Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors
Johnny Mahlangu,Johannes Oldenburg,Ido Paz-Priel,Claude Negrier,Markus Niggli,M Elisa Mancuso,M Elisa Mancuso,Christophe Schmitt,Víctor Jiménez-Yuste,Christine L. Kempton,Christophe Dhalluin,Michael U. Callaghan,Willem Bujan,Midori Shima,Joanne I. Adamkewicz,Elina Asikanius,Gallia G. Levy,Rebecca Kruse-Jarres +17 more
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TLDR
Empicizumab prophylaxis administered subcutaneously once weekly or every 2 weeks led to a significantly lower bleeding rate than no proPHylaxis among persons with hemophilia A without inhibitors; more than half of the participants who received prophYLaxis had no treated bleeding events.Abstract:
Background Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostas...read more
Citations
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Journal ArticleDOI
WFH Guidelines for the Management of Hemophilia, 3rd edition
Alok Srivastava,Elena Santagostino,Alison Dougall,Steve Kitchen,Megan Sutherland,Steven W. Pipe,Manuel Carcao,Johnny Mahlangu,Margaret V. Ragni,Jerzy Windyga,Adolfo Llinás,Nicholas J. Goddard,Richa Mohan,Pradeep M. Poonnoose,Brian M. Feldman,Sandra Zelman Lewis,H. Marijke van den Berg,Glenn F. Pierce +17 more
TL;DR: The WFH Guidelines for the Management of Hemophilia panelists and co-authors thank the panelists for their time and share their views on how to better understand and treat hemophilia.
Journal ArticleDOI
Bispecific antibodies: a mechanistic review of the pipeline.
TL;DR: The current bispecific antibody landscape is reviewed from a mechanistic perspective, including a comprehensive overview of the pipeline, to have the potential for novel functionalities that are not provided by mixtures of monoclonal antibodies.
Book ChapterDOI
Institute for Clinical and Economic Review
TL;DR: Changes include separation of budget impact analysis from value determinations, introduction of other value measures beyond the QALY, increasing consideration of contextual elements, and adoption of a “societal perspective” where data support it.
Journal ArticleDOI
Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study
Steven W. Pipe,Midori Shima,Michaela Lehle,Amy D. Shapiro,Sammy Chebon,Katsuyuki Fukutake,Nigel S. Key,Agnès Portron,Christophe Schmitt,Maria Podolak-Dawidziak,Nives Selak Bienz,Cédric Hermans,Avrita Campinha-Bacote,Anna Kiialainen,Kathelijne Peerlinck,Gallia G. Levy,Víctor Jiménez-Yuste +16 more
TL;DR: Emicizumab given once every 4 weeks showed clinically meaningful bleed control while being well tolerated, and could improve patient care by decreasing treatment burden and increasing adherence to effective prophylaxis, potentially decreasing the development of secondary complications for people with haemophilia A.
Journal ArticleDOI
A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.
Guy Young,Ri Liesner,Tiffany Chang,Robert F. Sidonio,Johannes Oldenburg,Víctor Jiménez-Yuste,Johnny Mahlangu,Rebecca Kruse-Jarres,Michael Wang,Marianne Uguen,Michelle Y. Doral,Lilyan Wright,Christophe Schmitt,Gallia G. Levy,Midori Shima,Maria Elisa Mancuso +15 more
TL;DR: Emicizumab prophylaxis has been shown to be a highly effective novel medication for children with hemophilia A and inhibitors and two of 88 participants developed antidrug antibodies (ADAs) with neutralizing potential, ie, associated with decreased emicIZumab plasma concentrations.
References
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Journal ArticleDOI
The hemophilias--from royal genes to gene therapy.
TL;DR: Of the various types of hemophilia, the most common of these lifelong bleeding disorders are due to an inherited deficiency of factor VIII or factor IX (Table 1).
Journal ArticleDOI
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
Lindsey A. George,Spencer K. Sullivan,Adam Giermasz,John E.J. Rasko,John E.J. Rasko,Benjamin J. Samelson-Jones,Jonathan M. Ducore,Adam Cuker,Lisa M. Sullivan,Suvankar Majumdar,Jerome M. Teitel,Catherine E. McGuinn,Margaret V. Ragni,Alvin Luk,Daniel Hui,J. Fraser Wright,Yifeng Chen,Yun Liu,Katie Wachtel,Angela Winters,Stefan Tiefenbacher,Valder R. Arruda,Johannes C.M. Van der Loo,Olga Zelenaia,Daniel Takefman,Marcus E. Carr,Linda B. Couto,Xavier M. Anguela,Katherine A. High +28 more
TL;DR: Transgene‐derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use in 10 participants with hemophilia who received the same vectors.
Journal ArticleDOI
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Savita Rangarajan,Liron Walsh,Will Lester,David Perry,Bella Madan,Michael Laffan,Hua Yu,Christian Vettermann,Glenn F. Pierce,Wing Yen Wong,K. John Pasi +10 more
TL;DR: The infusion of AAV5‐hFVIII‐SQ was associated with the sustained normalization of factor VIII activity level over a period of 1 year in six of seven participants who received a high dose, with stabilization of hemostasis and a profound reduction in factor VIII use in all seven participants.
Journal ArticleDOI
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
Takehisa Kitazawa,Tomoyuki Igawa,Zenjiro Sampei,Atsushi Muto,Tetsuo Kojima,Tetsuhiro Soeda,Kazutaka Yoshihashi,Yukiko Okuyama-Nishida,Hiroyuki Saito,Hiroyuki Tsunoda,Tsukasa Suzuki,Hideki Adachi,Taro Miyazaki,Shinya Ishii,Mika Kamata-Sakurai,Iida Takeo,Aya Harada,Keiko Esaki,Miho Funaki,Chifumi Moriyama,Eriko Tanaka,Yasufumi Kikuchi,Tetsuya Wakabayashi,Manabu Wada,Masaaki Goto,Takeshi Toyoda,Atsunori Ueyama,Sachiyo Suzuki,Kenta Haraya,Tatsuhiko Tachibana,Yoshiki Kawabe,Midori Shima,Akira Yoshioka,Kunihiro Hattori +33 more
TL;DR: A humanized bispecific antibody to factor IXa (FIXa) and factor X (FX), termed hBS23, that places these two factors into spatially appropriate positions and mimics the cofactor function of FVIII, exerted coagulation activity in FV III-deficient plasma, even in the presence of inhibitors.
Journal ArticleDOI
Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
Midori Shima,Hideji Hanabusa,Masashi Taki,Tadashi Matsushita,Tetsuji Sato,Katsuyuki Fukutake,Naoki Fukazawa,Koichiro Yoneyama,Hiroki Yoshida,Keiji Nogami +9 more
TL;DR: Once-weekly subcutaneous administration of emicizumab markedly decreased the bleeding rate in patients who had hemophilia A with or without factor VIII inhibitors, associated with neither serious adverse events nor clinically relevant coagulation abnormalities.
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Emicizumab Prophylaxis in Hemophilia A with Inhibitors
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