Sapienza University, Department of Cellular Biotechnologies and Hematology , Via Benevento 6, 00161, Rome , Italy +3906857951 ; +390644241984 ;
Expert Opin Pharmacother. 2014 Aug;15(11):1621-30. doi: 10.1517/14656566.2014.936849. Epub 2014 Jul 3.
Therapeutic strategies for elderly patients affected by myelodysplastic syndromes (MDS) are scarce and only few patients have an advantage in performing allogeneic bone marrow transplant.
Primary endpoints for treatment of elderly MDS patients were not curative, but rather allowing to maintain a good quality of life through prolongation of overall survival. In this context, azacitidine showed to improve responses in this subset of patients compared to conventional established regimens, such as intensive or low-dose chemotherapy and best supportive care. Good safety profile of the drug was reported either when it was used inside or outside clinical trials. Improved quality of response was observed when the drug was administered beyond the first response, and it is now usually recommended to continue it at the same dose and schedule in responding patients.
Evaluation of baseline prognostic factors and comorbidities may help to identify patients who can benefit from the prolonged administration of the drug. Real life data regarding efficacy and safety of azacitidine in MDS elderly patients are required in order to confirm the results of clinical trials.
针对骨髓增生异常综合征(MDS)老年患者的治疗策略较为匮乏,仅有少数患者适合进行异基因骨髓移植。
老年 MDS 患者的治疗主要目标并非治愈,而是通过延长总生存期来维持良好的生活质量。在这种情况下,与强化或低剂量化疗和最佳支持治疗等传统既定方案相比,阿扎胞苷可改善此类患者的反应。该药无论在临床试验内还是外使用,均报告了良好的安全性。当药物在首次反应后继续使用时,可观察到反应质量的改善,目前通常建议在有反应的患者中以相同剂量和方案继续使用。
评估基线预后因素和合并症有助于确定哪些患者可从药物的长期使用中获益。需要有 MDS 老年患者中阿扎胞苷的疗效和安全性的真实数据,以证实临床试验结果。