Individualized treatment with denosumab in children with osteogenesis imperfecta - follow up of a trial cohort.

ABSTRACT:

Background

Osteogenesis imperfecta (OI) is a rare disease leading to hereditary bone fragility. Nearly 90% of cases are caused by mutations in the collagen genes COL1A1/A2 (classical OI) leading to multiple fractures, scoliosis, short stature and nonskeletal findings as blue sclera, hypermobility of joints, bone pain and delayed motor function development. Bisphosphonates are used in most moderate and severely affected patients assuming that an increase of bone mineral density might reduce fractures and bone pain in patients with OI. Denosumab as a RANK ligand antibody inhibiting osteoclast maturation has been approved for osteoporosis treatment in adults. First data from small clinical trials promised a high efficacy of Denosumab in children with OI. Aim of this analysis was a ret

SUBMITTER: Hoyer-Kuhn H 

PROVIDER: S-EPMC6751648 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature