科研动态期刊摘要
[评论] RISE UP试验中的Mitapivat：镰状细胞病的一种新疗法
The Lancet · 2026-10-23 21:12
摘要
镰状细胞病是最常见的血红蛋白病，全球患病人数超过700万。1尽管其发病率和死亡率都很高，但获得有效改善病情治疗的可及性仍然有限。2,3羟基脲和慢性输血均已被证明可减少镰状细胞病的急性并发症，包括血管闭塞性危象和卒中，同时也能改善血液学参数。4,5然而，在某些情况下，两者的可及性和长期可行性可能受到限制。
English
[Comment] Mitapivat in the RISE UP trial: a new therapy for sickle cell disease
Sickle cell disease is the most common haemoglobinopathy, affecting more than 7 million people worldwide.1 Despite substantial morbidity and mortality, access to effective disease-modifying therapies remains limited.2,3 Hydroxyurea and chronic blood transfusions have both been shown to reduce acute sickle cell disease complications, including vaso-occlusive crises and stroke, while also improving haematological parameters.4,5 However, the availability and long-term feasibility of both could be limited in some settings.