Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine
Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine
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DOI：
10.1182/blood-2010-06-289280
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发表时间：
2011-01-13
期刊：
影响因子：
20.3
通讯作者：
Fenaux, Pierre
中科院分区：
文献类型：
--
作者：
Itzykson, Raphael;Thepot, Sylvain;Fenaux, Pierre
使用阿扎胞苷（AZA）治疗的高危骨髓增生异常综合征患者的反应和生存的预后因素仍然很大程度上未知。 282 名连续高风险或中度 2 风险骨髓增生异常综合征患者在一项富有同情心、以患者命名的计划中接受了 AZA 治疗。 4% 的诊断为 RA/RARS/RCMD，20% 的诊断为 RAEB-1，54% 的诊断为 RAEB-2，22% 的诊断为 RAEB-t（AML，其中 21%-30% 为骨髓原始细胞）。细胞遗传学风险为良好（31%）、中等（17%）、差（47%）。患者接受 AZA 治疗的中位数为 6 个周期 (1-52)。既往低剂量阿糖胞苷治疗 (P = .009)、骨髓母细胞 > 15% (P = .004) 和异常核型 (P = .03) 独立预测较低的缓解率。复杂核型预测反应较短 (P = .0003)。体能状态≥2、中危和低危细胞遗传学、循环母细胞的存在以及红细胞输注依赖性≥4单位/8周（所有P<10(-4)）独立预测较差的总生存期（OS）。基于这些因素的预后评分区分了 3 个未达到中位 OS 的风险组，分别为 15.0 个月和 6.1 个月 (P < 10(-4))。该预后评分在 AZA-001 试验中接受 AZA 治疗的一组独立患者中得到验证 (P = .003)。未获得完全或部分缓解的患者的血液学改善与 OS 改善相关 (P < 10(-4))。总之，常规测试可以识别接受 AZA 治疗后具有不同预后的患者亚组。 （血。2011；117（2）：403-411）
Prognostic factors for response and survival in higher-risk myelodysplastic syndrome patients treated with azacitidine (AZA) remain largely unknown. Two hundred eighty-two consecutive high or inter-mediate-2 risk myelodysplastic syndrome patients received AZA in a compassionate, patient-named program. Diagnosis was RA/RARS/RCMD in 4%, RAEB-1 in 20%, RAEB-2 in 54%, and RAEB-t (AML with 21%-30% marrow blasts) in 22%. Cytogenetic risk was good in 31%, intermediate in 17%, and poor in 47%. Patients received AZA for a median of 6 cycles (1-52). Previous low-dose cytosine arabinoside treatment (P = .009), bone marrow blasts > 15% (P = .004), and abnormal karyotype (P = .03) independently predicted lower response rates. Complex karyotype predicted shorter responses (P = .0003). Performance status >= 2, intermediate-and poor-risk cytogenetics, presence of circulating blasts, and red blood cell transfusion dependency >= 4 units/8 weeks (all P < 10(-4)) independently predicted poorer overall survival (OS). A prognostic score based on those factors discriminated 3 risk groups with median OS not reached, 15.0 and 6.1 months, respectively (P < 10(-4)). This prognostic score was validated in an independent set of patients receiving AZA in the AZA-001 trial (P = .003). Achievement of hematological improvement in patients who did not obtain complete or partial remission was associated with improved OS (P < 10(-4)). In conclusion, routine tests can identify subgroups of patients with distinct prognosis with AZA treatment. (Blood. 2011; 117(2):403-411)