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WFH:弥合全球差距——实现最佳护理。

WFH: closing the global gap--achieving optimal care.

机构信息

World Federation of Hemophilia, Washington, DC 20037-3303, USA.

出版信息

Haemophilia. 2012 Jul;18 Suppl 4:1-12. doi: 10.1111/j.1365-2516.2012.02822.x.

Abstract

For 50 years, the World Federation of Hemophilia (WFH) has been working globally to close the gap in care and to achieve Treatment for All patients, men and women, with haemophilia and other inherited bleeding disorders, regardless of where they might live. The WFH estimates that more than one in 1000 men and women has a bleeding disorder equating to 6,900,000 worldwide. To close the gap in care between developed and developing nations a continued focus on the successful strategies deployed heretofore will be required. However, in response to the rapid advances in treatment and emerging therapeutic advances on the horizon it will also require fresh approaches and renewed strategic thinking. It is difficult to predict what each therapeutic advance on the horizon will mean for the future, but there is no doubt that we are in a golden age of research and development, which has the prospect of revolutionizing treatment once again. An improved understanding of "optimal" treatment is fundamental to the continued evolution of global care. The challenges of answering government and payer demands for evidence-based medicine, and cost justification for the introduction and enhancement of treatment, are ever-present and growing. To sustain and improve care it is critical to build the body of outcome data for individual patients, within haemophilia treatment centers (HTCs), nationally, regionally and globally. Emerging therapeutic advances (longer half-life therapies and gene transfer) should not be justified or brought to market based only on the notion that they will be economically more affordable, although that may be the case, but rather more importantly that they will be therapeutically more advantageous. Improvements in treatment adherence, reductions in bleeding frequency (including microhemorrhages), better management of trough levels, and improved health outcomes (including quality of life) should be the foremost considerations. As part of a new WFH strategic plan (2012-2014) the WFH has identified several key initiatives for particular emphasis - continuation of the Global Alliance for Progress (GAP) program, a new initiative to address underserved countries and regions (The Cornerstone Initiative), enhancing health outcomes research and analysis, and a new research mentorship program. Despite our progress to date in closing the global gap in care, our work is not complete. Too many patients remain undiagnosed and too few receive adequate treatment. This paper will also discuss historical, present and future challenges and opportunities to close the gap in care and achieve Treatment for All.

摘要

五十年来,世界血友病联盟(WFH)一直致力于全球范围内缩小治疗差距,为所有男性和女性血友病及其他遗传性出血性疾病患者提供治疗,无论他们居住在哪里。WFH 估计,全世界每 1000 人中就有 1 人以上患有出血性疾病,相当于 690 万人。为了缩小发达国家和发展中国家之间的治疗差距,需要继续关注迄今为止取得的成功战略。然而,针对治疗方面的快速进展和即将出现的治疗新进展,也需要采取新的方法和更新的战略思维。很难预测即将出现的每一项治疗新进展对未来意味着什么,但毫无疑问,我们正处于研究和开发的黄金时代,这有望再次彻底改变治疗方法。对“最佳”治疗的更深入理解是全球治疗持续发展的基础。应对政府和支付方对循证医学的需求以及为引入和加强治疗进行成本论证的挑战始终存在且日益严峻。为了维持和改善治疗效果,至关重要的是在血友病治疗中心(HTCs)、国家、地区和全球范围内为每位患者建立个体治疗效果数据。新兴治疗进展(半衰期更长的疗法和基因转移)不应仅基于其在经济上更实惠的理念来证明或推向市场,尽管这可能是事实,但更重要的是,它们在治疗上更具优势。提高治疗依从性、减少出血频率(包括微出血)、更好地控制谷底水平以及改善健康结果(包括生活质量)应是首要考虑因素。作为世界血友病联盟新战略计划(2012-2014 年)的一部分,世界血友病联盟确定了几个特别强调的重点倡议——继续实施全球进步联盟(GAP)计划,发起一项新倡议以解决服务不足的国家和地区(基石倡议),加强健康结果研究和分析,以及开展新的研究指导计划。尽管我们在缩小全球治疗差距方面取得了进展,但我们的工作尚未完成。仍有许多患者未被诊断,接受充分治疗的患者也很少。本文还将讨论缩小治疗差距和实现人人享有治疗的历史、现状和未来挑战与机遇。

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