Meeting the challenges of haemophilia care and patient support in China and Brazil

Abstract

Projects are underway in many developing countries to try to improve the provision of treatment and access to care for people with haemophilia (PWH), as long-term prophylactic treatment, which improves quality of life for PWH, is still restricted to developed countries. In most developing countries, therapy is limited to on-demand treatment or even no replacement treatment at all. Combined with limited healthcare resources, this lack of treatment can lead to a vicious circle of lack of care, disability, unemployment and lack of access to health insurance for haemophilia patients. In China, the establishment of the Haemophilia Treatment Centre Collaborative Network of China (HTCCNC), in conjunction with the World Federation of Hemophilia, has improved haemophilia care and the identification of PWH. In Brazil, on-demand treatment has improved the health of PWH but does not prevent musculoskeletal (MSK) complications, the major cause of deterioration in quality of life for PWH. The Novo Nordisk Haemophilia Foundation BR2 project was therefore designed to improve quality of life of PWH through improvements in their physical, mental and social wellbeing. This paper will briefly review these projects and describe the current status of haemophilia care in these countries. While there is still a long way to go before optimal care becomes a reality for all PWH in developing countries, significant progress has been made, and knowledge of the impact and outcomes of these projects can inform best practice worldwide.

Principles of haemophilia care: The Asia‐Pacific perspective

“…PWH in countries without established haemophilia care programmes have a high mortality rate by early adulthood . The lack of adequately supported universal healthcare systems and significant out‐of‐pocket treatment costs, frequently associated with copayment systems, which is common in Asia‐Pacific, can lead to grossly suboptimal management of PWH with life‐threatening consequences . Even in some economically strong countries, there is often limited funding for treatment and care of PWH.…”

“…58 The lack of adequately supported universal healthcare systems and significant out-of-pocket treatment costs, frequently associated with copayment systems, which is common in Asia-Pacific, can lead to grossly suboptimal management of PWH with life-threatening consequences. 68 73 Adolescents tend to be less adherent with prophylaxis than younger patients which can be a particularly significant issue in Asia-Pacific, where the concept and implementation of home therapy is still evolving. 74 Peer-support programmes, education and acknowledgement of caregiver needs are valuable in addressing psychosocial needs of PWH and their families.…”

Efficacy and safety of prophylaxis with BAY 81‐8973 in Chinese patients with severe haemophilia A enrolled in the LEOPOLD II trial

“…1 Consequently, in countries such as China, in which access to haemophilia care is limited and patient incomes are low, most patients with haemophilia are treated on-demand, with FVIII administered at the time of a bleeding episode, and some patients may receive no treatment at all. 3,4 Lack of access to FVIII prophylaxis in some Chinese regions has historically led to this population being undertreated, which subsequently increases the likelihood of joint damage and compromises health-related quality of life in patients with haemophilia A. 6 It is possible that for this reason the joint status and treatment history of the Chinese patients differ from other populations.…”

Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A

“…In China, primary prophylaxis is usually not possible because of a lack of adequate resources, particularly in rural areas [10,11]. As a result, joint outcomes and QoL are worse in China compared with developed countries in which primary prophylaxis is standard care [12,13].…”