Division of Pediatric Hematology Oncology, Riley Hospital for Children, Indianapolis, IN, USA.
Division of Pediatric Hematology, Oncology & Blood/Marrow Transplantation, Cleveland Clinic Children's Hospital, Cleveland, OH, USA.
Lancet Child Adolesc Health. 2023 Nov;7(11):797-808. doi: 10.1016/S2352-4642(23)00201-8.
Sickle cell disease is the most common inherited pathological haemoglobinopathy. Over the past 30 years, disease-related morbidity and mortality have improved in high-income countries due to advances in preventive care and treatments. Established disease-modifying therapies, such as hydroxyurea (hydrocarbamide), are continuing to have an important role in the treatment of sickle cell disease, and newer agents also show promise. In the past 5 years, the US Food and Drug Administration approved three additional sickle cell disease-modifying medications, and new gene therapies have been developed as an alternative curative treatment to haematopoietic stem-cell transplantation. In this Review, we discuss the current treatment landscape for paediatric sickle cell disease and emerging innovations in care. We also review the need for close, long-term management for children receiving newer therapies and the importance of ongoing investment in people with sickle cell disease in low-income and middle-income countries.
镰状细胞病是最常见的遗传性血红蛋白病。在过去的 30 年中，由于预防保健和治疗方面的进步，高收入国家的与疾病相关的发病率和死亡率有所改善。羟基脲（hydrocarbamide）等已确立的疾病修正疗法在镰状细胞病的治疗中继续发挥重要作用，新型药物也显示出前景。在过去的 5 年中，美国食品和药物管理局批准了另外三种镰状细胞疾病修正药物，并且已经开发出新型基因疗法作为造血干细胞移植的替代治疗方法。在这篇综述中，我们讨论了儿科镰状细胞病的当前治疗现状和护理方面的新进展。我们还回顾了接受新型疗法的儿童需要密切、长期管理的必要性，以及在低收入和中等收入国家对镰状细胞病患者持续投资的重要性。
