Nadine Meyerhoff1, Dieter Haffner1, Hagen Staude2, Elke Wühl3, Michaela Marx4, Rolf Beetz5, Uwe Querfeld6, Martin Holder7, Heiko Billing8, Wolfgang Rabl9, Carmen Schröder10, Olaf Hiort11, Jürgen H Brämswig12, Annette Richter-Unruh12, Dirk Schnabel13, Miroslav Živičnjak14. 1. Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625, Hannover, Germany. 2. University Children's Hospital Rostock, Rostock, Germany. 3. University Children's Hospital Heidelberg, Heidelberg, Germany. 4. University Children's Hospital Erlangen, Erlangen, Germany. 5. University Children's Hospital Mainz, Mainz, Germany. 6. Department of Pediatric Nephrology, University Children's Hospital, Charité, Berlin, Germany. 7. Department of Pediatrics, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany. 8. University Children's Hospital Tübingen, Tübingen, Germany. 9. Children's Hospital of the Technical University Munich, Munich, Germany. 10. University Children's Hospital Greifswald, Greifswald, Germany. 11. Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics and Adolescent Medicine, University of Lübeck, Lübeck, Germany. 12. University Children's Hospital Münster, 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. 14. Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625, Hannover, Germany. zivicnjak.miroslav@mh-hannover.de.
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.
RCT Entities:
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.
Authors: G S Reusz; G Miltényi; G Stubnya; A Szabó; C Horváth; D J Byrd; F Péter; T Tulassay Journal: Pediatr Nephrol Date: 1997-10 Impact factor: 3.714
Authors: M Živičnjak; D Schnabel; H Staude; G Even; M Marx; R Beetz; M Holder; H Billing; D-C Fischer; W Rabl; M Schumacher; O Hiort; D Haffner Journal: J Clin Endocrinol Metab Date: 2011-10-12 Impact factor: 5.958
Authors: Michaël R Laurent; Jean De Schepper; Dominique Trouet; Nathalie Godefroid; Emese Boros; Claudine Heinrichs; Bert Bravenboer; Brigitte Velkeniers; Johan Lammens; Pol Harvengt; Etienne Cavalier; Jean-François Kaux; Jacques Lombet; Kathleen De Waele; Charlotte Verroken; Koenraad van Hoeck; Geert R Mortier; Elena Levtchenko; Johan Vande Walle Journal: Front Endocrinol (Lausanne) Date: 2021-03-19 Impact factor: 5.555