Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.

Adult Aged Aged, 80 and over Antibodies, Monoclonal, Humanized / adverse effects Complement C3 / antagonists & inhibitors Complement C5 / antagonists & inhibitors Complement Inactivating Agents / adverse effects Diarrhea / chemically induced Drug Therapy, Combination Erythrocyte Transfusion Hemoglobins / analysis Hemoglobinuria, Paroxysmal / blood Humans Injections, Subcutaneous / adverse effects Middle Aged Peptides / adverse effects Peptides, Cyclic

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
18 03 2021
Historique:
entrez: 17 3 2021
pubmed: 18 3 2021
medline: 31 3 2021
Statut: ppublish

Résumé

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan, a pegylated peptide targeting proximal complement protein C3, potentially inhibits both intravascular and extravascular hemolysis. We conducted a phase 3 open-label, controlled trial to assess the efficacy and safety of pegcetacoplan as compared with eculizumab in adults with PNH and hemoglobin levels lower than 10.5 g per deciliter despite eculizumab therapy. After a 4-week run-in phase in which all patients received pegcetacoplan plus eculizumab, we randomly assigned patients to subcutaneous pegcetacoplan monotherapy (41 patients) or intravenous eculizumab (39 patients). The primary end point was the mean change in hemoglobin level from baseline to week 16. Additional clinical and hematologic markers of hemolysis and safety were assessed. Pegcetacoplan was superior to eculizumab with respect to the change in hemoglobin level from baseline to week 16, with an adjusted (least squares) mean difference of 3.84 g per deciliter (P<0.001). A total of 35 patients (85%) receiving pegcetacoplan as compared with 6 patients (15%) receiving eculizumab no longer required transfusions. Noninferiority of pegcetacoplan to eculizumab was shown for the change in absolute reticulocyte count but not for the change in lactate dehydrogenase level. Functional Assessment of Chronic Illness Therapy-Fatigue scores improved from baseline in the pegcetacoplan group. The most common adverse events that occurred during treatment in the pegcetacoplan and eculizumab groups were injection site reactions (37% vs. 3%), diarrhea (22% vs. 3%), breakthrough hemolysis (10% vs. 23%), headache (7% vs. 23%), and fatigue (5% vs. 15%). There were no cases of meningitis in either group. Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients with PNH by providing broad hemolysis control, including control of intravascular and extravascular hemolysis. (Funded by Apellis Pharmaceuticals; PEGASUS ClinicalTrials.gov, NCT03500549.).

Sections du résumé

BACKGROUND
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan, a pegylated peptide targeting proximal complement protein C3, potentially inhibits both intravascular and extravascular hemolysis.
METHODS
We conducted a phase 3 open-label, controlled trial to assess the efficacy and safety of pegcetacoplan as compared with eculizumab in adults with PNH and hemoglobin levels lower than 10.5 g per deciliter despite eculizumab therapy. After a 4-week run-in phase in which all patients received pegcetacoplan plus eculizumab, we randomly assigned patients to subcutaneous pegcetacoplan monotherapy (41 patients) or intravenous eculizumab (39 patients). The primary end point was the mean change in hemoglobin level from baseline to week 16. Additional clinical and hematologic markers of hemolysis and safety were assessed.
RESULTS
Pegcetacoplan was superior to eculizumab with respect to the change in hemoglobin level from baseline to week 16, with an adjusted (least squares) mean difference of 3.84 g per deciliter (P<0.001). A total of 35 patients (85%) receiving pegcetacoplan as compared with 6 patients (15%) receiving eculizumab no longer required transfusions. Noninferiority of pegcetacoplan to eculizumab was shown for the change in absolute reticulocyte count but not for the change in lactate dehydrogenase level. Functional Assessment of Chronic Illness Therapy-Fatigue scores improved from baseline in the pegcetacoplan group. The most common adverse events that occurred during treatment in the pegcetacoplan and eculizumab groups were injection site reactions (37% vs. 3%), diarrhea (22% vs. 3%), breakthrough hemolysis (10% vs. 23%), headache (7% vs. 23%), and fatigue (5% vs. 15%). There were no cases of meningitis in either group.
CONCLUSIONS
Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients with PNH by providing broad hemolysis control, including control of intravascular and extravascular hemolysis. (Funded by Apellis Pharmaceuticals; PEGASUS ClinicalTrials.gov, NCT03500549.).

Identifiants

DOI: 10.1056/NEJMoa2029073 PMID: 33730455
pubmed: 33730455
doi: 10.1056/NEJMoa2029073
doi:

Substances chimiques

Antibodies, Monoclonal, Humanized 0
Complement C3 0
Complement C5 0
Complement Inactivating Agents 0
Hemoglobins 0
Peptides 0
Peptides, Cyclic 0
eculizumab A3ULP0F556
pegcetacoplan TO3JYR3BOU

Banques de données

ClinicalTrials.gov
['NCT03500549']

Types de publication

Clinical Trial, Phase III Comparative Study Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1028-1037

Commentaires et corrections

Type : CommentIn
Type : CommentIn
Type : CommentIn

Informations de copyright

Copyright © 2021 Massachusetts Medical Society.

Auteurs

Peter Hillmen (P)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Jeff Szer (J)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Ilene Weitz (I)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Alexander Röth (A)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Britta Höchsmann (B)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Jens Panse (J)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Kensuke Usuki (K)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Morag Griffin (M)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Jean-Jacques Kiladjian (JJ)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Carlos de Castro (C)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Hisakazu Nishimori (H)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Lisa Tan (L)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Mohamed Hamdani (M)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Pascal Deschatelets (P)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Cedric Francois (C)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Federico Grossi (F)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Temitayo Ajayi (T)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Antonio Risitano (A)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

Régis Peffault de la Tour (R)

From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).

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questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Pegcetacoplan versus Eculizumab in Paroxysmal...
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