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. 2022 Dec 21;17(1):444.
doi: 10.1186/s13023-022-02590-5.

Growth hormone treatment improves final height in children with X-linked hypophosphatemia

Julia André #  1 , Volha V Zhukouskaya #  2   3 , Anne-Sophie Lambert  1   4 , Jean-Pierre Salles  5 , Brigitte Mignot  6 , Claire Bardet  7 , Catherine Chaussain  1   7   8 , Anya Rothenbuhler  1 , Agnès Linglart  1   9
Affiliations

Growth hormone treatment improves final height in children with X-linked hypophosphatemia

Julia André et al. Orphanet J Rare Dis. .

Abstract

Background/aim: Despite optimal conventional treatment (oral phosphate supplements and active vitamin D analogs), about 40-50% of children with well-controlled X-linked hypophosphatemia (XLH) show linear growth failure, making them less likely to achieve an acceptable final height. Here, we studied the hypothesis that rhGH treatment improves final height in children with XLH and growth failure.

Methods: Two cohorts of children with XLH were included in this retrospective longitudinal analysis: (1) a cohort treated with rhGH for short stature (n = 34) and (2) a cohort not treated with rhGH (n = 29). The mean duration of rhGH treatment was 4.4 ± 2.9 years. We collected the auxological parameters at various time points during follow-up until final height.

Results: In rhGH-treated children, 2 years of rhGH therapy was associated with a significant increase in height from - 2.4 ± 0.9 to - 1.5 ± 0.7 SDS (p < 0.001). Their mean height at rhGH discontinuation was - 1.2 ± 0.9 SDS and at final height was - 1.3 ± 0.9 SDS corresponding to 165.5 ± 6.4 cm in boys and 155.5 ± 6.3 cm in girls. Notably, the two groups had similar final heights; i.e., the final height in children not treated with rhGH being - 1.2 ± 1.1 SDS (165.4 ± 6.8 cm in boys and 153.7 ± 7.8 cm in girls), p = 0.7.

Conclusion: Treatment with rhGH permits to improve final height in children with XLH and growth failure, despite optimal conventional treatment. We propose therefore that rhGH therapy could be considered as an option for short stature in the context of XLH.

Keywords: Fibroblast growth factor 23; Final height; Recombinant human growth hormone; Rickets; X-linked hypophosphatemia.

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Conflict of interest statement

JA nothing to disclose; VVZ reports the speaker’s fees and the travel grants from Kyowa Kirin, Novonordisk, unrelated to this research; ASL nothing to disclose; JPS nothing to disclose; BM nothing to disclose; CB nothing to disclose; CC nothing to disclose; AR reports the speaker’s fees and the travel grants from Kyowa Kirin; AL reports e research grant from Kyowa Kirin, honoraria and consulting fees from Pfizer, Novonordisk, Merck Serono and Sandoz, unrelated to this work.

Figures

Fig. 1
Fig. 1
Height SDS in children with X-linked hypophosphatemia from diagnosis to adult height. Changes in height of children with XLH who received rhGH are plotted with the red dotted line. Children treated with rhGH were diagnosed with XLH at a mean age of 3.4 years. The rhGH treatment was started at average of 9.8 years and discontinued at average of 14.2 years. The age at last visit was ~ 19.2 years. Changes in height of children with XLH who did not receive rhGH are plotted with the blue line. The children in this cohort were diagnosed with XLH at a mean age of 2.6 years. The age at last visit was at average of 16.9 years. *p < 0.001. XLH: X-linked hypophosphatemia; SDS: standard deviation score or Z-score; rhGH: recombinant human growth hormone
See this image and copyright information in PMC

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