Universität zu Köln

Hoyer-Kuhn, H., Franklin, J., Allo, G., Kron, M., Netzer, C., Eysel, P., Hero, B., Schoenau, E. and Semler, O. (2016). Safety and efficacy of denosumab in children with osteogenesis imperfecta - a first prospective trial. J. Musculoskelet. Neuronal Interact., 16 (1). S. 24 - 33. NAFPLION: JMNI. ISSN 1108-7161

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Abstract

Objectives: Osteogenesis imperfecta (OI) is a rare hereditary disease leading to bone fragility. Denosumab as a RANK ligand antibody inhibiting osteoclast maturation has been approved for osteoporosis treatment in adults. Aim of this study was a 48-week, open-label, pilot study of the safety and efficacy of denosumab in 10 children with OI. Methods: Ten patients (age range: 5.0-11.0 years; at least two years of prior bisphosphonate treatment) with genetically confirmed OI were studied. Denosumab was administered subcutaneously every 12 weeks with 1 mg/kg body weight. Primary endpoint was change of areal bone mineral density (aBMD) using dual energy x-ray absorptiometry of the lumbar spine after 48 weeks. Safety was assessed by bone metabolism markers and adverse event reporting. Results: Mean relative change of lumbar aBMD was +19 % (95%-CI: 7-31%). Lumbar spine aBMD Z-Scores increased from -2.23 +/- 2.03 (mean +/- SD) to -1.27 +/- 2.37 (p=0.0006). Mobility did not change (GMFM-88 +2.72 +/- 4.62% (p=0.16); one-minute walking test +11.00 +/- 15.82 m (p=0.15). No severe side effects occurred. Conclusions: On average, there was a significant increase in lumbar spine aBMD percent change after 48 weeks of denosumab. There was no change in mobility parameters and no serious adverse events. Further trials are necessary to assess long-term side effects and efficacy.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Hoyer-Kuhn, H. UNSPECIFIED UNSPECIFIED UNSPECIFIED Franklin, J. UNSPECIFIED UNSPECIFIED UNSPECIFIED Allo, G. UNSPECIFIED UNSPECIFIED UNSPECIFIED Kron, M. UNSPECIFIED UNSPECIFIED UNSPECIFIED Netzer, C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Eysel, P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Hero, B. UNSPECIFIED UNSPECIFIED UNSPECIFIED Schoenau, E. UNSPECIFIED UNSPECIFIED UNSPECIFIED Semler, O. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-282972
Journal or Publication Title:	J. Musculoskelet. Neuronal Interact.
Volume:	16
Number:	1
Page Range:	S. 24 - 33
Date:	2016
Publisher:	JMNI
Place of Publication:	NAFPLION
ISSN:	1108-7161
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language GIANT-CELL TUMOR; POSTMENOPAUSAL WOMEN; BONE TURNOVER; PAMIDRONATE TREATMENT; ANTIBODY DENOSUMAB; MARKERS; ALENDRONATE; DEFORMITY; THERAPY; LIGAND Multiple languages Neurosciences; Physiology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/28297