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Effects of growth hormone treatment on adult height in severely short children with X-linked hypophosphatemic rickets

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Abstract

Background

We recently showed that a 3-year growth hormone (GH) treatment improves linear growth in severely short children with X-linked hypophosphatemic rickets (XLH). It is unknown if GH therapy increases adult height in XLH patients.

Methods

We carried out a follow-up analysis of a randomized controlled open-label GH study in short prepubertal children with XLH on phosphate and active vitamin D treatment. The changes in SD scores (SDS) of height, sitting height, leg and arm length, and sitting height index (i.e., the ratio between sitting height and height) were analyzed in 11 out of 16 patients followed-up until adult height.

Results

At baseline, XLH patients showed disproportionately short stature with reduced standardized height (−3.2 ± 0.6), sitting height (−1.7 ± 0.6), leg (−3.7 ± 0.7) and arm (−2.5 ± 0.8) length, and markedly elevated sitting height index (3.3 ± 0.6; each p < 0.01 versus healthy children). In GH-treated patients, adult height, sitting height, leg length, and arm length exceeded baseline values by 0.7 SDS, 1.7 SDS, 0.7 SDS, and 1.2 SDS respectively, although this was only significant for sitting height. In controls, no significant changes in linear body dimensions were noted. Adult height did not statistically differ between groups (−2.4 ± 0.7 vs −3.3 ± 1.2, p = 0.082). GH did not exaggerate body disproportion.

Conclusions

Growth hormone treatment did not significantly increase adult height in this group of short children with XLH, which may be at least partly due to the small number of patients included in our study.

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Acknowledgements

We very much appreciate the willingness of our patients and their families to participate in this trial. We thank all the members of the Hypophosphatemic Rickets Study Group of the “Deutsche Gesellschaft für Kinderendokrinologie und -diabetologie” (German Society for Pediatric Endocrinology and Diabetology) and the “Gesellschaft für Pädiatrische Nephrologie” (German Society for Pediatric Nephrology) for their help in the design and realization of this project. We especially thank E. Schmid (Kinderpraxen Hirschaid, Germany) and D. Wölfel (Children’s Hospital Göppingen, Germany, who recruited patients into this study. This project was supported by a research grant from Pfizer Pharma GmbH, who also provided Genotropin™ for treatment.

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Authors and Affiliations

  1. Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625, Hannover, Germany

    Nadine Meyerhoff, Dieter Haffner & Miroslav Živičnjak

  2. University Children’s Hospital Rostock, Rostock, Germany

    Hagen Staude

  3. University Children’s Hospital Heidelberg, Heidelberg, Germany

    Elke Wühl

  4. University Children’s Hospital Erlangen, Erlangen, Germany

    Michaela Marx

  5. University Children’s Hospital Mainz, Mainz, Germany

    Rolf Beetz

  6. Department of Pediatric Nephrology, University Children’s Hospital, Charité, Berlin, Germany

    Uwe Querfeld

  7. Department of Pediatrics, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany

    Martin Holder

  8. University Children’s Hospital Tübingen, Tübingen, Germany

    Heiko Billing

  9. Children’s Hospital of the Technical University Munich, Munich, Germany

    Wolfgang Rabl

  10. University Children’s Hospital Greifswald, Greifswald, Germany

    Carmen Schröder

  11. Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics and Adolescent Medicine, University of Lübeck, Lübeck, Germany

    Olaf Hiort

  12. University Children’s Hospital Münster, Münster, Germany

    Jürgen H. Brämswig & Annette Richter-Unruh

  13. Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Center for Chronically Sick Children, Pediatric Endocrinology, Berlin, Germany

    Dirk Schnabel

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  1. Nadine Meyerhoff
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  8. Martin Holder
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  16. Miroslav Živičnjak
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on Behalf of the Hypophosphatemic Rickets Study Group of the “Deutsche Gesellschaft für Kinderendokrinologie und -diabetologie” and “Gesellschaft für Pädiatrische Nephrologie”

Corresponding author

Correspondence to Miroslav Živičnjak.

Ethics declarations

The study received full approval from the Ethics Committee of the Charité Hospital, Berlin and each participating center. Assent and written informed consent for participation was obtained from all patients and/or their parents.

Conflicts of interest

DH has served as a consultant for Sandoz and has received honoraria and/or research support from Amgen Inc., Horizon Pharma, Kyowa Kirin, Merck Serono, Pfizer, and Sandoz. DS has served as a consultant for Sandoz and Novo Nordisk and has received honoraria from Kyowa Kirin, Alexion, Merck Serono, Pfizer, and Ipsen. OH has served as a consultant for Novo Nordisk and has received honoraria from Sandoz, Ipsen, Merck Serono, and Ultragenyx. MŽ, NM, HS, EW, MM, RB, UQ, MH, HB, WR, CS, JB, and AR declare that they have no conflicts of interest to declare.

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Meyerhoff, N., Haffner, D., Staude, H. et al. Effects of growth hormone treatment on adult height in severely short children with X-linked hypophosphatemic rickets. Pediatr Nephrol 33, 447–456 (2018). https://doi.org/10.1007/s00467-017-3820-3

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  • DOI: https://doi.org/10.1007/s00467-017-3820-3

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