Karkoska Kristine, McGann Patrick T
Division of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.
Pediatr Blood Cancer. 2021 Dec;68(12):e29363. doi: 10.1002/pbc.29363. Epub 2021 Sep 22.
Finally,after decades of stagnation, the therapeutic landscape for sickle cell disease (SCD) is changing with an increasing number of novel therapeutics. Hydroxyurea remains the primary disease-modifying therapy and, when started early in life with maintenance of an optimal dose, can reduce many SCD-related complications. To complement hydroxyurea, there are a growing number of pharmacologic options with additional efforts focused on the development and optimization of curative therapies. Here, we review current treatment options and provide recommendations as to how to approach the treatment of children and adolescents within this evolving therapeutic landscape to allow for full and healthy lives.
最后，经过数十年的停滞不前，镰状细胞病（SCD）的治疗格局正在随着越来越多的新型疗法而发生变化。羟基脲仍然是主要的疾病改善疗法，在生命早期开始并维持最佳剂量时，可以减少许多与SCD相关的并发症。为了补充羟基脲，有越来越多的药物选择，同时也加大了对治愈性疗法的研发和优化力度。在此，我们回顾当前的治疗选择，并就如何在这一不断发展的治疗格局中对儿童和青少年进行治疗提供建议，以实现充实而健康的生活。
