Centre for Haemoglobinopathies, Department of Haematology, Herlev and Gentofte Hospital, University of Copenhagen, Herlev, Denmark.
Sickle Cell Disease Reference Center, Department of General Pediatrics and Pediatric Infectious Diseases, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
Br J Haematol. 2020 Jul;190(2):149-172. doi: 10.1111/bjh.16504. Epub 2020 Mar 6.
Despite sickle cell disease (SCD) being the most common and severe inherited condition worldwide, therapeutic options are limited. To date, hydroxyurea remains the main treatment option in SCD. However, in the last decade the numbers of interventional clinical trials focussing on therapies for SCD have increased significantly. Many new drugs with various pharmacological targets have emerged and, although the majority have failed to show benefit in clinical trials, some have produced encouraging results. It seems probable that more drugs will soon become available for the treatment of SCD. Furthermore, promising clinical trials with improved outcomes have recently changed the perspective of curative therapies in SCD. Nevertheless, the application of novel therapeutic agents and potential curative treatments will most likely be limited to high-income countries and, thus, will remain unavailable for the majority of people with SCD in the foreseeable future.
尽管镰状细胞病（SCD）是世界上最常见和最严重的遗传性疾病，但治疗选择有限。迄今为止，羟基脲仍然是 SCD 的主要治疗选择。然而，在过去十年中，专注于 SCD 治疗的干预性临床试验数量显著增加。许多具有不同药理靶点的新药已经出现，尽管大多数药物在临床试验中未能显示出获益，但有些药物产生了令人鼓舞的结果。似乎很快就会有更多的药物可用于治疗 SCD。此外，最近一些具有改善结局的有前景的临床试验改变了 SCD 中治愈疗法的观点。然而，新型治疗药物的应用和潜在的治愈治疗很可能仅限于高收入国家，因此，在可预见的未来，SCD 的大多数人仍无法获得这些治疗。
