The past and future of haemophilia: diagnosis, treatments, and its complications

F Peyvandi, I Garagiola, G Young - The Lancet, 2016 - thelancet.com
Haemophilia A and B are hereditary haemorrhagic disorders characterised by deficiency or
dysfunction of coagulation protein factors VIII and IX, respectively. Recurrent joint and …

Risk factors for inhibitor development in severe hemophilia A

I Garagiola, R Palla, F Peyvandi - Thrombosis research, 2018 - Elsevier
Although significant advances in hemophilia treatment have improved patient outcomes and
quality of life, one of the greatest complications in severe hemophilia A is the development of …

A randomized trial of factor VIII and neutralizing antibodies in hemophilia A

F Peyvandi, PM Mannucci, I Garagiola… - … England Journal of …, 2016 - Mass Medical Soc
Background The development of neutralizing anti–factor VIII alloantibodies (inhibitors) in
patients with severe hemophilia A may depend on the concentrate used for replacement …

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

G Young, RI Liesner, T Chang… - Blood, The Journal …, 2019 - ashpublications.org
Emicizumab, a bispecific humanized monoclonal antibody, bridges activated factor IX (FIX)
and FX to restore the function of missing activated FVIII in hemophilia A. Emicizumab …

The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab

M Carcao, C Escuriola‐Ettingshausen… - …, 2019 - Wiley Online Library
Introduction As a result of the new treatment paradigm that the haemophilia community will
face with the availability of novel (non‐factor) therapies, an updated consensus on ITI …

Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors

PT Ebbert, F Xavier, CD Seaman, MV Ragni - Haemophilia, 2020 - Wiley Online Library
Introduction Emicizumab is a bispecific monoclonal antibody that mimics factor VIII (FVIII) by
binding to factors IXa and X to promote hemostasis in haemophilia A (HA) and HA with …

Mortality in congenital hemophilia A–a systematic literature review

CRM Hay, F Nissen, SW Pipe - Journal of Thrombosis and …, 2021 - Wiley Online Library
Against a background of a rapidly evolving treatment landscape, a contemporary, evidence‐
based consolidated understanding of mortality in people with congenital hemophilia A …

Laboratory monitoring in emicizumab-treated persons with hemophilia A

J Müller, I Pekrul, B Pötzsch, B Berning… - Thrombosis and …, 2019 - thieme-connect.com
Hemophilia A (HA) is an X-linked hereditary bleeding disorder caused by deficiency of
coagulation factor (F) VIII activity. One of the greatest complications in the treatment of HA is …

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

MA Mazepa, PE Monahan, JR Baker… - Blood, The Journal …, 2016 - ashpublications.org
The availability of longitudinal data collected prospectively from 1998 to 2011 at federally
funded US hemophilia treatment centers provided an opportunity to construct a descriptive …

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

SW Pipe, P Collins, C Dhalluin, G Kenet, C Schmitt… - Blood, 2024 - ashpublications.org
Subcutaneous emicizumab enables prophylaxis for people with hemophilia A (HA) from
birth, potentially reducing risk of bleeding and intracranial hemorrhage (ICH). HAVEN 7 …