Consideration of Rituximab for fibrodysplasia ossificans progressiva.
Fibrodysplasia ossificans progressiva (FOP) is a severe, progressive disease of the musculoskeletal system. Muscles, tendons and other connective tissues ossify after minor trauma, and patients often become encased in a second immobile skeleton. There is no known cure or treatment for FOP. It has been found that lymphocytes from FOP patients elaborate excess levels of bone morphogenic protein-4 (BMP-4). Given this, it has been suggested that allogenic bone marrow transplantation (BMT) possibly could be a cure for FOP, and drawn attention to a previously unappreciated case of an FOP patient who had successful BMT for aplastic anemia with apparent short- and medium-term arresting of the FOP disease process. However, BMT has non-trivial associated morbidity and mortality. Here, it is noted that if B cells are found to be the lymphocytes responsible for excess BMP-4 production in FOP, use of Rituximab, a monoclonal anti-CD20 antibody which effectively targets B cells, could be a less permanent and less risky treatment alternative for FOP.
Altschuler
Eric Lewin
EL
Rehabitilation Medicine, Mt. Sinai School of Medicine, 1425 Madison Avenue, P.O. Box 1240, NY 10029, USA. eric.altschuler@mssm.edu
D000911:Antibodies, Monoclonal / Q000008:administration & dosage*; D058846:Antibodies, Monoclonal, Murine-Derived; D001402:B-Lymphocytes / Q000187:drug effects* / Q000276:immunology*; D016026:Bone Marrow Transplantation / Q000379:methods; D002986:Clinical Trials as Topic; D019317:Evidence-Based Medicine; D006801:Humans; D009221:Myositis Ossificans / Q000188:drug therapy* / Q000276:immunology* / Q000601:surgery; D000069283:Rituximab; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000069283:Rituximab