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Emicizumab Prophylaxis in Hemophilia A with Inhibitors.
Oldenburg, Johannes; Mahlangu, Johnny N; Kim, Benjamin; Schmitt, Christophe; Callaghan, Michael U; Young, Guy; Santagostino, Elena; Kruse-Jarres, Rebecca; Negrier, Claude; Kessler, Craig; Valente, Nancy; Asikanius, Elina; Levy, Gallia G; Windyga, Jerzy; Shima, Midori.
Afiliação
  • Oldenburg J; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Mahlangu JN; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Kim B; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Schmitt C; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Callaghan MU; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Young G; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Santagostino E; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Kruse-Jarres R; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Negrier C; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Kessler C; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Valente N; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Asikanius E; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Levy GG; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Windyga J; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
  • Shima M; From Universitätsklinikum Bonn, Bonn, Germany (J.O.); the Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg (J.N.M.); Genentech, South San Francisco (B.K., N.V., G.G.L.), and Children's Hospital Los
N Engl J Med ; 377(9): 809-818, 2017 08 31.
Article em En | MEDLINE | ID: mdl-28691557
ABSTRACT

BACKGROUND:

Emicizumab (ACE910) bridges activated factor IX and factor X to restore the function of activated factor VIII, which is deficient in persons with hemophilia A. This phase 3, multicenter trial assessed once-weekly subcutaneous emicizumab prophylaxis in persons with hemophilia A with factor VIII inhibitors.

METHODS:

We enrolled participants who were 12 years of age or older. Those who had previously received episodic treatment with bypassing agents were randomly assigned in a 21 ratio to emicizumab prophylaxis (group A) or no prophylaxis (group B). The primary end point was the difference in bleeding rates between group A and group B. Participants who had previously received prophylactic treatment with bypassing agents received emicizumab prophylaxis in group C.

RESULTS:

A total of 109 male participants with hemophilia A with inhibitors were enrolled. The annualized bleeding rate was 2.9 events (95% confidence interval [CI], 1.7 to 5.0) among participants who were randomly assigned to emicizumab prophylaxis (group A, 35 participants) versus 23.3 events (95% CI, 12.3 to 43.9) among those assigned to no prophylaxis (group B, 18 participants), representing a significant difference of 87% in favor of emicizumab prophylaxis (P<0.001). A total of 22 participants in group A (63%) had zero bleeding events, as compared with 1 participant (6%) in group B. Among 24 participants in group C who had participated in a noninterventional study, emicizumab prophylaxis resulted in a bleeding rate that was significantly lower by 79% than the rate with previous bypassing-agent prophylaxis (P<0.001). Overall, 198 adverse events were reported in 103 participants receiving emicizumab prophylaxis; the most frequent events were injection-site reactions (in 15% of participants). Thrombotic microangiopathy and thrombosis were reported in 2 participants each (in the primary analysis) who had received multiple infusions of activated prothrombin complex concentrate for breakthrough bleeding. No antidrug antibodies were detected.

CONCLUSIONS:

Emicizumab prophylaxis was associated with a significantly lower rate of bleeding events than no prophylaxis among participants with hemophilia A with inhibitors. (Funded by F. Hoffmann-La Roche and Chugai Pharmaceutical; HAVEN 1 ClinicalTrials.gov number, NCT02622321 .).
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Anticorpos Biespecíficos / Anticorpos Monoclonais Humanizados / Hemofilia A / Hemorragia Limite: Adolescent / Adult / Aged / Child / Humans / Male / Middle aged Idioma: En Ano de publicação: 2017
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Anticorpos Biespecíficos / Anticorpos Monoclonais Humanizados / Hemofilia A / Hemorragia Limite: Adolescent / Adult / Aged / Child / Humans / Male / Middle aged Idioma: En Ano de publicação: 2017