Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.

Humans ADAMTS13 Protein / deficiency Purpura, Thrombotic Thrombocytopenic / drug therapy Recombinant Proteins / therapeutic use Male Female Adult Cross-Over Studies Young Adult Adolescent Middle Aged Child

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:
02 May 2024
Historique:
medline: 2 5 2024
pubmed: 2 5 2024
entrez: 1 5 2024
Statut: ppublish

Résumé

Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known. In this phase 3, open-label, crossover trial, we randomly assigned patients in a 1:1 ratio to two 6-month periods of prophylaxis with recombinant ADAMTS13 (40 IU per kilogram of body weight, administered intravenously) or standard therapy, followed by the alternate treatment; thereafter, all the patients received recombinant ADAMTS13 for an additional 6 months. The trigger for this interim analysis was trial completion by at least 30 patients. The primary outcome was acute TTP events. Manifestations of TTP, safety, and pharmacokinetics were assessed. Patients who had an acute TTP event could receive on-demand treatment. A total of 48 patients underwent randomization; 32 completed the trial. No acute TTP event occurred during prophylaxis with recombinant ADAMTS13, whereas 1 patient had an acute TTP event during prophylaxis with standard therapy (mean annualized event rate, 0.05). Thrombocytopenia was the most frequent TTP manifestation (annualized event rate, 0.74 with recombinant ADAMTS13 and 1.73 with standard therapy). Adverse events occurred in 71% of the patients with recombinant ADAMTS13 and in 84% with standard therapy. Adverse events that were considered by investigators to be related to the trial drug occurred in 9% of the patients with recombinant ADAMTS13 and in 48% with standard therapy. Trial-drug interruption or discontinuation due to adverse events occurred in no patients with recombinant ADAMTS13 and in 8 patients with standard therapy. No neutralizing antibodies developed during recombinant ADAMTS13 treatment. The mean maximum ADAMTS13 activity after recombinant ADAMTS13 treatment was 101%, as compared with 19% after standard therapy. During prophylaxis with recombinant ADAMTS13 in patients with congenital TTP, ADAMTS13 activity reached approximately 100% of normal levels, adverse events were generally mild or moderate in severity, and TTP events and manifestations were rare. (Funded by Takeda Development Center Americas and Baxalta Innovations; ClinicalTrials.gov number, NCT03393975.).

Sections du résumé

BACKGROUND BACKGROUND
Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known.
METHODS METHODS
In this phase 3, open-label, crossover trial, we randomly assigned patients in a 1:1 ratio to two 6-month periods of prophylaxis with recombinant ADAMTS13 (40 IU per kilogram of body weight, administered intravenously) or standard therapy, followed by the alternate treatment; thereafter, all the patients received recombinant ADAMTS13 for an additional 6 months. The trigger for this interim analysis was trial completion by at least 30 patients. The primary outcome was acute TTP events. Manifestations of TTP, safety, and pharmacokinetics were assessed. Patients who had an acute TTP event could receive on-demand treatment.
RESULTS RESULTS
A total of 48 patients underwent randomization; 32 completed the trial. No acute TTP event occurred during prophylaxis with recombinant ADAMTS13, whereas 1 patient had an acute TTP event during prophylaxis with standard therapy (mean annualized event rate, 0.05). Thrombocytopenia was the most frequent TTP manifestation (annualized event rate, 0.74 with recombinant ADAMTS13 and 1.73 with standard therapy). Adverse events occurred in 71% of the patients with recombinant ADAMTS13 and in 84% with standard therapy. Adverse events that were considered by investigators to be related to the trial drug occurred in 9% of the patients with recombinant ADAMTS13 and in 48% with standard therapy. Trial-drug interruption or discontinuation due to adverse events occurred in no patients with recombinant ADAMTS13 and in 8 patients with standard therapy. No neutralizing antibodies developed during recombinant ADAMTS13 treatment. The mean maximum ADAMTS13 activity after recombinant ADAMTS13 treatment was 101%, as compared with 19% after standard therapy.
CONCLUSIONS CONCLUSIONS
During prophylaxis with recombinant ADAMTS13 in patients with congenital TTP, ADAMTS13 activity reached approximately 100% of normal levels, adverse events were generally mild or moderate in severity, and TTP events and manifestations were rare. (Funded by Takeda Development Center Americas and Baxalta Innovations; ClinicalTrials.gov number, NCT03393975.).

Identifiants

DOI: 10.1056/NEJMoa2314793 PMID: 38692292
pubmed: 38692292
doi: 10.1056/NEJMoa2314793
doi:

Substances chimiques

ADAMTS13 Protein EC 3.4.24.87
Recombinant Proteins 0
ADAMTS13 protein, human EC 3.4.24.87

Banques de données

ClinicalTrials.gov
['NCT03393975']

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

1584-1596

Subventions

Organisme : Takeda Pharmaceutical Company
ID : Funded by Takeda Development Center Americas, Inc.

Investigateurs

Paul Knöbl (P)
Claire Berger (C)
Nathalie Biebuyck (N)
Paul Coppo (P)
Claire Dossier (C)
Wolf-Achim Hassenpflug (WA)
Karim Kentouche (K)
Silvia Maria Trisolini (SM)
Satoshi Higasa (S)
Masataka Ishimura (M)
Michiko Kajiwara (M)
Anna Klukowska (A)
Jerzy Windyga (J)
Miriam Vara Pampliega (MV)
Santiago Bonanad (S)
Maria Fernanda (M)
Lopez Fernandez (L)
Pascual Marco (P)
Vera Marie Scully (VM)
Ana Antun (A)
Spero Cataland (S)
Thomas Ortel (T)
Sami Ibrahimi (S)

Informations de copyright

Copyright © 2024 Massachusetts Medical Society.

Auteurs

Marie Scully (M)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Ana Antun (A)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Spero R Cataland (SR)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Paul Coppo (P)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Claire Dossier (C)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Nathalie Biebuyck (N)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Wolf-Achim Hassenpflug (WA)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Karim Kentouche (K)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Paul Knöbl (P)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Johanna A Kremer Hovinga (JA)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

M Fernanda López-Fernández (MF)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Masanori Matsumoto (M)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Thomas L Ortel (TL)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Jerzy Windyga (J)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Indranil Bhattacharya (I)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Michael Cronin (M)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Hong Li (H)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Björn Mellgård (B)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Munjal Patel (M)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Parth Patwari (P)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Shan Xiao (S)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Pinghai Zhang (P)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Linda T Wang (LT)

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

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Classifications MeSH

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