Alendronate for the Treatment of Pediatric Osteogenesis Imperfecta: A Randomized Placebo-Controlled Study

Oral alendronate (5 or 10 mg daily) for 2 years in children with osteogenesis imperfecta was well-tolerated, significantly increased spine bone mineral density, and decreased bone turnover. Context: Information on the use of oral bisphosphonate agents to treat pediatric osteogenesis imperfecta (OI)...

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Published in:The journal of clinical endocrinology and metabolism 2011-02, Vol.96 (2), p.355-364
Main Authors: Ward, L. M, Rauch, F, Whyte, M. P, D'Astous, J, Gates, P. E, Grogan, D, Lester, E. L, McCall, R. E, Pressly, T. A, Sanders, J. O, Smith, P. A, Steiner, R. D, Sullivan, E, Tyerman, G, Smith-Wright, D. L, Verbruggen, N, Heyden, N, Lombardi, A, Glorieux, F. H
Format: Article
Language:English
Subjects:
Adolescent
Alendronate - adverse effects
Alendronate - therapeutic use
Biological and medical sciences
Bone and Bones - diagnostic imaging
Bone and Bones - metabolism
Bone Density
Bone Density Conservation Agents - adverse effects
Bone Density Conservation Agents - therapeutic use
Child
Child, Preschool
Double-Blind Method
Endocrinopathies
Feeding. Feeding behavior
Female
Follow-Up Studies
Fracture Healing - drug effects
Fractures, Bone - epidemiology
Fundamental and applied biological sciences. Psychology
Humans
Ilium - diagnostic imaging
Ilium - pathology
Male
Medical sciences
Muscle Strength - physiology
Osteogenesis Imperfecta - drug therapy
Osteogenesis Imperfecta - metabolism
Osteogenesis Imperfecta - pathology
Pain - etiology
Patient Compliance
Radiography
Self Care
Spine - diagnostic imaging
Vertebrates: anatomy and physiology, studies on body, several organs or systems
Vertebrates: endocrinology
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Summary:Oral alendronate (5 or 10 mg daily) for 2 years in children with osteogenesis imperfecta was well-tolerated, significantly increased spine bone mineral density, and decreased bone turnover. Context: Information on the use of oral bisphosphonate agents to treat pediatric osteogenesis imperfecta (OI) is limited. Objective: The objective of the investigation was to study the efficacy and safety of daily oral alendronate (ALN) in children with OI. Design and Participants: We conducted a multicenter, double-blind, randomized, placebo-controlled study. One hundred thirty-nine children (aged 4–19 yr) with type I, III, or IV OI were randomized to either placebo (n = 30) or ALN (n = 109) for 2 yr. ALN doses were 5 mg/d in children less than 40 kg and 10 mg/d for those 40 kg and greater. Main Outcome Measures: Spine areal bone mineral density (BMD) z-score, urinary N-telopeptide of collagen type I, extremity fracture incidence, vertebral area, iliac cortical width, bone pain, physical activity, and safety parameters were measured. Results: ALN increased spine areal BMD by 51% vs. a 12% increase with placebo (P < 0.001); the mean spine areal BMD z-score increased significantly from −4.6 to −3.3 (P < 0.001) with ALN, whereas the change in the placebo group (from −4.6 to −4.5) was insignificant. Urinary N-telopeptide of collagen type I decreased by 62% in the ALN-treated group, compared with 32% with placebo (P < 0.001). Long-bone fracture incidence, average midline vertebral height, iliac cortical width, bone pain, and physical activity were similar between groups. The incidences of clinical and laboratory adverse experiences were also similar between the treatment and placebo groups. Conclusions: Oral ALN for 2 yr in pediatric patients with OI significantly decreased bone turnover and increased spine areal BMD but was not associated with improved fracture outcomes.
ISSN:0021-972X
1945-7197
DOI:10.1210/jc.2010-0636