Achieving the unimaginable: Health equity in haemophilia.
Achieving the unimaginable: Health equity in haemophilia.
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DOI：
10.1111/hae.13862
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发表时间：
2020-01
期刊：
影响因子：
--
通讯作者：
Berntorp E
中科院分区：
文献类型：
--
作者：
Skinner MW;Nugent D;Wilton P;O'Mahony B;Dolan G;O'Hara J;Berntorp E
从历史上看，基于凝血因子替代的治疗导致了血友病（PwH）患者因子缺乏纠正的晦涩指南。虽然所有其他疾病实体都寻求将功能恢复到正常水平，但PwH仅限于仍相当于轻度或中度疾病的因子最低点，导致出血的风险持续存在。需要一种基于PwH明确需求的新治疗模式。由血友病提供者、患者倡导者和卫生经济学家组成的小组开发了一种治疗模式，以确定具体的治疗里程碑和目标结果。该小组定义了一系列治疗里程碑，以表征与因子缺乏纠正水平相关的活动和结果。所有人都同意，最终目标应该是“功能性治愈”和“卫生公平”。确定了实现功能性治愈的七个层次：(a)维持生命；(b)最小限度的关节损伤；(c)无任何自发性出血；(d)实现“正常”行动能力；(e)能够承受轻微创伤而无需额外干预；(f)承受大手术或创伤的能力；(g)正常止血。确定了一组平行的患者报告的结果，以实现卫生公平。这些指导方针现在可与其他疾病相比较，其目标是替换缺失的蛋白质以达到正常的活动水平。由于重组因子、模拟物的制造和基因治疗的早期成功，我们不再受血浆供应的限制，健康公平现在是可以实现的。
Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be ‘functional cure’ and ‘health equity’. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds; (d) Attainment of ‘normal’ mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient‐reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable.
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