Nguyen Stéphanie, Leblanc Thierry, Fenaux Pierre, Witz Francis, Blaise Didier, Pigneux Arnaud, Thomas Xavier, Rigal-Huguet Françoise, Lioure Bruno, Auvrignon Anne, Fière Denis, Reiffers Josy, Castaigne Sylvie, Leverger Guy, Harousseau Jean-Luc, Socié Gérard, Dombret Hervé
Department of Hematology, Hôpital Saint-Louis, Paris, France.
Blood. 2002 May 15;99(10):3517-23. doi: 10.1182/blood.v99.10.3517.
While the t(8;21) translocation is one of the most recurrent chromosomal abnormalities in acute myeloid leukemia, prognostic studies have been hampered by the relatively few number of patients reported. We thus performed a large retrospective study in 161 adults and children with t(8;21) acute myeloid leukemia, all prospectively enrolled in 6 different trials conducted in France between 1987 and 1998 (median follow-up 4.9 years). Prognostic studies were performed in the 154 patients who achieved a complete remission. Individual data were registered, including sex, age, blood and marrow counts, extramedullary disease, and cytogenetics. The value of allogeneic stem cell transplantation versus chemotherapy as postremission therapy was evaluated according to the intent-to-treat principle. Estimated 5-year disease-free survival (DFS) and overall survival were 52% and 59%, respectively. Outcome was not significantly better in patients from the stem cell transplantation group (estimated 5-year DFS and survival, 56% vs 52% and 67% vs 57%; P =.55 and.64, respectively). White blood cell count (WBC) was the only identified prognostic factor. To further take into account the spontaneous differentiation potential of the leukemic clone, a WBC index was derived as the product of WBC by the ratio of marrow blast. This WBC index was a more powerful factor than the original WBC, allowing us to distinguish 3 subgroups of patients with different outcomes (low index, < 2.5; intermediate index, 2.5-20; high index, 20 or more). In multivariate analysis, the WBC index was the only prognostic factor for DFS (P =.003), complete remission duration (P =.002), and overall survival (P =.04).
虽然t(8;21)易位是急性髓系白血病中最常见的染色体异常之一，但由于报告的患者数量相对较少，预后研究受到了阻碍。因此，我们对161例患有t(8;21)急性髓系白血病的成人和儿童进行了一项大型回顾性研究，所有患者均前瞻性纳入了1987年至1998年在法国进行的6项不同试验（中位随访4.9年）。对154例实现完全缓解的患者进行了预后研究。记录了个体数据，包括性别、年龄、血液和骨髓计数、髓外疾病和细胞遗传学。根据意向性治疗原则评估了异基因干细胞移植与化疗作为缓解后治疗的价值。估计5年无病生存率（DFS）和总生存率分别为52%和59%。干细胞移植组患者的结局并无显著改善（估计5年DFS和生存率分别为56%对52%和67%对57%；P分别为0.55和0.64）。白细胞计数（WBC）是唯一确定的预后因素。为了进一步考虑白血病克隆的自发分化潜能，得出了一个WBC指数，即WBC与骨髓原始细胞比例的乘积。该WBC指数比原始WBC更具影响力，使我们能够区分出结局不同的3个患者亚组（低指数，<2.5；中等指数，2.5 - 20；高指数，20或更高）。在多变量分析中，WBC指数是DFS（P = 0.003）、完全缓解持续时间（P = 0.002）和总生存率（P = 0.04）的唯一预后因素。
