Orphanet Journal of Rare Diseases
International physician survey on management of FOP: a modified Delphi study | Orphanet Journal of Rare Diseases | Full Text
International physician survey on management of FOP: a modified Delphi study
Orphanet Journal of Rare Diseases201712:110
DOI: 10.1186/s13023-017-0659-4
© The Author(s). 2017
Received: 28 February 2017
Accepted: 19 May 2017
Published: 12 June 2017
Abstract
Fibrodysplasia ossificans progressiva (FOP), a disabling disorder of progressive heterotopic ossification (HEO), is caused by heterozygous gain-of- function mutations in Activin receptor A, type I (ACVR1, also known as ALK2), a bone morphogenetic protein (BMP) type I receptor. Presently, symptomatic management is possible, but no definitive treatments are available. Although extensive guidelines for symptomatic management are widely used, regional preferences exist. In order to understand if there was worldwide consensus among clinicians treating FOP patients, an expert panel of physicians directly involved in FOP patient care was convened. Using a modified Delphi method, broad international consensus was reached on four main topics: diagnosis, prevention of flare-ups, patient and family-centered care and general clinical management issues. This study of physician preferences provides a basis for standardization of clinical management for FOP.
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