| Title: |
Effects of Burosumab Treatment on Mineral Metabolism in Children and Adolescents With X-linked Hypophosphatemia. |
| Authors: |
Ewert, Annika, Rehberg, Mirko, Schlingmann, Karl Peter, Hiort, Olaf, John-Kroegel, Ulrike, Metzing, Oliver, Wühl, Elke, Schaefer, Franz, Kemper, Markus J., Derichs, Ute, Richter-Unruh, Annette, Patzer, Ludwig, Albers, Norbert, Dunstheimer, Desiree, Haberland, Holger, Heger, Sabine, Schröder, Carmen, Jorch, Norbert, Schmid, Elmar, Staude, Hagen |
| Source: |
Journal of Clinical Endocrinology & Metabolism; Oct2023, Vol. 108 Issue 10, pe998-e1006, 9p |
| Subject Terms: |
MINERAL metabolism, HYPOPHOSPHATEMIA, GLOMERULAR filtration rate |
| Abstract: |
Context: Burosumab has been approved for the treatment of children and adults with X-linked hypophosphatemia (XLH). Real-world data and evidence for its efficacy in adolescents are lacking. Objective: To assess the effects of 12 months of burosumab treatment on mineral metabolism in children (aged <12 years) and adolescents (aged 12-18 years) with XLH. Design: Prospective national registry. Setting: Hospital clinics. Patients: A total of 93 patients with XLH (65 children, 28 adolescents). Main Outcome Measures: Z scores for serum phosphate, alkaline phosphatase (ALP), and renal tubular reabsorption of phosphate per glomerular filtration rate (TmP/GFR) at 12 months. Results: At baseline, patients showed hypophosphatemia (−4.4 SD), reduced TmP/GFR (−6.5 SD), and elevated ALP (2.7 SD, each P < .001 vs healthy children) irrespective of age, suggesting active rickets despite prior therapy with oral phosphate and active vitamin D in 88% of patients. Burosumab treatment resulted in comparable increases in serum phosphate and TmP/GFR in children and adolescents with XLH and a steady decline in serum ALP (each P < .001 vs baseline). At 12 months, serum phosphate, TmP/GFR, and ALP levels were within the age-related normal range in approximately 42%, 27%, and 80% of patients in both groups, respectively, with a lower, weight-based final burosumab dose in adolescents compared with children (0.72 vs 1.06 mg/kg, P < .01). Conclusions: In this real-world setting, 12 months of burosumab treatment was equally effective in normalizing serum ALP in adolescents and children, despite persistent mild hypophosphatemia in one-half of patients, suggesting that complete normalization of serum phosphate is not mandatory for substantial improvement of rickets in these patients. Adolescents appear to require lower weight-based burosumab dosage than children. [ABSTRACT FROM AUTHOR] |
|
Copyright of Journal of Clinical Endocrinology & Metabolism is the property of Oxford University Press / USA and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.) |
| Database: |
Complementary Index |
