Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study

被引：10
|
作者：
Pipe, Steven W. [1 ]
Collins, Peter [2 ]
Dhalluin, Christophe [3 ]
Kenet, Gili [4 ,5 ]
Schmitt, Christophe [3 ]
Buri, Muriel [3 ]
Jimenez-Yuste, Victor [6 ]
Peyvandi, Flora [7 ,8 ,9 ]
Young, Guy [10 ]
Oldenburg, Johannes [11 ]
Mancuso, Maria Elisa [12 ]
Kiialainen, Anna [3 ]
Chang, Tiffany [13 ]
Lehle, Michaela [3 ]
Fijnvandraat, Karin [14 ]
机构：
[1] Univ Michigan, Ann Arbor, MI 48109 USA
[2] Cardiff Univ, Sch Med, Cardiff, Wales
[3] F Hoffmann La Roche Ltd, Basel, Switzerland
[4] Sheba Med Ctr, Ramat Gan, Israel
[5] Tel Aviv Univ, Tel Aviv, Israel
[6] Univ Autonoma Madrid, La Paz Univ Hosp, Madrid, Spain
[7] Fdn IRCCS CaGranda Osped Maggiore Policlin, Angelo Bianchi Bon Hemophilia, Milan, Italy
[8] Thrombosis Ctr, Milan, Italy
[9] Univ Milan, Milan, Italy
[10] Childrens Hosp Los Angeles, Los Angeles, CA 90027 USA
[11] Univ Bonn, Bonn, Germany
[12] IRCCS Humanitas Res Hosp, Rozzano, Italy
[13] Spark Therapeut Inc, San Francisco, CA USA
[14] Univ Amsterdam, Amsterdam, Netherlands
来源：
BLOOD | 2022年 / 140卷
关键词：
D O I：
10.1182/blood-2022-157264
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页数：3
相关论文
共 50 条
  • [31] Longitudinal dynamic changes in factor VIII inhibitor titers in patients with hemophilia A and inhibitors receiving emicizumab prophylaxis
    Naruto Shimonishi
    Kana Sasai
    Kenichi Ogiwara
    Shoko Furukawa
    Yuto Nakajima
    Kuniyoshi Mizumachi
    Koji Yada
    Masahiro Takeyama
    Midori Shima
    Narumi Mizuno
    Keiji Nogami
    [J]. International Journal of Hematology, 2023, 118 : 690 - 698
  • [32] Emicizumab prophylaxis and health-related outcomes in persons with hemophilia a (PwHA) with inhibitors: HAVEN 1 study
    Oldenburg, J.
    Mahlangu, J. N.
    Bujan, W.
    Trask, P. C.
    von Mackensen, S.
    Callaghan, M. U.
    Young, G.
    Asikanius, E.
    Peyvandi, F.
    Santagostino, E.
    Kruse-Jarres, R.
    Negrier, C.
    Kessler, C.
    Levy, G. G.
    Windyga, J.
    Shima, M.
    [J]. HAEMOPHILIA, 2018, 24 : 89 - 90
  • [33] Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and results from the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with haemophilia A without FVIII inhibitors
    Kempton, Christine
    Trask, Peter
    Parnes, Aric
    Niggli, Markus
    Campinha-Bacote, Avrita
    Callaghan, Michael U.
    O'Connell, Niamh
    Paz-Priel, Ido
    Mahlangu, Johnny N.
    [J]. HAEMOPHILIA, 2021, 27 (02) : 221 - 228
  • [34] CHANGES IN INHIBITORS AFTER RE-EXPOSURE TO FACTOR VIII CONCENTRATE DURING EMICIZUMAB PROPHYLAXIS IN CHILDREN WITH HEMOPHILIA A WITH INHIBITORS
    Kodera, Asami
    Sato, Atsushi
    Hayasaka, Hiroe
    Ogawa, Maki
    Nanjo, Yuka
    Suzuki, Tasuku
    Suzuki, Nobu
    Onuma, Masaei
    Rikiishi, Takeshi
    Imaizumi, Masue
    [J]. PEDIATRIC BLOOD & CANCER, 2024, 71 : S101 - S102
  • [35] Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study
    Feldman, BM
    Pai, M
    Rivard, GE
    Israels, S
    Poon, MC
    Demers, C
    Robinson, S
    Luke, KH
    Wu, JKM
    Gill, K
    Lillicrap, D
    Babyn, P
    McLimont, M
    Blanchette, VS
    [J]. JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2006, 4 (06) : 1228 - 1236
  • [36] Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors-a single-center cohort
    Barg, Assaf A.
    Avishai, Einat
    Budnik, Ivan
    Levy-Mendelovich, Sarina
    Barazani, Tami B.
    Kenet, Gili
    Livnat, Tami
    [J]. PEDIATRIC BLOOD & CANCER, 2019, 66 (11)
  • [37] The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies
    Skinner, Mark W.
    Negrier, Claude
    Paz-Priel, Ido
    Chebon, Sammy
    Jimenez-Yuste, Victor
    Callaghan, Michael U.
    Lehle, Michaela
    Niggli, Markus
    Mahlangu, Johnny
    Shapiro, Amy
    Shima, Midori
    Campinha-Bacote, Avrita
    Levy, Gallia G.
    Oldenburg, Johannes
    von Mackensen, Sylvia
    Pipe, Steven W.
    [J]. HAEMOPHILIA, 2021, 27 (05) : 854 - 865
  • [38] Rescue factor VIII replacement to secure hemostasis in a patient with hemophilia A and inhibitors on emicizumab prophylaxis undergoing hip replacement
    Santagostino, Elena
    Elisa Mancuso, Maria
    Novembrino, Cristina
    Solimeno, Luigi Piero
    Tripodi, Armando
    Peyvandi, Flora
    [J]. HAEMATOLOGICA, 2019, 104 (08) : E380 - E382
  • [39] RESOURCE CONSUMPTION ANALYSIS IN PATIENTS WITH HEMOPHILIA A WITHOUT INHIBITORS ON PROPHYLAXIS WITH FVIII BEFORE SWITCHING TO EMICIZUMAB
    Giacomini, E.
    Leogrande, M.
    Motta, L.
    Tempre, R.
    Bendinelli, S.
    Degli Esposti, L.
    [J]. VALUE IN HEALTH, 2023, 26 (12) : S69 - S69
  • [40] ELIMINATION OF FACTOR-VIII INHIBITORS IN 7 CHILDREN WITH SEVERE HEMOPHILIA-A
    KREUZ, W
    SCHARRER, I
    [J]. HAEMOSTASIS, 1985, 15 (01) : 20 - 20
12345