Division of Hematology, Mayo Clinic, Rochester, Minnesota.
Am J Hematol. 2015 May;90(5):411-6. doi: 10.1002/ajh.23962. Epub 2015 Apr 1.
Among 274 patients with chronic myelomonocytic leukemia (CMML) and followed for a median of 17.1 months, blast transformation (BT) occurred in 36 (13%). On multivariable analysis, risk factors for BT were presence of circulating blasts (HR 5.7; 95% CI 2.8-11.9) and female gender (HR 2.6; 95% CI 1.3-5.1); the results remained unchanged when analysis was restricted to CMML-1. ASXL1/SRSF2/SF3B1/U2AF1/SETBP1 mutational frequencies were not significantly different between time of CMML diagnosis and BT. Median survival post-BT was 4.7 months (5-year survival 6%) and better with allogeneic stem cell transplant (SCT) (14.3 months vs. 4.3 months for chemotherapy vs. 0.9 months for supportive care; P = 0.03). Neither karyotype nor mutational status was independently associated with risk of BT or post-BT survival. We conclude that female patients with CMML and those with circulating blasts are at a higher risk of BT. Post-BT survival is dismal and our observations suggest consideration of allogeneic SCT prior to BT.
在 274 例慢性粒单核细胞白血病 (CMML) 患者中，中位随访时间为 17.1 个月，有 36 例（13%）发生了白血病转化（BT）。多变量分析显示，BT 的危险因素包括循环原始细胞的存在（HR 5.7；95%CI 2.8-11.9）和女性（HR 2.6；95%CI 1.3-5.1）；当分析仅限于 CMML-1 时，结果仍然不变。ASXL1/SRSF2/SF3B1/U2AF1/SETBP1 突变频率在 CMML 诊断和 BT 之间没有显著差异。BT 后的中位生存时间为 4.7 个月（5 年生存率为 6%），异基因造血干细胞移植（SCT）的生存时间更好（14.3 个月 vs. 化疗的 4.3 个月 vs. 支持治疗的 0.9 个月；P = 0.03）。核型和突变状态均与 BT 风险或 BT 后生存无关。我们得出结论，女性 CMML 患者和循环原始细胞患者发生 BT 的风险更高。BT 后的生存时间很差，我们的观察结果表明，在 BT 之前应考虑进行异基因 SCT。
