The Journal of clinical endocrinology and metabolism · 2026
Real-world impact of treatment on growth in children with X-linked hypophosphatemia.
СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод в очереди
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Fukumoto Seiji, Haffner Dieter, Imel Erik A, Ozono Keiichi, Brandi Maria Luisa, Ishii Haruka, Li Zhiyi, Sandilands Kerry, Joos-Vandewalle Paul, Lee Cheuk, Kanematsu Masanori, McCullough Keith P, Carpenter Thomas O
Аннотация
CONTEXT: X-linked hypophosphatemia (XLH) is a rare, genetic, progressive, lifelong disorder manifest by impaired growth and disproportionate short stature. Burosumab, a monoclonal antibody against fibroblast growth factor 23, is approved for treating patients with XLH.
OBJECTIVE: To understand the impact of burosumab treatment on growth in a real-world setting.
DESIGN: Interim data from 3 ongoing, real-world observational studies (NCT03651505, NCT03193476, and NCT03745521), were unified into a single study (APEX).
SETTING: Outpatient clinics.
PATIENTS: Children aged 2-17 years with XLH.
INTERVENTION(S): Subcutaneous burosumab vs oral phosphate salts and active vitamin D or no treatment.
MAIN OUTCOME MEASURE(S): Retrospective and prospective height data from children enrolled in APEX were analyzed to provide long-term estimation of the impact of treatment on growth. Growth and height were estimated by a mixed regression model with separate models for children and adolescents.
RESULTS: In total, 641 participants were analyzed (402 female) with 498 burosumab-treated and 143 burosumab naïve. Median (interquartile range [IQR]) enrollment ages were 8 (5-12) years for burosumab-treated and 10 (4-14) years for burosumab-naïve participants. Burosumab-treated participants experienced improved growth over burosumab-naïve participants (additional growth velocity: 0.085 Z-score/year for children [P < .0001]; 0.121 Z-score/year for adolescents [P < .0001]). Modeling predicted greater adult height among burosumab-treated participants.
CONCLUSION: Burosumab had a robust, positive association with improved growth outcomes in male and female children and adolescents with XLH. Modeling based on a median of 3.3 years of follow-up predicted burosumab can support improvement of growth that will likely result in greater adult height.
OBJECTIVE: To understand the impact of burosumab treatment on growth in a real-world setting.
DESIGN: Interim data from 3 ongoing, real-world observational studies (NCT03651505, NCT03193476, and NCT03745521), were unified into a single study (APEX).
SETTING: Outpatient clinics.
PATIENTS: Children aged 2-17 years with XLH.
INTERVENTION(S): Subcutaneous burosumab vs oral phosphate salts and active vitamin D or no treatment.
MAIN OUTCOME MEASURE(S): Retrospective and prospective height data from children enrolled in APEX were analyzed to provide long-term estimation of the impact of treatment on growth. Growth and height were estimated by a mixed regression model with separate models for children and adolescents.
RESULTS: In total, 641 participants were analyzed (402 female) with 498 burosumab-treated and 143 burosumab naïve. Median (interquartile range [IQR]) enrollment ages were 8 (5-12) years for burosumab-treated and 10 (4-14) years for burosumab-naïve participants. Burosumab-treated participants experienced improved growth over burosumab-naïve participants (additional growth velocity: 0.085 Z-score/year for children [P < .0001]; 0.121 Z-score/year for adolescents [P < .0001]). Modeling predicted greater adult height among burosumab-treated participants.
CONCLUSION: Burosumab had a robust, positive association with improved growth outcomes in male and female children and adolescents with XLH. Modeling based on a median of 3.3 years of follow-up predicted burosumab can support improvement of growth that will likely result in greater adult height.