Emerging treatments

Teriparatide

Teriparatide, a recombinant formulation of parathyroid hormone (PTH), is an anabolic bone agent that has been shown to increase bone mineral density and estimated bone strength in adults with mild, but not severe, forms of OI.[86] The Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic Acid (TOPaZ) trial aims to determine if 2 years of treatment with teriparatide followed by treatment with zoledronic acid reduces the risk of fracture in OI by comparison with standard care.[87] Based on experience in practice, a specialist may consider the use of teriparatide in adults with OI with one or more of the following: very low areal bone mineral density in the lumbar spine (e.g., T-score <-3.0); vertebral fractures; absence of response to treatment with bisphosphonates. Teriparatide is contraindicated in children.

Monoclonal antibodies

Monoclonal antibodies that inhibit sclerostin and transforming growth factor beta are under investigation in clinical trials as therapeutic options for OI.[88][89][90][91]

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