Intravenous pamidronate in osteogenesis imperfecta type VII

Calcif Tissue Int. 2009 Mar;84(3):203-9. doi: 10.1007/s00223-008-9211-9. Epub 2009 Jan 10.

Abstract

Cyclical intravenous treatment with pamidronate is widely used to treat osteogenesis imperfecta (OI) types I, III, and IV, which are due to dominant mutations affecting collagen type I alpha chains. There is no information about the effects of pamidronate in children with OI type VII, an autosomal-recessive form of OI caused by a mutation in the cartilage-associated protein gene. In this retrospective single-center study, we compared the effects of pamidronate in four girls with OI type VII (age range 3.9-12.7 years) to those in eight girls with OI types caused by collagen type I mutations who were matched for age and disease severity. During 3 years of pamidronate therapy, lumbar spine areal bone mineral density increased and lumbar vertebral bodies improved in shape in patients with OI type VII. Other outcomes such as fracture rates and mobility scores did not show statistically significant changes in this small study cohort. There were no significant side effects noted during the time of follow-up. Thus, intravenous treatment with pamidronate seems to be safe and of some benefit in patients with OI type VII.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Blood Chemical Analysis
  • Bone Density / drug effects
  • Bone Density / physiology
  • Bone Density Conservation Agents / administration & dosage
  • Bone Density Conservation Agents / therapeutic use*
  • Bone and Bones / metabolism
  • Child
  • Child, Preschool
  • Collagen / genetics
  • Collagen Type I / genetics
  • Collagen Type I, alpha 1 Chain
  • Diphosphonates / administration & dosage
  • Diphosphonates / therapeutic use*
  • Female
  • Follow-Up Studies
  • Fractures, Bone / prevention & control*
  • Humans
  • Injections, Intravenous
  • Mutation / genetics
  • Osteogenesis Imperfecta / blood
  • Osteogenesis Imperfecta / drug therapy*
  • Osteogenesis Imperfecta / genetics
  • Pamidronate
  • Retrospective Studies

Substances

  • Bone Density Conservation Agents
  • Collagen Type I
  • Collagen Type I, alpha 1 Chain
  • Diphosphonates
  • Collagen
  • Pamidronate