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Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial.
Risitano, Antonio M; Röth, Alexander; Soret, Juliette; Frieri, Camilla; de Fontbrune, Flore Sicre; Marano, Luana; Alashkar, Ferras; Benajiba, Lina; Marotta, Serena; Rozenberg, Izabela; Milojevic, Julie; End, Peter; Nidamarthy, Prasanna K; Junge, Guido; Peffault de Latour, Régis.
Afiliación
  • Risitano AM; Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy; Haematology and Bone Marrow Transplant Unit, AORN Moscati, Avellino, Italy. Electronic address: amrisita@unina.it.
  • Röth A; West German Cancer Center, Department of Hematology and Stem Cell Transplantation, University Hospital Essen, Essen, Germany.
  • Soret J; Hôpital Saint-Louis, Department of Hematology and Bone Marrow Transplant, Paris, France.
  • Frieri C; Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy; French Reference Center for Aplastic and Paroxysmal Nocturnal Haemoglobinuria, Paris, France.
  • de Fontbrune FS; French Reference Center for Aplastic and Paroxysmal Nocturnal Haemoglobinuria, Paris, France.
  • Marano L; Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy; Haematology and Bone Marrow Transplant Unit, AORN Moscati, Avellino, Italy.
  • Alashkar F; West German Cancer Center, Department of Hematology and Stem Cell Transplantation, University Hospital Essen, Essen, Germany.
  • Benajiba L; Hôpital Saint-Louis, Department of Hematology and Bone Marrow Transplant, Paris, France.
  • Marotta S; Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy; Haematology and Bone Marrow Transplant Unit, AORN Cardarelli, Napoli, Italy.
  • Rozenberg I; Novartis Institutes for Biomedical Research, Department of Translational Medicine, Basel, Switzerland.
  • Milojevic J; Novartis Institutes for Biomedical Research, Department of Translational Medicine, Basel, Switzerland.
  • End P; Novartis Institutes for Biomedical Research, Department of Translational Medicine, Basel, Switzerland.
  • Nidamarthy PK; Novartis Healthcare Private, Department of Biostatistics and Pharmacometrics, Hyderabad, India.
  • Junge G; Novartis Institutes for Biomedical Research, Department of Translational Medicine, Basel, Switzerland.
  • Peffault de Latour R; Hôpital Saint-Louis, Department of Hematology and Bone Marrow Transplant, Paris, France; French Reference Center for Aplastic and Paroxysmal Nocturnal Haemoglobinuria, Paris, France.
Lancet Haematol ; 8(5): e344-e354, 2021 May.
Article en En | MEDLINE | ID: mdl-33765419
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Factor B del Complemento / Inactivadores del Complemento / Anticuerpos Monoclonales Humanizados / Hemoglobinuria Paroxística / Hemólisis Tipo de estudio: Clinical_trials Límite: Adult / Aged / Aged80 / Female / Humans / Male / Middle aged Idioma: En Revista: Lancet Haematol Año: 2021 Tipo del documento: Article
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Factor B del Complemento / Inactivadores del Complemento / Anticuerpos Monoclonales Humanizados / Hemoglobinuria Paroxística / Hemólisis Tipo de estudio: Clinical_trials Límite: Adult / Aged / Aged80 / Female / Humans / Male / Middle aged Idioma: En Revista: Lancet Haematol Año: 2021 Tipo del documento: Article