Istituto di Ematologia, Università Cattolica del Sacro Cuore, 00168, Roma, Italy.
J Hematol Oncol. 2012 Aug 1;5:44. doi: 10.1186/1756-8722-5-44.
Therapy-related myeloid neoplasms (t-MN), including myelodysplastic syndromes and acute myeloid leukemia (t-MDS and t-AML) are associated to clinical and biologic unfavorable prognostic features, including high levels of DNA methylation.
We retrospectively evaluated 50 t-MN patients (34 MDS and 16 AML) selected among all patients receiving azacitidine (AZA) at 10 Italian Hematology Centers. Patients had developed a t-MN at a median of 6.5 years (range 1.7- 29) after treatment of the primary tumor (hematological neoplasm, 27 patients; solid tumor, 23 patients).
The overall response rate was 42% (complete remission: 10 patients, partial remission: 2 and hematological improvement: 8 patients) and was obtained after a median of 3 cycles (range 1-6). Median overall survival (OS) was 21 months (range 1-53.6+) from AZA start. OS was significantly better in patients with less than 20% blasts, in normal karyotype t-AML and when AZA was used as front-line treatment. This was confirmed by the multivariate analysis.
This study reports efficacy of AZA in the largest series of therapy-related MN patients treated with 5-AZA. Our data show that blasts and karyotype maintain their important prognostic role in t-MN also in the azacitidine era.
治疗相关髓系肿瘤（t-MN），包括骨髓增生异常综合征和急性髓系白血病（t-MDS 和 t-AML）与临床和生物学不良预后特征相关，包括高水平的 DNA 甲基化。
我们回顾性评估了在 10 家意大利血液中心接受阿扎胞苷（AZA）治疗的所有患者中选择的 50 例 t-MN 患者（34 例 MDS 和 16 例 AML）。患者在治疗原发性肿瘤（血液系统肿瘤，27 例；实体肿瘤，23 例）后中位 6.5 年（范围 1.7-29）时发展为 t-MN。
总缓解率为 42%（完全缓解：10 例，部分缓解：2 例，血液学改善：8 例），并在中位 3 个周期（范围 1-6）后获得。从 AZA 开始的中位总生存期（OS）为 21 个月（范围 1-53.6+）。 blast<20%、核型正常的 t-AML 患者以及 AZA 作为一线治疗时，OS 明显更好。这在多变量分析中得到了证实。
这项研究报告了 AZA 在接受 5-AZA 治疗的最大系列治疗相关 MN 患者中的疗效。我们的数据表明，在阿扎胞苷时代，blast 和核型在 t-MN 中仍然具有重要的预后作用。
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