Appelbaum Frederick R, Kopecky Kenneth J, Tallman Martin S, Slovak Marilyn L, Gundacker Holly M, Kim Haesook T, Dewald Gordon W, Kantarjian Hagop M, Pierce Sherry R, Estey Elihu H
Southwest Oncology Group Statistical Center, Seattle, WA, USA.
Br J Haematol. 2006 Oct;135(2):165-73. doi: 10.1111/j.1365-2141.2006.06276.x. Epub 2006 Aug 25.
To better understand the spectrum of adult acute myeloid leukaemia (AML) associated with core binding factor (CBF) translocations, 370 patients with newly diagnosed CBF-associated AML were analysed. Patients' age ranged from 16-83 years (median 39 years) with a slight male predominance (55%); 53% had inv(16); 47% had t(8;21). Patients with t(8;21) tended to be younger (P = 0.056), have lower peripheral blood white cell counts (P < 0.0001) and were more likely to have additional cytogenetic abnormalities (P < 0.0001). Loss of sex chromosome, del(9q) and complex abnormalities were more common among patients with t(8;21), while +22 and +21 were more common with inv(16). Overall, 87% [95% confidence interval (CI) 83-90%] of patients achieved complete response (CR) with no difference between t(8;21) and inv(16); however, the CR rate was lower in older patients due to increased resistant disease and early deaths. Ten-year overall survival (OS) was 44% (95% CI 39-50%) and, in multivariate analysis, was shorter with increasing age (P < 0.0001), increased peripheral blast percentage (P = 0.0006), in patients with complex cytogenetic abnormalities in addition to the CBF translocation (P = 0.021), and in patients with t(8;21) (P = 0.025). OS was superior in patients who received regimens with high-dose cytarabine, a combination of fludarabine and intermediate-dose cytarabine, or haematopoietic cell transplantation.
为了更好地了解与核心结合因子（CBF）易位相关的成人急性髓系白血病（AML）的谱系，对370例新诊断的CBF相关AML患者进行了分析。患者年龄在16至83岁之间（中位年龄39岁），男性略占优势（55%）；53%的患者有inv(16)；47%的患者有t(8;21)。t(8;21)患者往往更年轻（P = 0.056），外周血白细胞计数更低（P < 0.0001），并且更有可能有其他细胞遗传学异常（P < 0.0001）。性染色体丢失、del(9q)和复杂异常在t(8;21)患者中更常见，而+22和+21在inv(16)患者中更常见。总体而言，87%[95%置信区间（CI）83 - 90%]的患者达到完全缓解（CR），t(8;21)和inv(16)之间无差异；然而，由于耐药疾病增加和早期死亡，老年患者的CR率较低。十年总生存率（OS）为44%（95% CI 39 - 50%），在多变量分析中，随着年龄增加（P < 0.0001）、外周原始细胞百分比增加（P = 0.0006）、除CBF易位外还有复杂细胞遗传学异常的患者（P = 0.021）以及t(8;21)患者（P = 0.025），OS更短。接受高剂量阿糖胞苷方案、氟达拉滨与中剂量阿糖胞苷联合方案或造血细胞移植的患者OS更好。
