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Long-Term Bisphosphonate Therapy in Osteogenesis Imperfecta

  • Pediatrics (L Ward and E Imel, Section Editors)
  • Published:
Current Osteoporosis Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Osteogenesis imperfecta (OI) is a genetic bone disorder resulting in bone fragility. It has a heterogeneous phenotype which typically includes reduced bone mass, multiple fractures, deformity, and chronic disability. Bisphosphonate treatment remains the first-line medical management, but there is still debate on aspects of its effectiveness. This review summarizes current knowledge about long-term bisphosphonate use in OI with recommendations on clinical application.

Recent Findings

Bisphosphonates increase bone mineral density, most notably of the vertebrae, and reduce fracture risk in the pediatric OI population. Gains in strength and mobility, together with the permissive effect on orthopedic surgery (e.g., in combination with intramedullary rodding) and physiotherapy, have resulted in improved quality of life for those with OI.

Summary

As experience in its use continues, the risks and benefits of long-term bisphosphonate treatment in OI are slowly emerging. Patient registries containing data on genotype, phenotype, fractures, bisphosphonate treatment, orthopedic intervention, and functional outcomes are essential for systematic evaluation given the lack of large multi-centered randomized control trials.

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Authors and Affiliations

  1. Institute of Endocrinology & Diabetes, Children’s Hospital Westmead, Westmead, NSW, Australia

    A. Biggin & C. F. Munns

  2. Discipline of Child & Adolescent Health, University of Sydney, Locked Bag 4001, Westmead, NSW, 2145, Australia

    A. Biggin & C. F. Munns

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Correspondence to A. Biggin.

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Conflict of Interest

Andrew Biggin and Craig Munns report participating in drug trials sponsored by Novartis. Dr. Munns has received funding for trials from Novartis.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Pediatrics

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Biggin, A., Munns, C.F. Long-Term Bisphosphonate Therapy in Osteogenesis Imperfecta. Curr Osteoporos Rep 15, 412–418 (2017). https://doi.org/10.1007/s11914-017-0401-0

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