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

Abstract

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.

Osteochemonecrosis: An Overview

“…Of additional interest may be the study or review of bisphosphonate use in children with osteogenesis imperfecta. At the current time no cases of osteonecrosis of the jaw in children receiving bisphosphonates have been reported, even after multiple tooth extractions [47][48][49].…”

THERAPY OF ENDOCRINE DISEASE: Treatment of osteogenesis imperfecta in adults

“…Individuals with mild OI are treated conservatively in most centers. In moderateto-severe cases, with multiple long-bone fractures and/or vertebral compression fractures, bisphosphonate treatment is being used in children and often initiated at a young age, even in infancy (59,60,61,62,63). However, considerably less is known about how best to treat adults with moderate-to-severe OI, as only few trials, each comprising small numbers of treated patients with different OI types, have been published.…”

“…In humans, bisphosphonates have been shown to increase lumbar spine BMD, ameliorate negative bone phenotypes, and improve vertebral height and areal measurements in children with OI (59,61,63,65,66,67). However, initial reports on decreased pain and improved ambulation regrettably have not been possible to replicate in later controlled trials, and data on fracture reduction are equivocal (62,63,68). Recently a randomised, double-blind, placebo-controlled trial of orally administrated risedronate in children with predominantly mild OI has demonstrated a reduction in the rate of clinical fractures (69).…”

Whole-Body Vibration Exercise Improves Functional Parameters in Patients With Osteogenesis Imperfecta: A Systematic Review With a Suitable Approach

“…Children born with OI are given medication from an early age (2-5 years) and often for long periods at a time aming to try to improve the impact on the growing skeleton (Van Dijk et al, 2014). The evidence for this effect on reducing the risk for fractures is however not clear (Castillo and Samson-Fang, 2009). The accumulation of those drugs and the impact thereof on bone remodelling over long periods results in highly mineralized bone of poor quality (Forlino et al, 2011).…”