Sickle cell disease (SCD) is one of the most common genetic diseases with some 250,000 new births each year. Most patients suffer intermittent pain crises and life-threatening events while life expectancy is considerably reduced. Until the last decade management was purely preventative or supportive aimed at symptom control. Apart from stem cell transplant, there is no cure but the oral chemotherapeutic drug hydroxyurea (HU) has now established a role in ameliorating the disease and improving life expectancy for most patients. There are side effects and risks of HU treatment in SCD but for moderate and severely affected patients, the benefits can be significant.

中文翻译：

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羟基脲在镰状细胞疾病管理中的作用。

镰状细胞病（SCD）是最常见的遗传疾病之一，每年约有25万例新出生。大多数患者遭受间歇性疼痛危机和威胁生命的事件，而预期寿命却大大缩短。直到最近十年，治疗仅是针对症状控制的预防或支持。除干细胞移植外，尚无治愈方法，但口服化学治疗药物羟基脲（HU）现在已在缓解疾病和提高大多数患者的预期寿命方面发挥了作用。在SCD中进行HU治疗存在副作用和风险，但是对于中度和重度感染的患者，获益可能是巨大的。