Osteogenesis imperfecta: a new, early therapeutic approach with biphosphonates. A case report

• Published in: Archives de pédiatrie : organe officiel de la Société française de pédiatrie Vol. 8; no. 2; pp. 172 - 175
• Main Authors: Guillot, M, Eckart, P, Desrosieres, H, Amiour, M, al-Jazayri, Z
• Format: Journal Article
• Language: French
• Published: France 01-02-2001
• Subjects: Age Factors; Alkaline Phosphatase - blood; Bone Density; Diphosphonates - therapeutic use; Drug Administration Schedule; Growth Disorders - etiology; Humans; Infant; Infusions, Intravenous; Male; Osteogenesis Imperfecta - classification; Osteogenesis Imperfecta - complications; Osteogenesis Imperfecta - diagnostic imaging; Osteogenesis Imperfecta - drug therapy; Osteogenesis Imperfecta - metabolism; Pain - etiology; Pamidronate; Radiography; Treatment Outcome
• ISSN: 0929-693X

Management of type III osteogenesis imperfecta (O.I.) (brittle bone disease) is primarily supportive; early introduction of cyclic intravenous pamidronate administration in children younger than 2 years of age is an innovative and promising therapeutic approach.
We present the case of a 6-month-old infant, whose preliminary data have already been partly published, with severe type III O.I. referred because of aching and crumbling from multiple fractures. Cyclic intravenous disodic pamidronate administration improved the clinical status (fracture incidence, pain, growth curve) and biological status (bone density, osseous alkaline phosphatases, urinary desoxypiridoline excretion), allowing a remarkable recovery.
Biphosphonates are a new and innovative therapeutic agent in O.I. Clinical safety, easy administration, and overall efficacy are likely to extend their use in severe type III O.I. from the very first months of life, the time of best efficacy.