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Real-world impact of treatment on growth in children with X-linked hypophosphatemia
Seiji Fukumoto1, Dieter Haffner2, Erik A Imel3
1Department of Medicine, Tamaki-Aozora Hospital, Tokushima 779-3125, Japan.
Insights
Burosumab treatment significantly improved growth velocity in children and adolescents with X-linked hypophosphatemia (XLH). This real-world study predicts burosumab therapy leads to greater adult height for XLH patients.
Area of Science:
- Pediatric Endocrinology
- Genetic Disorders
- Pharmacological Treatments
Background:
- X-linked hypophosphatemia (XLH) is a rare genetic disorder causing impaired growth and short stature.
- Burosumab, a fibroblast growth factor 23 inhibitor, is an approved treatment for XLH.
Purpose of the Study:
- To evaluate the real-world impact of burosumab on growth in pediatric XLH patients.
- To compare growth outcomes between burosumab-treated and burosumab-naïve XLH individuals.
Main Methods:
- Interim data from three observational studies (APEX) were analyzed.
- Included 641 children (aged 2-17) with XLH, comparing burosumab treatment to other therapies or no treatment.
- Mixed regression models estimated growth and height changes over time.
Main Results:
- Burosumab-treated patients showed significantly improved growth velocity compared to burosumab-naïve patients (children: +0.085 Z-score/year; adolescents: +0.121 Z-score/year).
- Statistical modeling predicted greater final adult height in the burosumab-treated group.
- The positive association between burosumab and growth was observed in both males and females.
Conclusions:
- Burosumab demonstrates a significant positive association with improved growth outcomes in pediatric XLH.
- Real-world data suggest burosumab treatment supports growth, likely resulting in increased adult height.
- Long-term follow-up modeling indicates sustained benefits of burosumab for XLH growth.
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.
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