Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.

Autor: 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.)., 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.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2024 May 02; Vol. 390 (17), pp. 1584-1596.

OC 14.1 Phase 3 Prospective, Randomized, Controlled, Open-Label, Multicenter, Crossover Study of Recombinant ADAMTS13 in Patients with Congenital Thrombotic Thrombocytopenic Purpura

Autor: Scully, M., Windyga, J., Mellgård, B., Ortel, T., Li, H., Ayash-Rashkovsky, M., Cataland, S., Wang, L.

Publikováno v: In Research and Practice in Thrombosis and Haemostasis October 2023 7 Supplement 2

Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results.

Autor: Leebeek FWG; Department of Hematology, University Medical Center, Erasmus MC, Rotterdam, The Netherlands., Peyvandi F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy., Escobar M; University of Texas Health Science Center at Houston, Houston, TX., Tiede A; Department of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany., Castaman G; Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy., Wang M; Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora, CO., Wynn T; Department of Pediatrics, University of Florida, Gainesville, FL., Baptista J; Takeda Development Center Americas, Inc., Lexington, MA; and., Wang Y; Takeda Development Center Americas, Inc, Cambridge, MA., Zhang J; Takeda Development Center Americas, Inc, Cambridge, MA., Mellgård B; Takeda Development Center Americas, Inc, Cambridge, MA., Özen G; Takeda Development Center Americas, Inc, Cambridge, MA.

Publikováno v: Blood [Blood] 2022 Jul 14; Vol. 140 (2), pp. 89-98.

The Highs and Lows of ADAMTS13 Activity.

Autor: Shaw, Rebecca J.^1,2 (AUTHOR) r.shaw3@nhs.net, Abrams, Simon T.² (AUTHOR) stabrams@liverpool.ac.uk, Badu, Samuel¹ (AUTHOR) samuel.badu@liverpoolftt.nhs.uk, Toh, Cheng-Hock^1,2 (AUTHOR) toh@liverpool.ac.uk, Dutt, Tina¹ (AUTHOR) tina.dutt@liverpoolft.nhs.uk

Publikováno v: Journal of Clinical Medicine. Sep2024, Vol. 13 Issue 17, p5152. 12p.

Apadamtase Alfa: First Approval.

Autor: Heo YA; Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Publikováno v: Drugs [Drugs] 2024 Apr; Vol. 84 (4), pp. 467-472.