OBJECTIVE: To evaluate the functional abilities and the level of ambulation during pamidronate therapy in children with moderate to severe osteogenesis imperfecta. STUDY DESIGN: Functional abilities, ambulation, and grip force were assessed in 59 patients (mean age, 6.1 years; range, 0.5-15.7 years; 30 girls) during 3 years of pamidronate treatment. Functional skills (mobility and self-care) were both assessed by using the Pediatric Evaluation of Disability Inventory. Ambulation level was assessed by using the modified Bleck score. For 48 patients, results after 3 years of pamidronate treatment could be matched to those of patients with similar age and disease severity who had not received pamidronate. RESULTS: Mobility and self-care scores increased during the study period (+43% and +30%, respectively). The average ambulation score changed from 0.8 to 1.9. Maximal isometric grip force increased by 63%. Mobility and ambulation scores and grip force measures were significantly higher than in patients who had not received pamidronate. The difference in self-care scores did not reach significance. CONCLUSION: This study suggests that cyclical pamidronate treatment improves mobility, ambulation level, and muscle force in children with moderate to severe osteogenesis imperfecta.
OBJECTIVE: To evaluate the functional abilities and the level of ambulation during pamidronate therapy in children with moderate to severe osteogenesis imperfecta. STUDY DESIGN: Functional abilities, ambulation, and grip force were assessed in 59 patients (mean age, 6.1 years; range, 0.5-15.7 years; 30 girls) during 3 years of pamidronate treatment. Functional skills (mobility and self-care) were both assessed by using the Pediatric Evaluation of Disability Inventory. Ambulation level was assessed by using the modified Bleck score. For 48 patients, results after 3 years of pamidronate treatment could be matched to those of patients with similar age and disease severity who had not received pamidronate. RESULTS: Mobility and self-care scores increased during the study period (+43% and +30%, respectively). The average ambulation score changed from 0.8 to 1.9. Maximal isometric grip force increased by 63%. Mobility and ambulation scores and grip force measures were significantly higher than in patients who had not received pamidronate. The difference in self-care scores did not reach significance. CONCLUSION: This study suggests that cyclical pamidronate treatment improves mobility, ambulation level, and muscle force in children with moderate to severe osteogenesis imperfecta.
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: Christopher S Constantino; Joseph J Krzak; Alissa V Fial; Karen M Kruger; Jacob R Rammer; Katarina Radmanovic; Peter A Smith; Gerald F Harris Journal: JBMR Plus Date: 2019-10-18
Authors: Karen M Kruger; Angela Caudill; Mercedes Rodriguez Celin; Sandesh C S Nagamani; Jay R Shapiro; Robert D Steiner; Michael B Bober; Tracy Hart; David Cuthbertson; Jeff Krischer; Peter H Byers; Michaela Durigova; Francis H Glorieux; Frank Rauch; V Reid Sutton; Brendan Lee; Eric T Rush; Peter A Smith; Gerald F Harris Journal: Genet Med Date: 2019-03-28 Impact factor: 8.822
Authors: L Ward; A C Tricco; P Phuong; A Cranney; N Barrowman; I Gaboury; F Rauch; P Tugwell; D Moher Journal: Cochrane Database Syst Rev Date: 2007-10-17