Haemophilia B: Where are we now and what does the future hold?
Dolan G, Benson G, Duffy A, Hermans C, Jiménez-Yuste V, Lambert T, Ljung R, Morfini M, Zupančić Šalek S.
Blood Rev. 2018 Jan;32(1):52-60. doi: 10.1016/j.blre.2017.08.007. Epub 2017 Aug 16.
PMID:28826659
Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial.
Young G, Srivastava A, Kavakli K, Ross C, Sathar J, You CW, Tran H, Sun J, Wu R, Poloskey S, Qiu Z, Kichou S, Andersson S, Mei B, Rangarajan S.
Lancet. 2023 Apr 29;401(10386):1427-1437. doi: 10.1016/S0140-6736(23)00284-2. Epub 2023 Mar 29.
PMID:37003287
Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial.
Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, You CW, Xu W, Malan N, Frenzel L, Bagot CN, Stasyshyn O, Chang CY, Poloskey S, Qiu Z, Andersson S, Mei B, Pipe SW.
Lancet Haematol. 2023 May;10(5):e322-e332. doi: 10.1016/S2352-3026(23)00037-6. Epub 2023 Mar 29.
PMID:37003278
Haemophilia.
Chowdary P, Carcao M, Kenet G, Pipe SW.
Lancet. 2025 Mar 1;405(10480):736-750. doi: 10.1016/S0140-6736(24)02139-1.
PMID:40023652
Haemophilias A and B.
Bolton-Maggs PH, Pasi KJ.
Lancet. 2003 May 24;361(9371):1801-9. doi: 10.1016/S0140-6736(03)13405-8.
PMID:12781551
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators.
Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1.
PMID:38437857
Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial.