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Observational Study
. 2023 Nov;182(11):5191-5202.
doi: 10.1007/s00431-023-05190-y. Epub 2023 Sep 14.

Linear growth of children with X-linked hypophosphatemia treated with burosumab: a real-life observational study

Yael Levy-Shraga  1   2 , Shelly Levi  3   4 , Ravit Regev  3   5 , Shoshana Gal  6   7 , Avivit Brener  3   5 , Yael Lebenthal  3   5 , David Gillis  8   9 , David Strich  9   10 , Amnon Zung  9   11 , Roxana Cleper  3   12 , Yael Borovitz  3   4 , Rachel Bello  3   13 , Ariel Tenenbaum  3   13 , Zvi Zadik  9   11 , Miriam Davidovits  3   4 , Leonid Zeitlin #  3   14 , Dov Tiosano #  6   7
Affiliations
Observational Study

Linear growth of children with X-linked hypophosphatemia treated with burosumab: a real-life observational study

Yael Levy-Shraga et al. Eur J Pediatr. 2023 Nov.

Abstract

To assess the long-term efficacy of burosumab for pediatric patients with X-linked hypophosphatemia, focusing on linear growth. This multi-center retrospective study included 35 pediatric patients who began treatment with burosumab between January 2018 and January 2021. We collected clinical data, anthropometric measurements, laboratory results, and Rickets Severity Score (RSS), from 2 years prior to treatment initiation and up to 4 years after. Burosumab was initiated at a mean age of 7.5 ± 4.4 years (range 0.6-15.9), with a mean initial dose of 0.8 ± 0.3 mg/kg, which was subsequently increased to 1.1 ± 0.4 mg/kg. The patients were followed for 2.9 ± 1.4 years (range 1-4) after initiating burosumab. Serum phosphorus levels increased from 2.7 ± 0.8 mg/dl at burosumab initiation to 3.4 ± 0.6 mg/dl after 3 months and remained stable (p < 0.001). Total reabsorption of phosphorus increased from 82.0 ± 6.8 to 90.1 ± 5.3% after 12 months of treatment (p = 0.041). The RSS improved from 1.7 ± 1.0 at burosumab initiation to 0.5 ± 0.6 and 0.3 ± 0.6 after 12 and 24 months, respectively (p < 0.001). Both height z-score and weight z-score improved from burosumab initiation to the end of the study: from - 2.07 ± 1.05 to - 1.72 ± 1.04 (p < 0.001) and from - 0.51 ± 1.12 to - 0.11 ± 1.29 (p < 0.001), respectively. Eight children received growth hormone combined with burosumab treatment. Height z-score improved among those who received growth hormone (from - 2.33 ± 1.12 to - 1.94 ± 1.24, p = 0.042) and among those who did not (from - 2.01 ± 1.01 to - 1.66 ± 1.01, p = 0.001).

Conclusion: Burosumab treatment in a real-life setting improved phosphate homeostasis and rickets severity and enhanced linear growth.

What is known: • Compared to conventional therapy, burosumab treatment has been shown to increase serum phosphate levels and reduce the severity of rickets. • The effect of burosumab on growth is still being study.

What is new: • Height z-score improved between the start of burosumab treatment and the end of the study (-2.07 ± 1.05 vs. -1.72 ± 1.04, p < 0.001). • Eight children received burosumab combined with growth hormone treatment without side effects during the concomitant treatments.

Keywords: Burosumab; Children; Growth; Growth hormone; X-linked hypophosphatemia.

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References

    1. Baroncelli GI, Mora S (2021) X-linked hypophosphatemic rickets: multisystemic disorder in children requiring multidisciplinary management. Front Endocrinol (Lausanne) 12:688309. https://doi.org/10.3389/fendo.2021.688309
    1. Haffner D, Emma F, Eastwood DM, Duplan MB, Bacchetta J, Schnabel D et al (2019) Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia. Nat Rev Nephrol 15(7):435–455. https://doi.org/10.1038/s41581-019-0152-5 - DOI - PubMed - PMC
    1. Kuro OM, Moe OW (2017) FGF23-alphaKlotho as a paradigm for a kidney-bone network. Bone 100:4–18. https://doi.org/10.1016/j.bone.2016.11.013 - DOI
    1. Imel EA, White KE (2019) Pharmacological management of X-linked hypophosphataemia. Br J Clin Pharmacol 85(6):1188–1198. https://doi.org/10.1111/bcp.13763 - DOI - PubMed
    1. Zivicnjak M, Schnabel D, Billing H, Staude H, Filler G, Querfeld U et al (2011) Age-related stature and linear body segments in children with X-linked hypophosphatemic rickets. Pediatr Nephrol 26(2):223–231. https://doi.org/10.1007/s00467-010-1705-9 - DOI - PubMed

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