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Emerging therapies in sickle cell disease.
British Journal of Haematology ( IF 6.5 ) Pub Date : 2020-03-06 , DOI: 10.1111/bjh.16504 Amina Nardo-Marino 1 , Valentine Brousse 2 , David Rees 3
British Journal of Haematology ( IF 6.5 ) Pub Date : 2020-03-06 , DOI: 10.1111/bjh.16504 Amina Nardo-Marino 1 , Valentine Brousse 2 , David Rees 3
Affiliation
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.
中文翻译:
镰状细胞疾病的新兴疗法。
尽管镰状细胞病(SCD)是全世界最常见和最严重的遗传病,但治疗选择有限。迄今为止,羟基脲仍是SCD中的主要治疗选择。然而,在过去的十年中,专注于SCD治疗的介入性临床试验的数量已大大增加。已经出现了许多具有各种药理学靶标的新药,尽管大多数没有在临床试验中显示出益处,但有些却产生了令人鼓舞的结果。似乎很快有更多药物可用于治疗SCD。此外,最近有希望的临床试验具有改善的结果,最近改变了SCD的治疗方法。不过,
更新日期:2020-03-06
中文翻译:
镰状细胞疾病的新兴疗法。
尽管镰状细胞病(SCD)是全世界最常见和最严重的遗传病,但治疗选择有限。迄今为止,羟基脲仍是SCD中的主要治疗选择。然而,在过去的十年中,专注于SCD治疗的介入性临床试验的数量已大大增加。已经出现了许多具有各种药理学靶标的新药,尽管大多数没有在临床试验中显示出益处,但有些却产生了令人鼓舞的结果。似乎很快有更多药物可用于治疗SCD。此外,最近有希望的临床试验具有改善的结果,最近改变了SCD的治疗方法。不过,