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

  • Meyerhoff, Nadine1
  • Haffner, Dieter1
  • Staude, Hagen2
  • Wühl, Elke3
  • Marx, Michaela4
  • Beetz, Rolf5
  • Querfeld, Uwe6
  • Holder, Martin7
  • Billing, Heiko8
  • Rabl, Wolfgang9
  • Schröder, Carmen10
  • Hiort, Olaf11
  • Brämswig, Jürgen H.12
  • Richter-Unruh, Annette12
  • Schnabel, Dirk13
  • Živičnjak, Miroslav1
  • 1 Hannover Medical School, Department of Pediatric Kidney, Liver and Metabolic Diseases, Carl-Neuberg-Strasse 1, Hannover, 30625, Germany , Hannover (Germany)
  • 2 University Children’s Hospital Rostock, Rostock, Germany , Rostock (Germany)
  • 3 University Children’s Hospital Heidelberg, Heidelberg, Germany , Heidelberg (Germany)
  • 4 University Children’s Hospital Erlangen, Erlangen, Germany , Erlangen (Germany)
  • 5 University Children’s Hospital Mainz, Mainz, Germany , Mainz (Germany)
  • 6 University Children’s Hospital, Charité, Department of Pediatric Nephrology, Berlin, Germany , Berlin (Germany)
  • 7 Klinikum Stuttgart, Olgahospital, Department of Pediatrics, Stuttgart, Germany , Stuttgart (Germany)
  • 8 University Children’s Hospital Tübingen, Tübingen, Germany , Tübingen (Germany)
  • 9 Children’s Hospital of the Technical University Munich, Munich, Germany , Munich (Germany)
  • 10 University Children’s Hospital Greifswald, Greifswald, Germany , Greifswald (Germany)
  • 11 University of Lübeck, Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics and Adolescent Medicine, Lübeck, Germany , Lübeck (Germany)
  • 12 University Children’s Hospital Münster, Münster, Germany , Münster (Germany)
  • 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 , Berlin (Germany)
Published Article
Pediatric Nephrology
Publication Date
Oct 20, 2017
DOI: 10.1007/s00467-017-3820-3
Springer Nature


BackgroundWe 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.MethodsWe 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.ResultsAt 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.ConclusionsGrowth 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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