Journal article

Fabry in the older patient: Clinical consequences and possibilities for treatment

Olivier Lidove, Frederic Barbey, Dau-Ming Niu, Eva Brand, Kathleen Nicholls, Svetlana Bizjajeva, Derralynn A Hughes

Molecular Genetics and Metabolism | ACADEMIC PRESS INC ELSEVIER SCIENCE | Published : 2016


Baseline demographic and phenotypic characteristics of patients aged ≥50years in the Fabry Outcome Survey (Shire; data extracted June 2014) were compared with younger adults to investigate potential factors influencing treatment decisions in later life. Age groups were defined using age at treatment initiation or at FOS entry for untreated patients: 18-49 (n=1344; 49.5% male; 64.6% received agalsidase alfa enzyme replacement therapy [ERT]); 50-64 (n=537; 35.4% male; 74.3% treated); 65-74 (n=137; 32.1% male; 68.6% treated); and ≥75years (n=26; 26.9% male; 50.0% treated). Successive age groups showed higher median age at first symptom and diagnosis. Median alpha-galactosidase A activity, measu..

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Funding Acknowledgements

Shire provided funding in the form of a salary for author Svetlana Bizjajeva, and also funded medical writing support, provided by Tina Rose of Excel Scientific Solutions. The authors would like to thank the following investigators, and their patients, who contributed data to FOS: Dr. Aaron, Hopital Jean Jacques Coeur, France; Dr. Andrade Campos, Hospital Universitario Miguel Servet, Spain; Dr. Arbustini, Fondazione IRCCS Policlinico San Matteo, Italy; Dr. Arnalich Fernandez, Hospital Universitario La Paz, Spain; Dr. Avila, Hospital Universitario Doctor Peset, Spain; Dr. Azevedo, Centro Hospitalar do Alto Ave, EPE - Hospital de Guimaraes, Portugal; Dr. Barba, Complejo Hospitalario Universitario de Albacete, Spain; Dr. Bataille, CHU Boulogne sur Mer, France; Dr. Bichet, Hopital Du Sacre-Coeur de Montreal, Canada; Dr. Borsini, Azienda Ospedaliero-Universitaria Careggi, Italy; Dr. Bosch, Hospital Universitario Son Espases, Spain; Dr. Bottcher, Dietrich Bonhoeffer Klinikum Neubrandenburg, Germany; Dr. Boudet, Hopital Brive la Gaillarde, France; Dr. Cabades O'Callaghan, Hospital Comarcal De Vinaros, Spain; Dr. Catassi, Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona-Umberto I-G.M. Lancisi-G. Salesi, Italy; Dr. Cecchi, Azienda Ospedaliero-Universitaria Careggi, Italy; Dr. Chan, University of Alberta, Canada; Dr. Climent, Hospital General Universitario de Alicante, Spain; Dr. Cole, University Hospital of Wales, UK; Dr. Concolino, Azienda Ospedaliera Pugliese Ciaccio, Italy; Dr. de Arriba de la Fuente, Hospital Universitario de Guadalajara, Spain; Dr. De Precigout, Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin, France; Dr. de Toro Santos, Complejo Hospitalario Universitario de Ourense, Spain; Dr. Deegan, Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital, UK; Dr. Degremont, Centre Hospitalier, France; Dr. DiVito, Unita Operativa di Nefrologia e Dialisi, Ospedale G. Bernabeo, Italy; Dr. Dostalova, Vseobecna fakultni nemocnice v Praze, Czech Republic; Dr. Douillard, CHRU Lille, France; Dr. Drobacheff-Thiebaut, Hopital Jean Minjoz, France; Dr. Dussol, Hopital de La Conception, France; Dr. Eto, Jikei University Hospital, Japan; Dr. Eyskens, UZ Antwerpen, Belgium; Dr. Fakhouri, Hotel-Dieu, France; Dr. Feriozzi Italy; Dr. Fernandez Martin, Hospital do Meixoeiro, Spain; Dr. Fouilhoux, Groupement Hospitalier EST-Hopital Mere-Enfant, France; Dr. Franco, Hospital General Universitario de Alicante, Spain; Dr. Fritz, Hopital de la Rochelle, France; Dr. Gaborieau, Hopital Francois Mitterand, France; Dr. Gaedeke, Charite - Universitatsmedizin Berlin, Germany; Dr. Galan Montesano, Hospital Comarcal de Don Benito, Spain; Dr. Garcia Uriarte, Hospital de Santiago Apostol, Spain; Dr. Ghafari, Institut Arnault Tzanck, France; Dr. Giugliani, Hospital de Clinicas de Porto Alegre, Brazil; Dr. Goizet, Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin, France; Dr. Hachulla, CHRU Lille, France; Dr. Hennermann, Universitatsmedizin der Johannes Gutenberg-Universitat Mainz, Germany; Dr. Herrera, Hospital Universitario Central de Asturias, Spain; Dr. Hiwot, Queen Elizabeth Hospital, UK; Dr. Jaussaud, CHU de Reims, France; Dr. Kaminsky, CHU de Nancy-Hopital Brabois Adulte, France; Dr. Kantola, Turun Yliopistollinen Keskussairaala, Finland; Dr. Khan, Alberta Childrens Hospital, Canada; Dr. Kurschat, Universitatsklinikum Koln, Germany; Dr. Lavigne, CHU Angers, France; Dr. Leguy, CHU Dijon, France; Dr. Linthorst, Academisch Medisch Centrum Amsterdam, Netherlands; Dr.Manna, Policlinico Universitario Agostino Gemelli, Italy; Dr. Maranda, Centre Hospitalier Universitaire de Sherbrooke, Canada; Dr. Marodi, Debreceni Egyetem Orvos-es Egeszsegtudomanyi Centrum Kazincbarcikai Korhaz Nonprofit Kft., Hungary; Dr. Martin, Hospital Universitari Arnau de Vilanova, Spain; Dr. Martinez, Hospital de Torrecardenas, Spain; Dr. Mehta, Royal Free Hospital, UK; Dr. Mora, Hospital General Universitario de Elche, Spain; Dr. Morel, Mount Sinai Hospital, Canada; Dr. Moreno, Hospital General Universitario Morales Meseguer; Dr. Muschol, Universitatsklinikum Hamburg-Eppendorf, Germany; Dr. Nassogne, Cliniques Universitaires Saint-Luc, Belgium; Dr. Neumann, Universitatsklinikum Freibiurg, Germany; Dr. Noel, Hopital Civil, France; Dr. Nowak, UniversitatsSpital Zurich, France; Dr. Orosa, Hospital Francesc de Borja, Spain; Dr. Paniagua, Hospital El Bierzo, Spain; Dr. Parini, Azienda Ospedaliera San Gerardo, Italy; Dr. Pastores, New York University School of Medicine; Dr. Perez Lopez, Hospital Universitario Vall d'Hebron, Spain; Dr. Perticoni, SC Neurofisiopatologia, Policlinico, Silvestrini, Italy; Dr. Pintos Morell, Hospital Universitario Germans Trias i Pujol, Spain; Dr. Pisani, Azienda Ospedaliera Universitaria Federico II, Italy; Dr. Ploeckinger, Charite - Campus Virchow-Klinikum, Germany; Dr. Roland, Institut de Pathologie et de Genetique a.s.b.l., Belgium; Dr. Rolfs, University of Rostock; Dr. Rozenfeld, National University of La Plata, Argentina; Dr. Schiffmann, Baylor University Medical Center, US; Dr. Serena Leal, Hospital Universitari de Girona Dr. Josep Trueta, Spain; Dr. Sirrs, Vancouver General Hospital, Canada; Dr. Thevenot, CH Laon, France; Dr. Torra, Fundacio Puigvert, Spain; Dr. Torras, Hospital Universitari de Bellvitge, Spain; Dr. Tuttolomondo, Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone, Italy; Dr. Valverde, Hospital de Elda Virgen de La Salud, Spain; Dr. Vidau, Hospital Universitario Central de Asturias, Spain; Dr. Vijay, Birmingham Children's Hospital NHS Foundation Trust, UK; Dr. Vuillemet, Hopital Louis Pasteur, France; Dr. Vujkovac, General Hospital Slovenj Gradec, Slovenia; Dr. Wanner, Universitatsklinikum Wurzburg, Wanner; Dr. West, Queen Elizabeth II Health Sciences Center, Canada; Dr. Zoli, Ospedale SS. Annunziata, Italy.