Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A.

Autor: Malec L; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Peyvandi F; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Chan AKC; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Königs C; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Zulfikar B; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Yuan H; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Simpson M; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Álvarez Román MT; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Carcao M; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Staber JM; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Dunn AL; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Chou SC; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., d'Oiron R; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Albisetti M; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Demissie M; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Santagostino E; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Yarramaneni A; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Wong N; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Abad-Franch L; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Gunawardena S; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.)., Fijnvandraat K; From Versiti Blood Research Institute, and the Division of Hematology and Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin - both in Milwaukee (L.M.); IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); McMaster Children's Hospital, McMaster University, Hamilton, ON (A.K.C.C.), and the Division of Hematology-Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto (M.C.) - both in Canada; Goethe University Frankfurt, University Hospital, Department of Pediatrics and Adolescent Medicine, Frankfurt, Germany (C.K.); the Department of Pediatric Hematology, Istanbul University Oncology Institute, Inherited Bleeding Disorders, Istanbul, Turkey (B.Z.); Sanofi, Cambridge, MA (H.Y., M.D.); Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago (M.S.); Hospital Universitario La Paz, Autonoma University of Madrid, IdiPAZ, Madrid (M.T.Á.R.); University of Iowa Stead Family Children's Hospital, Carver College of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Iowa City (J.M.S.); the Division of Hematology, Oncology, and Blood and Marrow Transplant at Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (S.-C.C.); Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles and Hémostase Inflammation Thrombose, Unité Mixte de Recherche S1176, INSERM, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Le Kremlin-Bicêtre, France (R.O.); University Children's Hospital, Zurich (M.A.), and Sobi, Basel (E.S., L.A.-F.) - both in Switzerland; Sanofi, Bridgewater, NJ (A.Y., N.W., S.G.); and Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam (K.F.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2024 Jul 18; Vol. 391 (3), pp. 235-246.

Timing and severity of inhibitor development in recombinant versus plasma‐derived factor VIII concentrates: a SIPPET analysis

Autor: Peyvandi, F. ^*, Cannavò, A., Garagiola, I., Palla, R., Mannucci, P.M., Rosendaal, F.R., El‐Beshlawy, A., Elalfy, M., Ramanan, V., Eshghi, P., Hanagavadi, S., Varadarajan, R., Karimi, M., Manglani, M.V., Ross, C., Young, G., Seth, T., Apte, S., Nayak, D.M., Santagostino, E., Elisa Mancuso, M., Sandoval Gonzalez, A.C., Mahlangu, J.N., Bonanad Boix, S., Cerqueira, M., Ewing, N.P., Male, C., Owaidah, T., Soto Arellano, V., Kobrinsky, N.L., Majumdar, S., Perez Garrido, R., Sachdeva, A., Simpson, M., Thomas, M., Zanon, E., Antmen, B., Kavakl, K., Manco‐Johnson, M.J., Martinez, M., Marzouka, E., Mazzucconi, M.G., Neme, D., Palomo Bravo, A., Paredes Aguilera, R., Prezotti, A., Schmitt, K., Wicklund, B.M., Zulfikar, B.

Publikováno v: In Journal of Thrombosis and Haemostasis January 2018 16(1):39-43

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial.

Autor: Kenet G; The National Hemophilia Centre, Amalia Biron Thrombosis Research Institute, Sheba Medical Centre, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel., Nolan B; Department of Hematology, Children's Health Ireland at Crumlin, Dublin, Ireland., Zulfikar B; Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey., Antmen B; Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation Unit, Faculty of Medicine, Acibadem University, Adana Hospital, Adana, Turkey., Kampmann P; Department of Hematology, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark., Matsushita T; Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan., You CW; Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea., Vilchevska K; Department of Hematology, Ohmatdyt National Children's Specialized Hospital, Kyiv, Ukraine., Bagot CN; Department of Haematology, Glasgow Royal Infirmary, Glasgow, United Kingdom., Sharif A; Hospital Sultanah Aminah, Johor Bahru, Malaysia., 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., Young G; Hemostasis and Thrombosis Center, Cancer and Blood Diseases Institute, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA., Negrier C; UR4609 Hemostasis and Thrombosis, Claude Bernard University Lyon 1, Lyon, France., Chi J; Sanofi, Bridgewater, NJ., Kittner B; Sanofi, Bridgewater, NJ., Sussebach C; Sanofi, Frankfurt, Germany., Shammas F; Sanofi, Montreal, Quebec, Canada., Mei B; Sanofi, Cambridge, MA., Andersson S; Sanofi, Cambridge, MA., Kavakli K; Department of Haematology, Ege University Faculty of Medicine, Children's Hospital, Izmir, Turkey.

Publikováno v: Blood [Blood] 2024 May 30; Vol. 143 (22), pp. 2256-2269.

CHILDHOOD EWING SARCOMA FAMILY TUMORS: 25-YEAR EXPERIENCE OF A SINGLE CENTER IN TURKEY

Autor: Kebudi, R., Gorgun, O., ÇAKIR, FATMA BETÜL, Agaoglu, F. Yaman, Bay, S. Buyukkapu, Koc, B., Zulfikar, B., Ozger, H., Ayan, I., Darendeliler, E.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______9651::54c44407a456c4df7c254ef8836cce09
https://hdl.handle.net/20.500.12645/16322

LB 01.1 Efanesoctocog Alfa Prophylaxis for Previously Treated Patients

Autor: Malec, L., Peyvandi, F., Chan, A., Koenigs, C., Zulfikar, B., Yuan, H., Simpson, M., Alvarez-Román, M., Carcao, M., Staber, J., Dunn, A., Chou, S., D’Oiron, R., Albisetti, M., Demissie, M., Santagostino, E., Yarramaneni, A., Abad-Franch, L., Gunawardena, S., Fijnvandraat, K.

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