Unit of Hematology and Stem Cell Transplantation, IRCCS, Centro di Riferimento Oncologico della Basilicata, Via Padre Pio 1, 85028 Rionero in Vulture, Potenza, Italy.
Cancer. 2010 Mar 15;116(6):1485-94. doi: 10.1002/cncr.24894.
Azacitidine induces responses and prolongs overall survival compared with conventional care regimens in patients who have high-risk myelodysplastic syndromes (MDS). However, limited data are available concerning the efficacy and safety of azacitidine in patients who have lower risk MDS.
The authors retrospectively evaluated 74 patients with International Prognostic Scoring System low-risk or intermediate 1-risk MDS, who received azacitidine on a national named patient program. At baseline, 84% of patients were transfusion-dependent, 57% had received erythropoietin, and 51% were aged >70 years. Azacitidine was administered subcutaneously for 5 days (n = 29 patients), 7 days (n = 43 patients), or 10 days (n = 2 patients) every month at a dose of 75 mg/m(2) daily (n = 45 patients) or at a fixed dose of 100 mg daily (n = 29 patients) and for a median of 7 cycles (range, 1-30 cycles).
According to the 2006 International Working Group criteria, overall response rate (ORR) was 45.9%, including complete responses (10.8%), partial responses (9.5%), hematologic improvements (20.3%), and bone marrow complete responses (5.4%). The ORR was 51.6% in 64 patients who completed > or = 4 cycles of treatment. The median duration of response was 6 months (range, 1-30 months). After a median follow-up of 15 months, 71% of patients remained alive. A survival benefit was observed in responders versus nonresponders (94% vs 54% of patients projected to be alive at 2.5 years, respectively; P < .0014). The most common grade 3 or 4 adverse events were myelosuppression (21.6%) and infection (6.8%).
The current results indicated that azacitidine may be a feasible and effective treatment for patients with lower risk MDS.
阿扎胞苷与传统治疗方案相比，可诱导高危骨髓增生异常综合征（MDS）患者发生缓解并延长总生存期。然而，有关低危 MDS 患者应用阿扎胞苷的疗效和安全性的数据有限。
作者回顾性评估了 74 例国际预后评分系统低危或中危 1 危 MDS 患者，这些患者在全国性的指定患者项目中接受了阿扎胞苷治疗。基线时，84%的患者依赖输血，57%的患者接受了促红细胞生成素治疗，51%的患者年龄＞70 岁。阿扎胞苷皮下给药，5 天（29 例患者）、7 天（43 例患者）或 10 天（2 例患者）方案，剂量为 75mg/m²，每日（45 例患者）或固定剂量 100mg 每日（29 例患者），中位疗程为 7 个周期（范围为 1-30 个周期）。
根据 2006 年国际工作组标准，总缓解率（ORR）为 45.9%，包括完全缓解（10.8%）、部分缓解（9.5%）、血液学改善（20.3%）和骨髓完全缓解（5.4%）。在完成＞4 个周期治疗的 64 例患者中，ORR 为 51.6%。缓解的中位持续时间为 6 个月（范围为 1-30 个月）。中位随访 15 个月后，71%的患者存活。与无应答者相比，应答者有生存获益（预计在 2.5 年内存活的患者分别为 94%和 54%；P＜.0014）。最常见的 3 级或 4 级不良事件为骨髓抑制（21.6%）和感染（6.8%）。
目前的结果表明，阿扎胞苷可能是低危 MDS 患者的一种可行且有效的治疗方法。
