Clinical course in two children with Juvenile Paget's disease during long-term treatment with intravenous bisphosphonates.

ABSTRACT: Juvenile Paget disease (JPD) is an ultra-rare disease, characterized by loss of function of osteoprotegerin. Osteoprotegerin inhibits osteoclast activation via the receptor activator of nuclear factor κB (RANK) pathway. Severely affected children suffer from bone deformities and pain and require long term anti-resorptive treatment. Due to the rarity of the disease, few long-term follow-up data on the clinical course in children are available. In this report, motor development during infancy and early childhood and the activity of the bone disease based on clinical, radiographic and biochemical parameters are reported in 2 children with severe forms of JPD during long term treatment (4 and 14 years) with bisphosphonates. Results of a bone biopsy in patient 1 after 10 years of treatment and

SUBMITTER: Hoppner J 

PROVIDER: S-EPMC8039828 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature