The Journal of clinical endocrinology and metabolism · 2020
Growth Curves for Children with X-linked Hypophosphatemia.
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Mao Meng, Carpenter Thomas O, Whyte Michael P, Skrinar Alison, Chen Chao-Yin, San Martin Javier, Rogol Alan D
Аннотация
CONTEXT: We characterized linear growth in infants and children with X-linked hypophosphatemia (XLH).
OBJECTIVE: Provide linear growth curves for children with XLH from birth to early adolescence.
DESIGN: Data from 4 prior studies of XLH were pooled to construct growth curves. UX023-CL002 was an observational, retrospective chart review. Pretreatment data were collected from 3 interventional trials: two phase 2 trials (UX023-CL201, UX023-CL205) and a phase 3 trial (UX023-CL301).
SETTING: Medical centers with expertise in treating XLH.
PATIENTS: Children with XLH, 1-14 years of age.
INTERVENTION: None.
MAIN OUTCOME MEASURE: Height-for-age linear growth curves, including values for the 5th, 10th, 25th, 50th, 75th, 90th, and 95th percentiles for children with XLH compared to population norms.
RESULTS: A total of 228 patients (132 girls, 96 boys) with 2381 height measurements were included. Nearly all subjects (> 99%) reported prior management with supplementation therapy. Compared to the Center for Disease Control and Prevention growth curves, boys at age 3 months, 6 months, 9 months, 1 year, and 2 years had median height percentiles of 46%, 37%, 26%, 18%, and 5%, respectively; for girls the median height percentiles were 52%, 37%, 25%, 18%, and 7%, respectively. Annual growth in children with XLH fell below that of healthy children near 1 year of age and progressively declined during early childhood, with all median height percentiles < 8% between 2 and 12 years old.
CONCLUSION: Children with XLH show decreased height gain by 1 year of age and remain below population norms thereafter. These data will help evaluate therapeutic interventions on linear growth for pediatric XLH.
OBJECTIVE: Provide linear growth curves for children with XLH from birth to early adolescence.
DESIGN: Data from 4 prior studies of XLH were pooled to construct growth curves. UX023-CL002 was an observational, retrospective chart review. Pretreatment data were collected from 3 interventional trials: two phase 2 trials (UX023-CL201, UX023-CL205) and a phase 3 trial (UX023-CL301).
SETTING: Medical centers with expertise in treating XLH.
PATIENTS: Children with XLH, 1-14 years of age.
INTERVENTION: None.
MAIN OUTCOME MEASURE: Height-for-age linear growth curves, including values for the 5th, 10th, 25th, 50th, 75th, 90th, and 95th percentiles for children with XLH compared to population norms.
RESULTS: A total of 228 patients (132 girls, 96 boys) with 2381 height measurements were included. Nearly all subjects (> 99%) reported prior management with supplementation therapy. Compared to the Center for Disease Control and Prevention growth curves, boys at age 3 months, 6 months, 9 months, 1 year, and 2 years had median height percentiles of 46%, 37%, 26%, 18%, and 5%, respectively; for girls the median height percentiles were 52%, 37%, 25%, 18%, and 7%, respectively. Annual growth in children with XLH fell below that of healthy children near 1 year of age and progressively declined during early childhood, with all median height percentiles < 8% between 2 and 12 years old.
CONCLUSION: Children with XLH show decreased height gain by 1 year of age and remain below population norms thereafter. These data will help evaluate therapeutic interventions on linear growth for pediatric XLH.
Предметные рубрики MeSH
AdolescentBody HeightChildChild DevelopmentChild, PreschoolClinical Trials, Phase II as TopicClinical Trials, Phase III as TopicFamilial Hypophosphatemic RicketsFemaleFibroblast Growth Factor-23Genetic Diseases, X-LinkedGrowth ChartsHumansInfantMalePhosphatesRetrospective StudiesTreatment OutcomeVitamin D