Irish Haemophilia Society Ltd. and Trinity College, Dublin, Ireland.
Haemophilia. 2020 Sep;26(5):752-758. doi: 10.1111/hae.14097. Epub 2020 Aug 4.
Haemophilia care has experienced unprecedented advances over the past 50 years, with the promise of a functional 'cure' being within reach in the not so distant future. This review outlines the challenges the haemophilia community in Europe has faced over the decades since the 1970s and how it has overcome them to steadily advance care. It will also look towards the future and anticipate the hurdles to accessing the next wave of innovation and apply how past experience can help reduce or eliminate these hurdles. The lessons we can learn from the past can inform not simply treatment decisions, but shape the policy, advocacy and access environment needed to make the future promise a reality. The future is bright, but only through active participation across the haemophilia community will we be able to achieve it. We need to work together, to ensure progress will not only be realized by those with haemophilia in Western Europe, but by all those with haemophilia and other inherited bleeding disorder throughout Europe.
过去 50 年来,血友病的治疗取得了前所未有的进展,在不久的将来,一种功能性的“治愈”方法即将问世。这篇综述概述了自 20 世纪 70 年代以来,欧洲血友病社区几十年来所面临的挑战,以及它如何克服这些挑战,稳步推进治疗。它还将展望未来,预测下一波创新的障碍,并探讨过去的经验如何帮助减少或消除这些障碍。我们从过去的经验中可以学到的东西不仅可以指导治疗决策,还可以影响政策、宣传和获得治疗所需的环境,以实现未来的承诺。未来是光明的,但只有通过血友病社区的积极参与,我们才能实现这一目标。我们需要共同努力,确保不仅在西欧的血友病患者中,而且在整个欧洲的所有血友病患者和其他遗传性出血性疾病患者中都能实现这一目标。