Principles of haemophilia care: The Asia‐Pacific perspective

Abstract

Optimal haemophilia care is best established and implemented through a well-coordinated plan guided by clearly defined principles and priorities. A document which enunciates those details is therefore important. A successful example of this approach is the definition of principles of haemophilia care (PHC) outlined by the European Association for Haemophilia and Associated Disorders (EAHAD) and also the World Federation of Hemophilia. A similar document applicable to the Asia-Pacific region must take into account not only the highly varied healthcare systems but also the tremendous socio-economic and cultural diversities which impact provision of such care. The Asia-Pacific Haemophilia Working Group (APHWG), representing the countries in this region, has prepared this perspective of the PHC. While endorsing the overall framework outlined by EAHAD, this APHWG document emphasizes regional priorities on education and training of healthcare personnel in the diagnosis and management of hereditary bleeding disorders. Central coordinating agencies with wide stakeholder input, networks of haemophilia treatment centres and national registries as well as robust processes for procurement and distribution of safe and effective clotting factor concentrates (CFCs), implementation of prophylaxis programmes and management of patients with inhibitors should also be developed. The implementation of these strategies should lead to establishment of good comprehensive care programmes. This document should also be an advocacy tool to lobby for improved care for people with haemophilia (PWH) in the region. We urge national healthcare policy makers to consider these principles and initiate strong and decisive action to reach these goals.

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

“…As a result, in some areas of China, many patients receive little or no treatment . Even low‐dose, short‐term prophylaxis treatment provides some clinical benefit for these patients, indicating that standard‐dose prophylaxis as recommended by the WFH would have substantial effects on clinical outcomes and quality of life . Indeed, results of the present study indicate that the benefits of standard‐dose prophylaxis for Chinese patients would likely be similar to those observed for the rest of the world.…”

“…Ideally, patients with haemophilia in China should receive individualized prophylaxis as recommended by the World Federation of Hemophilia (WFH) . Low‐dose prophylaxis has been explored as a cost‐saving approach to treatment of Chinese patients with haemophilia and results have been encouraging.…”

“…Patients have experienced fewer joint bleeding episodes and some improvement in daily activities compared with on‐demand treatment . For these reasons, it should be considered an initial step in haemophilia care in patients where high‐dose prophylaxis is not required or not possible due to the economic burden …”

“…10 For these reasons, it should be considered an initial step in haemophilia care in patients where high-dose prophylaxis is not required or not possible due to the economic burden. 8 BAY 81-8973 (Kovaltry ® ; Bayer, Berkeley, CA) is a full-length, unmodified recombinant human FVIII approved for prophylaxis and treatment of bleeding episodes in patients with haemophilia A 11,12 and according to Bayer's data on file has accumulated 8031 patient-years of exposure as of 31st August 2018. The efficacy and safety of BAY 81-8973 were demonstrated in the Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease (LEOPOLD) clinical trial program, which enrolled children, adolescents, and adults with severe haemophilia A at sites worldwide.…”

A survey of characteristics and current educational needs of hemophilia treatment centers within Asia Pacific

“…Although “diagnosis” was not identified as a high priority education topic across all economic capacity groups, it should be recognized that implementation of successful QA schemes will have a positive impact on accurate laboratory results and diagnosis 16, 30. We reiterate the recommendations outlined in the Asia Pacific Principles of Haemophilia Care10 and urge prioritization of the development and implementation of institutional or national protocols in hemostasis laboratories to achieve quality‐assured testing.…”