Beguin Y, Selleslag D, Meers S, Graux C, Bries G, Deeren D, Vrelust I, Ravoet C, Theunissen K, Voelter V, Potier H, Trullemans F, Noens L, Mineur P
Acta Clin Belg. 2015 Feb;70(1):34-43. doi: 10.1179/2295333714Y.0000000102. Epub 2014 Dec 2.
We evaluated azacitidine (Vidaza(®)) safety and efficacy in patients with myelodysplastic syndrome (MDS), acute myeloid leukaemia (AML), and chronic myelomonocytic leukaemia (CMML), in a real-life setting. Treatment response, dose, and schedule were assessed.
This non-interventional, post-marketing survey included 49/50 patients receiving azacitidine at 14 Belgian haematology centres from 2010-2012. Treatment-emergent adverse events (TEAEs), including treatment-related TEAEs, and serious TEAEs (TESAEs) were recorded throughout the study. Treatment response [complete response (CR), partial response (PR), haematological improvement (HI), stable disease (SD), treatment failure (TF)) and transfusion-independence (TI) were evaluated at completion of a 1-year observation period (1YOP) or at treatment discontinuation, and overall survival (OS), at study conclusion.
The median age of patients was 74·7 (range: 43·9-87·8) years; 69·4% had MDS, 26·5% had primary or secondary AML, and 4·1% had CMML. Treatment-related TEAEs, grade 3-4 TEAEs, and TESAEs were reported in 67·3%, 28·6%, and 18·4% of patients, respectively. During 1YOP, patients received a median of 7 (1-12) treatment cycles. Treatment response was assessed for 38/49 patients. Among MDS and CMML patients (n = 29), 41·4% had CR, PR, or HI, 41·4% had SD, and 17·2% had TF. Among AML patients (n = 9), 44·4% had CR or PR, 33·3% had SD, and 22·2% had TF. TI was observed in 14/32 (43·8%) patients who were transfusion-dependent at baseline. Median (95% confidence interval) OS was 490 (326-555) days; 1-year OS estimate was 0·571 (0·422-0·696).
Our data support previous findings that azacitidine has a clinically acceptable safety profile and shows efficacy.
我们在实际临床环境中评估了阿扎胞苷（维达莎®）对骨髓增生异常综合征（MDS）、急性髓系白血病（AML）和慢性粒单核细胞白血病（CMML）患者的安全性和疗效。评估了治疗反应、剂量和给药方案。
这项非干预性的上市后调查纳入了2010年至2012年期间在比利时14家血液学中心接受阿扎胞苷治疗的49/50例患者。在整个研究过程中记录治疗中出现的不良事件（TEAE），包括与治疗相关的TEAE和严重TEAE（TESAEs）。在1年观察期（1YOP）结束时或治疗中断时评估治疗反应[完全缓解（CR）、部分缓解（PR）、血液学改善（HI）、疾病稳定（SD）、治疗失败（TF）]和输血独立性（TI），并在研究结束时评估总生存期（OS）。
患者的中位年龄为74.7（范围：43.9 - 87.8）岁；69.4%患有MDS，26.5%患有原发性或继发性AML，4.1%患有CMML。分别有67.3%、28.6%和18.4%的患者报告了与治疗相关的TEAE、3 - 4级TEAE和TESAEs。在1YOP期间，患者接受的治疗周期中位数为7（1 - 12）个。对38/49例患者评估了治疗反应。在MDS和CMML患者（n = 29）中，41.4%达到CR、PR或HI，41.4%疾病稳定，17.2%治疗失败。在AML患者（n = 9）中，44.4%达到CR或PR，33.3%疾病稳定，22.2%治疗失败。在基线时依赖输血的32例患者中有14例（43.8%）实现了输血独立性。中位（95%置信区间）总生存期为490（326 - 555）天；1年总生存估计值为0.571（0.422 - 0.696）。
我们的数据支持先前的研究结果，即阿扎胞苷具有临床可接受的安全性且显示出疗效。
