Literature DB >> 26728503

Achievements, challenges and unmet needs for haemophilia patients with inhibitors: Report from a symposium in Paris, France on 20 November 2014.

Y Dargaud1, A Pavlova2, S Lacroix-Desmazes3, K Fischer4, M Soucie5, S Claeyssens6, D W Scott7, R d'Oiron8, G Lavigne-Lissalde9, G Kenet10, C Escuriola Ettingshausen11, A Borel-Derlon12, T Lambert13, G Pasta14, C Négrier15.   

Abstract

Over the past 20 years, there have been many advances in haemophilia treatment that have allowed patients to take greater control of their disease. However, the development of factor VIII (FVIII) inhibitors is the greatest complication of the disease and a challenge in the treatment of haemophilia making management of bleeding episodes difficult and surgical procedures very challenging. A meeting to discuss the unmet needs of haemophilia patients with inhibitors was held in Paris on 20 November 2014. Topics discussed were genetic and non-genetic risk factors for the development of inhibitors, immunological aspects of inhibitor development, FVIII products and inhibitor development, generation and functional properties of engineered antigen-specific T regulatory cells, suppression of immune responses to FVIII, prophylaxis in haemophilia patients with inhibitors, epitope mapping of FVIII inhibitors, current controversies in immune tolerance induction therapy, surgery in haemophilia patients with inhibitors and future perspectives for the treatment of haemophilia patients with inhibitors. A summary of the key points discussed is presented in this paper.
© 2016 John Wiley & Sons Ltd.

Entities:  

Keywords:  FVIII products; genetics; haemophilia; inhibitors; prophylaxis; surgery

Mesh:

Substances:

Year:  2016        PMID: 26728503      PMCID: PMC4989271          DOI: 10.1111/hae.12860

Source DB:  PubMed          Journal:  Haemophilia        ISSN: 1351-8216            Impact factor:   4.287


  129 in total

1.  Elective orthopaedic surgery for inhibitor patients.

Authors:  E C Rodriguez-Merchan; J D Wiedel; T Wallny; I Hvid; E Berntorp; G-E Rivard; N J Goddard; F Querol; H Caviglia
Journal:  Haemophilia       Date:  2003-09       Impact factor: 4.287

Review 2.  Identifying and managing inhibitor patients requiring orthopaedic surgery - the multidisciplinary team approach.

Authors:  C Ludlam
Journal:  Haemophilia       Date:  2005-11       Impact factor: 4.287

3.  Combined administration of FVIII and rFVIIa improves haemostasis in haemophilia A patients with high-responding inhibitors--a thrombin generation-guided pilot study.

Authors:  T Livnat; U Martinowitz; S Azar-Avivi; A Zivelin; T Brutman-Barazani; A Lubetsky; G Kenet
Journal:  Haemophilia       Date:  2013-05-10       Impact factor: 4.287

Review 4.  Novel products for haemostasis.

Authors:  M Shima; C Hermans; P de Moerloose
Journal:  Haemophilia       Date:  2014-05       Impact factor: 4.287

5.  Combined treatment with APCC (FEIBA®) and tranexamic acid in patients with haemophilia A with inhibitors and in patients with acquired haemophilia A--a two-centre experience.

Authors:  M Holmström; H T T Tran; P A Holme
Journal:  Haemophilia       Date:  2012-02-20       Impact factor: 4.287

6.  A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study.

Authors:  Jan Astermark; Sharyne M Donfield; Donna M DiMichele; Alessandro Gringeri; Steven A Gilbert; Jennifer Waters; Erik Berntorp
Journal:  Blood       Date:  2006-09-21       Impact factor: 22.113

7.  Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors.

Authors:  Cindy Leissinger; Alessandro Gringeri; Bülent Antmen; Erik Berntorp; Chiara Biasoli; Shannon Carpenter; Paolo Cortesi; Hyejin Jo; Kaan Kavakli; Riitta Lassila; Massimo Morfini; Claude Négrier; Angiola Rocino; Wolfgang Schramm; Margit Serban; Marusia Valentina Uscatescu; Jerzy Windyga; Bülent Zülfikar; Lorenzo Mantovani
Journal:  N Engl J Med       Date:  2011-11-03       Impact factor: 91.245

8.  HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII.

Authors:  J Oldenburg; J K Picard; R Schwaab; H H Brackmann; E G Tuddenham; E Simpson
Journal:  Thromb Haemost       Date:  1997-02       Impact factor: 5.249

9.  Consensus protocol for the use of recombinant activated factor VII [eptacog alfa (activated); NovoSeven] in elective orthopaedic surgery in haemophilic patients with inhibitors.

Authors:  P L F Giangrande; J T Wilde; B Madan; C A Ludlam; E G D Tuddenham; N J Goddard; G Dolan; J Ingerslev
Journal:  Haemophilia       Date:  2009-02-01       Impact factor: 4.287

10.  Clinical grade manufacturing of human alloantigen-reactive regulatory T cells for use in transplantation.

Authors:  A L Putnam; N Safinia; A Medvec; M Laszkowska; M Wray; M A Mintz; E Trotta; G L Szot; W Liu; A Lares; K Lee; A Laing; R I Lechler; J L Riley; J A Bluestone; G Lombardi; Q Tang
Journal:  Am J Transplant       Date:  2013-09-18       Impact factor: 8.086

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  3 in total

Review 1.  Advances and innovations in haemophilia treatment.

Authors:  Rob Peters; Tim Harris
Journal:  Nat Rev Drug Discov       Date:  2018-06-08       Impact factor: 84.694

2.  Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice.

Authors:  Weiqing Jing; Juan Chen; Yuanhua Cai; Yingyu Chen; Jocelyn A Schroeder; Bryon D Johnson; Weiguo Cui; Qizhen Shi
Journal:  Blood Adv       Date:  2019-10-22

3.  MG1113, a specific anti-tissue factor pathway inhibitor antibody, rebalances the coagulation system and promotes hemostasis in hemophilia.

Authors:  Heechun Kwak; Sumin Lee; Seunghyun Jo; Young Eun Kwon; Hyunju Kang; Gahee Choi; Myung Eun Jung; Mi-Jeong Kwak; Seonghoon Kim; Byung-Ha Oh; Dong-Sik Kim; Sung Ho Hwang
Journal:  Res Pract Thromb Haemost       Date:  2020-10-22
  3 in total

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