Feasibility and outcomes of low‐dose and low‐frequency prophylaxis with recombinant extended half‐life products (Fc‐rFVIII and Fc‐rFIX) in Ivorian children with hemophilia: Two‐year experience in the setting of World Federation of Haemophilia humanitarian aid programme

Abstract

Introduction In sub‐Saharan Africa, access to clotting factor concentrates (CFCs) is often extremely limited and published data on people with haemophilia on prophylaxis are almost not existent. Aims and Methods To assess the feasibility, barriers and outcomes of a low‐dose and low‐frequency prophylaxis with extended half‐life (EHL) recombinant Fc fusion FVIII and FIX in Ivorian children on a two‐year period in the setting of the World Federation of Hemophilia's (WFH) humanitarian aid programme. Results Twenty‐five boys with haemophilia were included. Mean (SD) age at inclusion was 5.6 (2.5) years. The median [range] follow‐up duration was 17 [11–24] months. Regimen of prophylaxis was 20 IU kg−1 1×/week in haemophilia A and every 10 days in haemophilia B. We observed a maximal reduction by 87.6% of the annual spontaneous joint bleeding rate and a slight decrease in the total HJHS scores (p = .047). Adherence problems related to parents' low education level and shortage in CFCs were the main issues to carry out the programme. Inhibitors occurred in 12.5%. Conclusion This study confirms the feasibility and efficacy of low‐dose and low‐frequency prophylaxis in young Ivorian children with haemophilia treated with EHL CFCs donated through the WFH humanitarian aid programme. This work also highlights the crucial role of adherence and the need for appropriate education to achieve prophylaxis. Finally, it reminds the paramount objective of achieving self‐sufficient, sustainable and available haemophilia replacement therapy for all worldwide.

Significant reduction in hemarthrosis in boys with severe hemophilia A: The China hemophilia individualized low‐dose secondary prophylaxis study

“…by investigators from China, 13,14 India, 25 Indonesia, 26 Thailand, 27 Tunisia, 28 and the Ivory Coast. 29 Collectively, these eight studies enrolled a total of 251 boys with hemophilia (hemophilia A = 232…”

“…[92.4%], hemophilia B = 19 [7.6%]) 29 over a 28-year period (1992-2020). The starting low-dose prophylaxis regimen most commonly used for boys with hemophilia A was 10 IU/kg given twice weekly.…”

Disruptive technology and hemophilia care: The multiple impacts of emicizumab

“…So while most patients in more developed countries have access to a wide range of increasingly ambitious treatment options, patients in less developed countries can only expect to have access to donated factor concentrates for minimal prophylaxis, and only in children. 29 Although there is a major gap between developed and less developed countries, the management of hemophilia is gradually and constantly improving in both worlds. The revolution of the therapeutic landscape in the developed world should not be at the expense of the less developed countries.…”

“…In many countries, it is now possible to treat young children with prophylactic regimens, including EHL FVIII and FIX concentrates used in reduced doses. So while most patients in more developed countries have access to a wide range of increasingly ambitious treatment options, patients in less developed countries can only expect to have access to donated factor concentrates for minimal prophylaxis, and only in children 29 . Although there is a major gap between developed and less developed countries, the management of hemophilia is gradually and constantly improving in both worlds.…”

Impact of humanitarian aid linked prophylaxis in Côte d'Ivoire (Ivory Coast)

“…WFH programmes such as Hemophilia Treatment Center Twinning (which occurred in this case between Belgium and Côte d'Ivoire, 10 the World Bleeding Disorders Registry (WBDR), which establishes the importance of prospective tracking and management of bleeding and treatment regimens in patient populations, 11 and regionally oriented haemophilia management training programmes to reinforce the principles of comprehensive care to healthcare providers form a more inclusive approach towards advancing prophylactic regimens in lower resource countries 7 . This includes reinforcing the need among physicians to develop home care therapy programmes, as initiated in Côte d'Ivoire 6 to obviate the noncompliance intrinsic to hospital‐based prophylactic programmes. In countries where there are great distances and inadequate transportation, hospital‐based prophylactic infusion programmes should not be the first choice.…”