Service d'Hématologie Clinique, Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris (AP-HP), and Université Paris 13, Bobigny, France.
Am J Hematol. 2014 Apr;89(4):410-6. doi: 10.1002/ajh.23654. Epub 2014 Feb 6.
Limited data are available on azacitidine (AZA) treatment and its prognostic factors in acute myeloid leukemia (AML). One hundred and forty-nine previously untreated AML patients considered ineligible for intensive chemotherapy received AZA in a compassionate patient-named program. AML diagnosis was de novo, post-myelodysplastic syndromes (MDS), post-MPN, and therapy-related AML in 51, 55, 13, and 30 patients, respectively. Median age was 74 years, median white blood cell count (WBC) was 3.2 × 10⁹ /L and 58% of the patients had ≥ 30% marrow blasts. Cytogenetics was adverse in 60 patients. Patients received AZA for a median of five cycles (range 1-31). Response rate (including complete remission/CR with incomplete recovery/partial remission) was 27.5% after a median of three cycles (initial response), and 33% at any time (best response). Only adverse cytogenetics predicted poorer response. Median overall survival (OS) was 9.4 months. Two-year OS was 51% in responders and 10% in non-responders (P<0.0001). Adverse cytogenetics, WBC >15 × 10⁹ /L and ECOG-PS ≥ 2 predicted poorer OS, while age and marrow blast percentage had no impact. Using MDS IWG 2006 response criteria, among patients with stable disease, those with hematological improvement had no significant survival benefit in a 7 months landmark analysis. Outcomes observed in this high-risk AML population treated with AZA deserve comparison with those of patients treated intensively in prospective studies.
关于阿扎胞苷（AZA）治疗急性髓系白血病（AML）及其预后因素的数据有限。149 例先前未经治疗且不符合强化化疗条件的 AML 患者在一项同情用药患者命名计划中接受 AZA 治疗。AML 诊断分别为初发、骨髓增生异常综合征（MDS）后、骨髓增生异常相关髓系肿瘤（MPN）后和治疗相关 AML，分别为 51、55、13 和 30 例患者。中位年龄为 74 岁，中位白细胞计数（WBC）为 3.2×10⁹/L，58%的患者骨髓原始细胞比例≥30%。60 例患者的细胞遗传学为不良。患者接受 AZA 治疗的中位数为五个周期（范围为 1-31）。中位三个周期（初始反应）后缓解率（包括完全缓解/CR 不完全缓解/部分缓解）为 27.5%，任何时间的缓解率为 33%。仅不良细胞遗传学预测反应较差。中位总生存期（OS）为 9.4 个月。在应答者中，两年 OS 为 51%，在无应答者中为 10%（P<0.0001）。不良细胞遗传学、WBC>15×10⁹/L 和 ECOG-PS≥2 预测 OS 较差，而年龄和骨髓原始细胞比例对 OS 无影响。使用 MDS IWG 2006 反应标准，在疾病稳定的患者中，在 7 个月的里程碑分析中，那些有血液学改善的患者在生存方面没有显著获益。在这项接受 AZA 治疗的高危 AML 人群中观察到的结果值得与接受前瞻性研究强化治疗的患者进行比较。
