TY - JOUR
T1 - The FOP Connection Registry
T2 - Design of an international patient-sponsored registry for Fibrodysplasia Ossificans Progressiva
AU - Mantick, Neal
AU - Bachman, Eric
AU - Baujat, Genevieve
AU - Brown, Matt
AU - Collins, Oliver
AU - De Cunto, Carmen
AU - Delai, Patricia
AU - Eekhoff, Marelise
AU - zum Felde, Roger
AU - Grogan, Donna Roy
AU - Haga, Nobuhiko
AU - Hsiao, Edward
AU - Kantanie, Sharon
AU - Kaplan, Frederick
AU - Keen, Richard
AU - Milosevic, Jelena
AU - Morhart, Rolf
AU - Pignolo, Robert
AU - Qian, Xiaobing
AU - di Rocco, Maja
AU - Scott, Christiaan
AU - Sherman, Adam
AU - Wallace, Marin
AU - Williams, Nicky
AU - Zhang, Keqin
AU - Bogard, Betsy
N1 - Publisher Copyright:
© 2017 Elsevier Inc.
PY - 2018/4
Y1 - 2018/4
N2 - The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry is an international, voluntary, observational study that directly captures demographic and disease information initially from patients with FOP (the patient portal) and in the near future from treating physicians (the physician portal) via a secure web-based tool. It was launched by the International FOP Association (IFOPA) with a guiding vision to develop and manage one unified, global, and coordinated Registry allowing the assembly of the most comprehensive data on FOP. This will ultimately facilitate greater access and sharing of patient data and enable better and faster development of therapies and tracking their long-term treatment effectiveness and safety. This report outlines the FOP Connection Registry's design and procedures for data collection and reporting, as well as the long-term sustainability of Registry. Patient-reported, aggregate data are summarized for the first 196 enrolled patients, representing participation from 42 countries and approximately 25% of the world's known FOP population. Fifty-seven percent of the current Registry participants are female with a mean age of 23.8 years (median = 21 years, range = 1, 76 years). Among the Registry participants who provided their FOP type, 51% reported FOP Classic (R206H), 41% reported FOP Type Unknown, and 8% reported FOP Variant. Patients reported 5.4 years (median = 3.0 years, range = 0, 45.8 years) as the mean age at which they noticed their first FOP symptoms and a mean age at final FOP diagnosis of 7.5 years (median = 5.0 years, range = 0.1, 48.4 years). Information on the patients’ diagnostic journeys in arriving at a correct diagnosis of FOP is also presented. These early patient-reported data suggest that the IFOPA's vision of one, unified, global, and coordinated approach to the FOP Connection Registry is well underway to being realized. In addition, the positive response from the FOP patient community to the initial launch of the Registry's patient portal has created a solid foundation upon which to build the largest international registry for monitoring the clinical progression of FOP among patients.
AB - The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry is an international, voluntary, observational study that directly captures demographic and disease information initially from patients with FOP (the patient portal) and in the near future from treating physicians (the physician portal) via a secure web-based tool. It was launched by the International FOP Association (IFOPA) with a guiding vision to develop and manage one unified, global, and coordinated Registry allowing the assembly of the most comprehensive data on FOP. This will ultimately facilitate greater access and sharing of patient data and enable better and faster development of therapies and tracking their long-term treatment effectiveness and safety. This report outlines the FOP Connection Registry's design and procedures for data collection and reporting, as well as the long-term sustainability of Registry. Patient-reported, aggregate data are summarized for the first 196 enrolled patients, representing participation from 42 countries and approximately 25% of the world's known FOP population. Fifty-seven percent of the current Registry participants are female with a mean age of 23.8 years (median = 21 years, range = 1, 76 years). Among the Registry participants who provided their FOP type, 51% reported FOP Classic (R206H), 41% reported FOP Type Unknown, and 8% reported FOP Variant. Patients reported 5.4 years (median = 3.0 years, range = 0, 45.8 years) as the mean age at which they noticed their first FOP symptoms and a mean age at final FOP diagnosis of 7.5 years (median = 5.0 years, range = 0.1, 48.4 years). Information on the patients’ diagnostic journeys in arriving at a correct diagnosis of FOP is also presented. These early patient-reported data suggest that the IFOPA's vision of one, unified, global, and coordinated approach to the FOP Connection Registry is well underway to being realized. In addition, the positive response from the FOP patient community to the initial launch of the Registry's patient portal has created a solid foundation upon which to build the largest international registry for monitoring the clinical progression of FOP among patients.
KW - Fibrodysplasia Ossificans Progressiva
KW - Heterotopic ossification
KW - International FOP Association
KW - Natural history
KW - Patient-reported
KW - Registry
UR - http://www.scopus.com/inward/record.url?scp=85030321418&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85030321418&partnerID=8YFLogxK
U2 - 10.1016/j.bone.2017.08.032
DO - 10.1016/j.bone.2017.08.032
M3 - Article
C2 - 28866367
AN - SCOPUS:85030321418
SN - 8756-3282
VL - 109
SP - 285
EP - 290
JO - Bone
JF - Bone
ER -