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Supplemental material for: Growth curves for children with X-linked hypophosphatemia"

Citation

Mao, Meng et al. (2020), Supplemental material for: Growth curves for children with X-linked hypophosphatemia", Dryad, Dataset, https://doi.org/10.5061/dryad.sqv9s4n1w

Abstract

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 four prior studies of XLH were pooled to construct growth curves. UX023-CL002 was an observational, retrospective chart review. Pre-treatment data were collected from three 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: 228 patients (132 girls, 96 boys) with 2,381 height measurements were included. Nearly all subjects (>99%) reported prior management with supplementation therapy. Compared to the CDC growth curves, boys at age 0.25, 0.50, 0.75, 1.0, and 2.0 years-old 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.

Funding

Ultragenyx Pharmaceutical Inc in partnership with Kyowa Hakko Kirin Co., Ltd.

Ultragenyx Pharmaceutical Inc in partnership with Kyowa Hakko Kirin Co., Ltd.