ESPE Abstracts (2019) 92 RFC2.2

Does the Treatment with Recombinant Human Growth Hormone Improve Final Height in Patients Affected by X-Linked Hypophosphatemia?

Julia André1, Volha V. Zhukouskaya1,2, Anya Rothenbuhler1, Anne-Sophie Lambert1, Jean-Pierre Salles3, Brigitte Mignot4, Agnès Linglart1


1APHP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Filiere OSCAR and Platform of expertise for rare diseases Paris-Sud, Bicêtre Paris-Sud Hospital, Le Kremlin Bicêtre, France. 2Department of Clinical Medicine and Surgery, Division of Endocrinology, University of Naples Federico II, Naples, Italy. 3CHU Purpan, Department of Endocrinology, Toulouse, France. 4CHU of Besancon, Department of Pediatrics, Besançon, France


Background/Aim: 25 to 40% of children with well-controlled X-linked hypophosphatemia (XLH) show linear growth failure, despite optimal conventional treatment (oral phosphate supplementation and active forms of vitamin D) with final height -2 SDS. Recombinant human growth hormone (rhGH) may be an adjuvant treatment of the growth retardation in these patients. Therefore, the main objective of this study was to describe how rhGH treatment improves final height in children with XLH.

Materials/Methods: In our retrospective observational study we included children with XLH who received rhGH for at least one year and reached their final height. Final height was defined as age >16 years, or bone age > 16 years, or growth velocity <2 cm/year. We collected weight, height, and body mass index (BMI) at birth⟶at XLH diagnosis⟶at start of rhGH treatment⟶2 two years after rhGH treatment⟶at the end of rhGH treatment⟶at the last visit.

Results: 34 patients (13 male / 21 female) were included. Mean age at start of rhGH treatment was 9.8±3.5 years. Duration of rhGH treatment and follow up were 3.4±2.9 and 4.5±3.1 years, respectively. The last visit was performed at 19.2±3.4 years. Mean doses of rhGH at initiation and the end of treatment were 77.4±14.5 and 66.8±20.5 µg/kg/day, respectively. The average height of patients significantly increased from -2.4±0.9 SDS at initiation, to -1.5±0.7 SDS (P<0.001) after 2 years of rhGH treatment, to -1.2±0.9 SDS (P=0.67) at rhGH discontinuation and to -1.3±0.9 SDS at final height. The global height increment of rhGH was 1.2±0.7 SDS.

Conclusion: Treatment with rhGH significantly increases final height SDS in comparison to pre-treatment height. Most height gain is obtained during the first 2 years of treatment and is sustained through final height despite treatment interruption.

Diagnosis of XLH Before rhGH2 years after rhGHEnd rhGHLast visit
Age
(Years)
3.4±3.49.8±3.511.9±3.414.2±3.119.2±3.4
Height ((SDS)-2.2±1.2-2.4±0.9-1.5±0.7-1.2±0.9-1.3±0.9
P0.887
0.001
0.670
0.996

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