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. 2023 Sep 18;108(10):e998-e1006.
doi: 10.1210/clinem/dgad223.

Effects of Burosumab Treatment on Mineral Metabolism in Children and Adolescents With X-linked Hypophosphatemia

Annika Ewert  1 , Mirko Rehberg  2 , Karl Peter Schlingmann  3 , Olaf Hiort  4 , Ulrike John-Kroegel  5 , Oliver Metzing  5 , Elke Wühl  6 , Franz Schaefer  6 , Markus J Kemper  7 , Ute Derichs  8 , Annette Richter-Unruh  9 , Ludwig Patzer  10 , Norbert Albers  11 , Desiree Dunstheimer  12 , Holger Haberland  13 , Sabine Heger  14 , Carmen Schröder  15 , Norbert Jorch  16 , Elmar Schmid  17 , Hagen Staude  18 , Marcus Weitz  19 , Clemens Freiberg  20 , Maren Leifheit-Nestler  1 , Miroslav Zivicnjak  1 , Dirk Schnabel  21 , Dieter Haffner  1
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Effects of Burosumab Treatment on Mineral Metabolism in Children and Adolescents With X-linked Hypophosphatemia

Annika Ewert et al. J Clin Endocrinol Metab. .

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.

Keywords: TmP/GFR; X-linked hypophosphatemia; adolescents; alkaline phosphatase; burosumab; children; hyperparathyroidism; phosphate; rickets.

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