Hemophilia treatment innovation: 50 years of progress and more to come

Towards achieving a haemophilia‐free mind

“…The development of a bispecific antibody mimicking the action of FVIII, administered subcutaneously on a weekly basis or every 2 or 4 weeksmakes it possible to maintain a constant haemostatic activity equivalent to approximately 15% of FVIII 7 . In clinical trials are various therapeutic agents which, by reducing antithrombin or inhibiting Tissue Factor Pathway Inhibitor or Activated Protein C, increase thrombin production independently of the supply of exogenous FVIII or FIX or their neutralization by allo‐antibodies 8,9 . These clotting cascade rebalancing agents, designed to decrease anti‐coagulant activity in persons with bleeding disorders, will need to be used judiciously since there have been reports of excess coagulation (thrombotic events) 10 .…”

“…7 In clinical trials are various therapeutic agents which, by reducing antithrombin or inhibiting Tissue Factor Pathway Inhibitor or Activated Protein C, increase thrombin production independently of the supply of exogenous FVIII or FIX or their neutralization by alloantibodies. 8,9 These clotting cascade rebalancing agents, designed to decrease anti-coagulant activity in persons with bleeding disorders, will need to be used judiciously since there have been reports of excess coagulation (thrombotic events). 10 They may be of benefit to selected patients since they can also be administered subcutaneously and some require infrequent injections.…”

Guidance for the antithrombotic management of persons with haemophilia and cardiovascular disease

“…Owing to the continuous progress of therapies for the haemophilias in the last 50 years, 1 persons with these congenital coagulation disorders have attained a life expectancy very close to that of their unaffected peers. This fulfilment is inevitably accompanied by an increase of such age‐related illnesses as cardiovascular disease (CVD) 2 .…”

“…21 However, unmet needs remained pertaining to the antithrombotic management of PWH, because recommendations were largely informed by expert opinions in the absence of high quality data. [12][13][14][15][16][17][18][19] Furthermore, the most recent therapeutic advances, such as emicizumab and gene transfer, 1 were not taken into account.…”

Perspectives and perception of haemophilia gene therapy by French patients

“…4 Moreover, non-factor therapies have been developed, mimicking factor VIII and allowing for subcutaneous prophylaxis. 5 These pharmacological progresses have already greatly improved the quality of life (QOL) of patients with hemophilia. 6 New innovative advances are ongoing.…”