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

Autor: André J; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France., Zhukouskaya VV; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France. volha.zhukouskaya@aphp.fr.; Laboratory Orofacial Pathologies, Imaging and Biotherapies URP2496 and FHU-DDS-Net, Dental School, and Plateforme d'Imagerie du Vivant (PIV), Université Paris Cité, Montrouge, France. volha.zhukouskaya@aphp.fr., Lambert AS; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France.; AP-HP, Medicine for Adolescents, Bicêtre Paris Saclay Hospital, Le Kremlin Bicêtre, France., Salles JP; Unit of Endocrinology and Bone Diseases, Children Hospital, Toulouse University Hospital, CHU de Toulouse, Université de Toulouse, ERN BOND, INSERM UMR 1291/CNRS 5051, INFINITY Center, Toulouse, France., Mignot B; Department of Pediatrics, CHU of Besancon, Besançon, France., Bardet C; Laboratory Orofacial Pathologies, Imaging and Biotherapies URP2496 and FHU-DDS-Net, Dental School, and Plateforme d'Imagerie du Vivant (PIV), Université Paris Cité, Montrouge, France., Chaussain C; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France.; Laboratory Orofacial Pathologies, Imaging and Biotherapies URP2496 and FHU-DDS-Net, Dental School, and Plateforme d'Imagerie du Vivant (PIV), Université Paris Cité, Montrouge, France.; AP-HP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Dental Medicine Department, Bretonneau Hospital, GHN-Universite de Paris, Paris, France., Rothenbuhler A; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France., Linglart A; AP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, DMU SEA, OSCAR Filière, EndoRare and BOND ERN, Bicêtre Paris Saclay Hospital, 78 Rue du Général Leclerc, 94270, Le Kremlin Bicêtre, France.; INSERM, Physiologie Et Physiopathologie Endocrinienne, Bicêtre Paris Saclay Hospital, Paris Saclay University, Le Kremlin Bicêtre, France.
Jazyk: angličtina
Zdroj: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 Dec 21; Vol. 17 (1), pp. 444. Date of Electronic Publication: 2022 Dec 21.
DOI: 10.1186/s13023-022-02590-5
Abstrakt: 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.
(© 2022. The Author(s).)
Databáze: MEDLINE
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