Universität zu Köln

Bishop, Nick, Adami, Silvano, Ahmed, S. Faisal, Anton, Jordi ORCID: 0000-0002-8792-4219, Arundel, Paul, Burren, Christine P., Devogelaer, Jean-Pierre, Hangartner, Thomas, Lane, Joseph M., Lorenc, Roman, Makitie, Outi ORCID: 0000-0002-4547-001X, Munns, Craig F., Paredes, Ana, Pavlov, Helene, Plotkin, Horacio, Raggio, Cathleen L., Loreto Reyes, Maria, Schoenau, Eckhard, Semler, Oliver ORCID: 0000-0003-0029-7556, Sillence, David O. and Steiner, Robert D. ORCID: 0000-0003-4177-4590 (2013). Risedronate in children with osteogenesis imperfecta: a randomised, double-blind, placebo-controlled trial. Lancet, 382 (9902). S. 1424 - 1433. NEW YORK: ELSEVIER SCIENCE INC. ISSN 1474-547X

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Abstract

Background Children with osteogenesis imperfecta are often treated with intravenous bisphosphonates. We aimed to assess the safety and efficacy of risedronate, an orally administered third-generation bisphosphonate, in children with the disease. Methods In this multicentre, randomised, parallel, double-blind, placebo-controlled trial, children aged 4-15 years with osteogenesis imperfecta and increased fracture risk were randomly assigned by telephone randomisation system in a 2: 1 ratio to receive either daily risedronate (2.5 or 5 mg) or placebo for 1 year. Study treatment was masked from patients, investigators, and study centre personnel. Thereafter, all children received risedronate for 2 additional years in an open-label extension. The primary efficacy endpoint was percentage change in lumbar spine areal bone mineral density (BMD) at 1 year. The primary efficacy analysis was done by ANCOVA, with treatment, age group, and pooled centre as fixed effects, and baseline as covariate. Analyses were based on the intention-to-treat population, which included all patients who were randomly assigned and took at least one dose of assigned study treatment. The trial is registered with ClinicalTrials.gov, number NCT00106028. Findings Of 147 patients, 97 were randomly assigned to the risedronate group and 50 to the placebo group. Three patients from the risedronate group and one from the placebo group did not receive study treatment, leaving 94 and 49 in the intention-to-treat population, respectively. The mean increase in lumbar spine areal BMD after 1 year was 16.3% in the risedronate group and 7.6% in the placebo group (difference 8.7%, 95% CI 5.7-11.7; p<0.0001). After 1 year, clinical fractures had occurred in 29 (31%) of 94 patients in the risedronate group and 24 (49%) of 49 patients in the placebo group (p=0.0446). During years 2 and 3 (open-label phase), clinical fractures were reported in 46 (53%) of 87 patients in the group that had received risedronate since the start of the study, and 32 (65%) of 49 patients in the group that had been given placebo during the first year. Adverse event profiles were otherwise similar between the two groups, including frequencies of reported upper-gastrointestinal and selected musculoskeletal adverse events. Interpretation Oral risedronate increased areal BMD and reduced the risk of first and recurrent clinical fractures in children with osteogenesis imperfecta, and the drug was generally well tolerated. Risedronate should be regarded as a treatment option for children with osteogenesis imperfecta.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Bishop, Nick UNSPECIFIED UNSPECIFIED UNSPECIFIED Adami, Silvano UNSPECIFIED UNSPECIFIED UNSPECIFIED Ahmed, S. Faisal UNSPECIFIED UNSPECIFIED UNSPECIFIED Anton, Jordi UNSPECIFIED orcid.org/0000-0002-8792-4219 UNSPECIFIED Arundel, Paul UNSPECIFIED UNSPECIFIED UNSPECIFIED Burren, Christine P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Devogelaer, Jean-Pierre UNSPECIFIED UNSPECIFIED UNSPECIFIED Hangartner, Thomas UNSPECIFIED UNSPECIFIED UNSPECIFIED Lane, Joseph M. UNSPECIFIED UNSPECIFIED UNSPECIFIED Lorenc, Roman UNSPECIFIED UNSPECIFIED UNSPECIFIED Makitie, Outi UNSPECIFIED orcid.org/0000-0002-4547-001X UNSPECIFIED Munns, Craig F. UNSPECIFIED UNSPECIFIED UNSPECIFIED Paredes, Ana UNSPECIFIED UNSPECIFIED UNSPECIFIED Pavlov, Helene UNSPECIFIED UNSPECIFIED UNSPECIFIED Plotkin, Horacio UNSPECIFIED UNSPECIFIED UNSPECIFIED Raggio, Cathleen L. UNSPECIFIED UNSPECIFIED UNSPECIFIED Loreto Reyes, Maria UNSPECIFIED UNSPECIFIED UNSPECIFIED Schoenau, Eckhard UNSPECIFIED UNSPECIFIED UNSPECIFIED Semler, Oliver UNSPECIFIED orcid.org/0000-0003-0029-7556 UNSPECIFIED Sillence, David O. UNSPECIFIED UNSPECIFIED UNSPECIFIED Steiner, Robert D. UNSPECIFIED orcid.org/0000-0003-4177-4590 UNSPECIFIED
URN:	urn:nbn:de:hbz:38-473859
DOI:	10.1016/S0140-6736(13)61091-0
Journal or Publication Title:	Lancet
Volume:	382
Number:	9902
Page Range:	S. 1424 - 1433
Date:	2013
Publisher:	ELSEVIER SCIENCE INC
Place of Publication:	NEW YORK
ISSN:	1474-547X
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language INTRAVENOUS PAMIDRONATE TREATMENT; BISPHOSPHONATE; INFANTS Multiple languages Medicine, General & Internal Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/47385