Department of Pediatrics, Boston Medical Center.
Division of Hematology.
Curr Opin Hematol. 2020 May;27(3):172-180. doi: 10.1097/MOH.0000000000000582.
Over the past several decades, hydroxyurea has emerged as a well tolerated and potent disease-modifying therapy for children and adults with sickle cell anemia (SCA). Strong, evidence-based recommendations from the National Institutes of Health, American Society of Hematology, and British Society of Haematology document that hydroxyurea is now standard of care treatment for SCA. In low-resource settings, however, hydroxyurea is rarely utilized due to lack of availability, inadequate treatment guidance, and excessive costs.
Research trials conducted within the Caribbean and sub-Saharan Africa confirm the efficacy of hydroxyurea as a well tolerated, feasible, and beneficial treatment in low-resource countries. Hydroxyurea is therefore vital to reaching the targets for control of SCA outlined by the WHO. To maximize its utilization toward real-world effectiveness, specific attention must be given to healthcare provider education and training, public and institutional awareness, and medication access and affordability.
Efforts to introduce hydroxyurea effectively into low-resource countries should urgently address the lack of treatment guidelines, gaps in knowledge and clinical infrastructure, and medication inaccessibility. Partnerships among governmental, academic, pharmaceutical, and charitable organizations must tackle these barriers so that all individuals living with SCA worldwide can benefit from hydroxyurea.
在过去几十年中,羟基脲已成为治疗镰状细胞贫血(SCA)儿童和成人的一种耐受性良好且有效的疾病修正疗法。美国国立卫生研究院、美国血液学会和英国血液学会的强有力的循证建议证明,羟基脲现在是 SCA 的标准治疗方法。然而,在资源匮乏的环境中,由于缺乏可用性、治疗指导不足和费用过高,羟基脲很少被使用。
在加勒比和撒哈拉以南非洲进行的研究试验证实,羟基脲作为一种耐受性良好、可行且有益的治疗方法,在资源匮乏的国家是有效的。因此,羟基脲对于实现世界卫生组织(WHO)制定的 SCA 控制目标至关重要。为了最大限度地提高其在实际效果中的应用,必须特别关注医疗保健提供者的教育和培训、公众和机构意识以及药物的可及性和可负担性。
将羟基脲有效地引入资源匮乏国家的工作应紧急解决缺乏治疗指南、知识和临床基础设施方面的差距以及药物不可及的问题。政府、学术、制药和慈善组织之间的伙伴关系必须解决这些障碍,以便全球所有患有 SCA 的人都能从羟基脲中受益。