Outcome of Emicizumab Prophylaxis in Patients of Haemophilia A with or without Inhibitor An Early Experience from A Tertiary Care Hospital From Eastern India

Prophylaxis in hemophilia consists of regular administration of therapeutic products aimed at maintaining hemostasis to prevent bleeding, especially joint hemorrhages, which would lead to arthropathy and disability. Prophylaxis should enable people with hemophilia to lead healthy and active lives including participation in most physical and social activities (at home, school, work, and in the community), similar to the non-hemophilic population.

Episodic therapy, regardless of the doses used, while essential in reducing the pain and debilitating impact of individual bleeds, does not alter the bleeding profile significantly and hence does not change the natural history of hemophilia leading to musculoskeletal damage and other complications due to bleeding. Therefore, the use of prophylaxis is always recommended over episodic therapy.

With the advent of innovative non-factor replacement therapies, which for the most part can be administered subcutaneously, prophylaxis is being redefined as the regular administration (intravenously, subcutaneously, or otherwise) of a hemostatic agent/ agents to enhance hemostasis and effectively prevent bleeding in people with hemophilia.

In order to optimize treatment and make economically sound clinical decisions, objective evidence of both short- and long-term outcomes of treatment regimens is required.

Outcome refers to the condition of a patient that results from a disease or medical intervention. It is assessed by clinical evaluation including the use of generic and disease-specific health-related quality of life (HRQoL) assessment instruments, measures of patient-reported outcomes (PROs), and laboratory tests including imaging studies. These instruments measure a variety of parameters including activities and participation, body structure and function, burden of disease, and subjective health status.

Outcome assessment may be used to follow an individual’s disease course, obtain information to guide routine clinical care, measure response to therapy, and determine whether there is a need to modify therapy. Outcome assessment may also be used to quantify the health of a group of patients, measure quality of care, and advocate for resources. In addition, outcome assessment may be used for research purposes such as to document the natural history of the disease, test new therapies, or compare different therapies which in turn may be used to guide informed decisions regarding expenditures on treatment.

Emicizumab is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors.

Till date Emicizumab is the only approved FVIII mimetic agent which can be used for prophylaxis in patients of Haemophilia A with or without inhibitors.