhinorrhea)
  • LOC
  • Vomiting
  • Severe headache
  • Severe mechanism
🚨 Red flags after head injury β€” go to ED Progressively worsening headache Β· repeated vomiting Β· seizures Β· focal weakness or numbness Β· slurred speech Β· severe drowsiness or difficulty waking Β· unequal pupils Β· confusion that worsens Β· neck pain
Treatment β€” modern approach
  • Initial relative rest 24–48 hrs (physical AND cognitive β€” limit screens, schoolwork)
  • Avoid strict prolonged rest β€” older "sit in a dark room until symptoms resolve" approach is outdated and actually prolongs recovery
  • Gradual symptom-limited return to activity after 24–48 hrs β€” light aerobic activity (walking, stationary bike) below symptom threshold actually speeds recovery
  • Return to school before return to play β€” graduated academic accommodations
  • Stepwise return-to-play protocol β€” each step takes β‰₯ 24 hrs; any symptom return β†’ step back and rest 24 hrs
  • Acetaminophen for headache (avoid NSAIDs in the first 24–48 hrs due to bleeding concerns, then OK)
Why "return to school before return to play" The brain needs to handle normal cognitive load before high-risk physical activity resumes. Returning to contact sports before cognitive symptoms have resolved risks second-impact syndrome β€” a second concussion before the first has healed can cause catastrophic cerebral edema, especially in adolescents. Return-to-play protocols exist for this reason.

END OF GUIDE

You've got this, Sasha.

Every condition here maps to the exam review transcript and Dr. [reviewer]'s emphasis. Walk through the three case studies one more time before exam day β€” the reasoning is the content.