title: Age at disease onset predicts likelihood and rapidity of growth failure among infants and young children with spinal muscular atrophy types 1 and 2.
abstract: Growth failure is nearly universal in spinal muscular atrophy type 1 and common in type 2, although acuity is often underappreciated at initial diagnosis. We reviewed 44 consecutive spinal muscular atrophy patients (28 type 1, 16 type 2) under 3 years at initial presentation. Growth failure was conventionally defined: weight below the fifth percentile or dropping 2 major percentiles over 6 months. Growth failure differed among subjects stratified by age at disease onset using the Kaplan-Meier method (P = 0.011). Median time to growth failure among subjects with onset between 0 to 3 months of age was 5 months; Only 1 of 22 avoided failure by 22 months of age. Median time to failure with disease onset between 4 to 6 months was 15 months. Most late onset (> 6 months) subjects avoided growth failure. Early clinical symptoms predict feeding dysfunction and growth failure. Immediate, proactive nutritional intervention is indicated for patients with early symptom onset.
authors.affiliation: Division of Pediatric Neurosciences, Department of Neurology, SMA Clinical Research Center, Columbia University Medical Center, New York, NY 10032-3791, USA.
authors.forename: Douglas M
authors.initials: DM
authors.lastname: Sproule
authors.forename: Ridda
authors.initials: R
authors.lastname: Hasnain
authors.forename: Dorcas
authors.initials: D
authors.lastname: Koenigsberger
authors.forename: Megan
authors.initials: M
authors.lastname: Montgomery
authors.forename: Darryl C
authors.initials: DC
authors.lastname: De Vivo
authors.forename: Petra
authors.initials: P
authors.lastname: Kaufmann
mesh_terms: D017668:Age of Onset; D000886:Anthropometry; D002675:Child, Preschool; D015331:Cohort Studies; D002658:Developmental Disabilities / Q000175:diagnosis* / Q000209:etiology* / Q000401:mortality; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D053208:Kaplan-Meier Estimate; D016013:Likelihood Functions; D008297:Male; D011237:Predictive Value of Tests; D012189:Retrospective Studies; D014897:Spinal Muscular Atrophies of Childhood / Q000150:complications*