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 typeI, 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.
RCT Entities:
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.
Authors: Keren Machol; Trevor D Hadley; Jake Schmidt; David Cuthbertson; Henri Traboulsi; Rodrigo C Silva; Chloe Citron; Sobiah Khan; Kate Citron; Erin Carter; Kenneth Brookler; Jay R Shapiro; Robert D Steiner; Peter H Byers; Francis H Glorieux; Michaela Durigova; Peter Smith; Michael B Bober; Vernon R Sutton; Brendan H Lee; Sandesh C S Nagamani; Cathleen Raggio Journal: Am J Med Genet A Date: 2019-12-26 Impact factor: 2.802
Authors: L A Bradbury; S Barlow; F Geoghegan; R A Hannon; S L Stuckey; J A H Wass; R G G Russell; M A Brown; E L Duncan Journal: Osteoporos Int Date: 2011-07-08 Impact factor: 4.507
Authors: Kyu Sang Joeng; Yi-Chien Lee; Ming-Ming Jiang; Terry K Bertin; Yuqing Chen; Annie M Abraham; Hao Ding; Xiaohong Bi; Catherine G Ambrose; Brendan H Lee Journal: Hum Mol Genet Date: 2014-03-14 Impact factor: 6.150
Authors: Diana Olvera; Rachel Stolzenfeld; Joan C Marini; Michelle S Caird; Kenneth M Kozloff Journal: J Bone Miner Res Date: 2018-05-07 Impact factor: 6.741