Effects of bisphosphonates in children with osteogenesis imperfecta: an AACPDM systematic review


    Members of the American Academy for Cerebral Palsy and Developmental Medicine Treatment Outcomes Committee Review Panel: Lisa Samson-Fang MD, Lesly Wiart MSC PT, Laura Vogtle PhD PT, Johanna Darrah PhD, Meg Barry-Michaels PhD PT PCS, Robbin Hickman PT MHSPCS, John McLaughlin MD, Lynne Logan MA PT, Michael Msall MD, Alexander Hoon MD, William Walker MD, Unni Narayanan MD.

Lisa Samson-Fang at Department of Pediatrics, University of Utah School of Medicine, 50 North Medical Drive, Salt Lake City, UT 84132, USA. E-mail: Lisa.Samson-Fang@hsc.utah.edu


This systematic review of the effects of bisphosphonate treatment in children with osteogenesis imperfecta was conducted using the American Academy for Cerebral Palsy and Developmental Medicine methodology for developing systematic reviews of treatment interventions (Revision 1.1) 2004. Despite a large body of published literature, there have been only eight studies with a sufficiently high level of internal validity to be truly informative. These studies confirm improvement in bone density. Many, but not all studies, demonstrate reduction in fracture rate and enhanced growth. There has been extremely limited evaluation of broader treatment impacts such as deformity, need for orthopedic surgery, pain, functioning, or quality of life. Short-term side effects were minimal. Which medication and dosing regimen is optimal and how long patients should be treated are unclear. This body of evidence would be strengthened by a larger controlled trial, because many studies lacked adequate power to evaluate stated outcomes. These studies do not address the impacts of bisphosphonates in children with milder forms of osteogenesis imperfecta and severe forms that are not due to mutations in the type I pro-collagen gene (e.g. types VII and VIII). Additional research is needed into treatment of infants. More studies evaluating medication choices, optimal dosing, duration of treatment, post-treatment impacts, and long-term side effects are necessary.