在 Paroxysmal 夜间血红蛋白尿症的口服伊普塔科潘单疗法

Régis Peffault de Latour¹, Alexander Röth¹, Austin G Kulasekararaj¹+50

• ¹From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).