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

Nadine Meyerhoff; Dieter Haffner; Hagen Staude; Elke Wühl; Michaela Marx; Rolf Beetz; Uwe Querfeld; Martin Holder; Heiko Billing; Wolfgang Rabl; Carmen Schröder; Olaf Hiort; Jürgen H. Brämswig; Annette Richter-Unruh; Dirk Schnabel; Miroslav Živičnjak

doi:10.1007/s00467-017-3820-3

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

Nadine Meyerhoff, Dieter Haffner, Hagen Staude, Elke Wühl, Michaela Marx, Rolf Beetz, Uwe Querfeld, Martin Holder, Heiko Billing, Wolfgang Rabl, Carmen Schröder, Olaf Hiort, Jürgen H. Brämswig, Annette Richter-Unruh, Dirk Schnabel, Miroslav Živičnjak^*

^*Corresponding author for this work

Clinic of Pediatric and Adolescent Medicine

41 Citations (Scopus)

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.

Original language	English
Journal	Pediatric Nephrology
Volume	33
Issue number	3
Pages (from-to)	447-456
Number of pages	10
ISSN	0931-041X
DOIs	https://doi.org/10.1007/s00467-017-3820-3
Publication status	Published - 01.03.2018

Funding

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 f?r Kinderendokrinologie und -diabetologie? (German Society for Pediatric Endocrinology and Diabetology) and the ?Gesellschaft f?r P?diatrische 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. W?lfel (Children?s Hospital G?ppingen, 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. The study received full approval from the Ethics Committee of the Charit? Hospital, Berlin and each participating center. Assent and written informed consent for participation was obtained from all patients and/or their parents.

Research Areas and Centers

Academic Focus: Center for Brain, Behavior and Metabolism (CBBM)

DFG Research Classification Scheme

2.22-17 Endocrinology, Diabetology, Metabolism
2.22-20 Pediatric and Adolescent Medicine

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1007/s00467-017-3820-3

Cite this

Meyerhoff, N., Haffner, D., Staude, H., Wühl, E., Marx, M., Beetz, R., Querfeld, U., Holder, M., Billing, H., Rabl, W., Schröder, C., Hiort, O., Brämswig, J. H., Richter-Unruh, A., Schnabel, D., & Živičnjak, M. (2018). Effects of growth hormone treatment on adult height in severely short children with X-linked hypophosphatemic rickets. Pediatric Nephrology, 33(3), 447-456. https://doi.org/10.1007/s00467-017-3820-3

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

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.",

author = "Nadine Meyerhoff and Dieter Haffner and Hagen Staude and Elke W{\"u}hl and Michaela Marx and Rolf Beetz and Uwe Querfeld and Martin Holder and Heiko Billing and Wolfgang Rabl and Carmen Schr{\"o}der and Olaf Hiort and Br{\"a}mswig, \{J{\"u}rgen H.\} and Annette Richter-Unruh and Dirk Schnabel and Miroslav {\v Z}ivi{\v c}njak",

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doi = "10.1007/s00467-017-3820-3",

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Meyerhoff, N, Haffner, D, Staude, H, Wühl, E, Marx, M, Beetz, R, Querfeld, U, Holder, M, Billing, H, Rabl, W, Schröder, C, Hiort, O, Brämswig, JH, Richter-Unruh, A, Schnabel, D & Živičnjak, M 2018, 'Effects of growth hormone treatment on adult height in severely short children with X-linked hypophosphatemic rickets', Pediatric Nephrology, vol. 33, no. 3, pp. 447-456. https://doi.org/10.1007/s00467-017-3820-3

TY - JOUR

T1 - Effects of growth hormone treatment on adult height in severely short children with X-linked hypophosphatemic rickets

AU - Meyerhoff, Nadine

AU - Haffner, Dieter

AU - Staude, Hagen

AU - Wühl, Elke

AU - Marx, Michaela

AU - Beetz, Rolf

AU - Querfeld, Uwe

AU - Holder, Martin

AU - Billing, Heiko

AU - Rabl, Wolfgang

AU - Schröder, Carmen

AU - Hiort, Olaf

AU - Brämswig, Jürgen H.

AU - Richter-Unruh, Annette

AU - Schnabel, Dirk

AU - Živičnjak, Miroslav

PY - 2018/3/1

Y1 - 2018/3/1

N2 - 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.

AB - 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.

UR - https://www.scopus.com/pages/publications/85031921549

U2 - 10.1007/s00467-017-3820-3

DO - 10.1007/s00467-017-3820-3

M3 - Journal articles

C2 - 29058153

AN - SCOPUS:85031921549

SN - 0931-041X

VL - 33

SP - 447

EP - 456

JO - Pediatric Nephrology

JF - Pediatric Nephrology

IS - 3

ER -

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

Abstract

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Research Areas and Centers

DFG Research Classification Scheme

UN SDGs

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