Literature DB >> 31846611

Factor VIII replacement is still the standard of care in haemophilia A.

Louis Aledort1, Pier Mannuccio Mannucci2, Wolfgang Schramm3, Michael Tarantino4.   

Abstract

Prophylactic factor VIII (FVIII) has dramatically improved haemophilia A treatment, preventing joint bleeding and halting the deterioration of joint status. FVIII products with an extended plasma half-life further improve patients' quality of life and increase therapeutic adherence. New licensed classes of non-replacement products include prophylactic emicizumab, which is administered subcutaneously up to every 4 weeks. However, this drug is not suitable for acute bleeding episodes or management of major surgery, and long-term data on the impact of emicizumab on joint health, FVIII inhibitor development and thrombotic risk are awaited. Prophylaxis with FVIII replacement remains the standard of care in haemophilia A, with the aim of achieving a level of haemostasis control that allows patients to meet their lifestyle goals.

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Year:  2019        PMID： 31846611      PMCID： PMC6917528          DOI： 10.2450/2019.0211-19

Source DB:  PubMed          Journal:  Blood Transfus        ISSN： 1723-2007            Impact factor:   3.443


  51 in total

Review 1.  Prophylactic factor replacement in hemophilia.

Authors:  Manuel D Carcao; Louis Aledort
Journal:  Blood Rev       Date:  2004-06       Impact factor: 8.250

Review 2.  Health-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trials.

Authors:  A O Oladapo; J D Epstein; E Williams; D Ito; A Gringeri; L A Valentino
Journal:  Haemophilia       Date:  2015-07-17       Impact factor: 4.287

Review 3.  Using pharmacokinetics to individualize hemophilia therapy.

Authors:  Alfonso Iorio
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2017-12-08

4.  Emicizumab Prophylaxis in Hemophilia A with Inhibitors.

Authors:  Johannes Oldenburg; Johnny N Mahlangu; Benjamin Kim; Christophe Schmitt; Michael U Callaghan; Guy Young; Elena Santagostino; Rebecca Kruse-Jarres; Claude Negrier; Craig Kessler; Nancy Valente; Elina Asikanius; Gallia G Levy; Jerzy Windyga; Midori Shima
Journal:  N Engl J Med       Date:  2017-07-10       Impact factor: 91.245

5.  Safety and efficacy of a glycoPEGylated rFVIII (turoctocog alpha pegol, N8-GP) in paediatric patients with severe haemophilia A.

Authors:  Sandrine Meunier; Jayanthi Alamelu; Silke Ehrenforth; Hideji Hanabusa; Faraizah Abdul Karim; Kaan Kavakli; Melanie Khodaie; Janice Staber; Oleksandra Stasyshyn; Donald L Yee; Lina Rageliene
Journal:  Thromb Haemost       Date:  2017-07-06       Impact factor: 5.249

6.  Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment.

Authors:  Nadine G Andersson; Günter Auerswald; Chris Barnes; Manuel Carcao; Amy L Dunn; Karin Fijnvandraat; Marianne Hoffmann; Kaan Kavakli; Gili Kenet; Rainer Kobelt; Karin Kurnik; Ri Liesner; Anne Mäkipernaa; Marilyn J Manco-Johnson; Maria E Mancuso; Angelo C Molinari; Beatrice Nolan; Rosario Perez Garrido; Pia Petrini; Helen E Platokouki; Amy D Shapiro; Runhui Wu; Rolf Ljung
Journal:  Br J Haematol       Date:  2017-07-12       Impact factor: 6.998

7.  Clinical evaluation of glycoPEGylated recombinant FVIII: Efficacy and safety in severe haemophilia A.

Authors:  Paul Giangrande; Tatiana Andreeva; Pratima Chowdary; Silke Ehrenforth; Hideji Hanabusa; Frank W G Leebeek; Steven R Lentz; Laszlo Nemes; Lone Hvitfeldt Poulsen; Elena Santagostino; Chur Woo You; Wan Hui Ong Clausen; Peter G Jönsson; Johannes Oldenburg
Journal:  Thromb Haemost       Date:  2016-12-01       Impact factor: 5.249

8.  Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A: Results of the PROTECT VIII clinical trial.

Authors:  Elena Santagostino; Shadan Lalezari; Mark T Reding; Jonathan Ducore; Heng Joo Ng; Lone H Poulsen; Lisa A Michaels; Camila C G Linardi
Journal:  Thromb Res       Date:  2019-08-26       Impact factor: 3.944

9.  International workshop on immune tolerance induction: consensus recommendations.

Authors:  D M DiMichele; W K Hoots; S W Pipe; G E Rivard; E Santagostino
Journal:  Haemophilia       Date:  2007-07       Impact factor: 4.287

10.  Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A.

Authors:  T E Coyle; M T Reding; J C Lin; L A Michaels; A Shah; J Powell
Journal:  J Thromb Haemost       Date:  2014-04       Impact factor: 5.824
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  22 in total

Review 1.  Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates?

Authors:  Patrick Ellsworth; Alice Ma
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2021-12-10

2.  Hidden blood loss of total knee arthroplasty in hemophilia arthritis: an analysis of influencing factors.

Authors:  Shao Ning Shen; Dong Xiao Wu; Shuai Jie Lv; Pei Jian Tong
Journal:  BMC Musculoskelet Disord       Date:  2022-06-17       Impact factor: 2.562

Review 3.  The neurosurgical management of Severe Hemophilia A and Moyamoya (SHAM): challenges, strategies, and literature review.

Authors:  Risheng Xu; Anita L Kalluri; Lisa R Sun; Courtney E Lawrence; Jennifer K Lee; Sujatha Kannan; Alan R Cohen
Journal:  Childs Nerv Syst       Date:  2022-03-09       Impact factor: 1.532

4.  Efficacy of rFVIIIFc versus Emicizumab for the Treatment of Patients with Hemophilia A without Inhibitors: Matching-Adjusted Indirect Comparison of A-LONG and HAVEN Trials.

Authors:  Robert Klamroth; Piotr Wojciechowski; Samuel Aballéa; Françoise Diamand; Zalmai Hakimi; Jameel Nazir; Lydia Abad-Franch; Stefan Lethagen; Elena Santagostino; Michael D Tarantino
Journal:  J Blood Med       Date:  2021-02-25

5.  Nonhuman glycans can regulate anti-factor VIII antibody formation in mice.

Authors:  Connie M Arthur; Patricia E Zerra; Sooncheon Shin; Jianmei Wang; Xeuzheng Song; Christopher B Doering; Pete Lollar; Shannon Meeks; Sean R Stowell
Journal:  Blood       Date:  2022-03-03       Impact factor: 25.476

6.  Monitoring of different factor VIII replacement products using a factor VIII one-stage clotting assay on cobas t 511/711 analysers.

Authors:  Carolin Ketteler; Ingrid Hoffmann; Simon Davidson; Andreas Tiede; Nina Richter
Journal:  Haemophilia       Date:  2021-09-30       Impact factor: 4.263

7.  Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94-9027 in severe haemophilia A: final results of the PROTECT VIII extension study.

Authors:  Mark T Reding; Ingrid Pabinger; Pal Andre Holme; Lone Poulsen; Claude Negrier; Pavani Chalasani; Monika Maas Enriquez; Maria Wang; Karina Meijer; Maria Elisa Mancuso; Shadan Lalezari
Journal:  Haemophilia       Date:  2021-04-05       Impact factor: 4.263

8.  Fc Gamma Receptors and Complement Component 3 Facilitate Anti-fVIII Antibody Formation.

Authors:  Patricia E Zerra; Connie M Arthur; Satheesh Chonat; Cheryl L Maier; Amanda Mener; Sooncheon Shin; Jerry William L Allen; W Hunter Baldwin; Courtney Cox; Hans Verkerke; Ryan P Jajosky; Christopher A Tormey; Shannon L Meeks; Sean R Stowell
Journal:  Front Immunol       Date:  2020-06-09       Impact factor: 7.561

Review 9.  Bridging the Missing Link with Emicizumab: A Bispecific Antibody for Treatment of Hemophilia A.

Authors:  Georg Gelbenegger; Christian Schoergenhofer; Paul Knoebl; Bernd Jilma
Journal:  Thromb Haemost       Date:  2020-07-27       Impact factor: 5.249

Review 10.  Hemophilia therapy: the future has begun.

Authors:  Pier Mannuccio Mannucci
Journal:  Haematologica       Date:  2020-02-14       Impact factor: 9.941
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