Wang Z Y, Chen Z, Huang W, Li X S, Lu J X, Huang L A, Zhang F Q, Gu L J, Ouyang R R, Chen S J
Shanghai Institute of Hematology, Rui-jin Hospital, Shanghai Second Medical University, PR China.
Blood Cells. 1993;19(3):633-41; discussion 642-7.
A large number of acute promyelocytic leukemia (APL) patients, treated with all-trans retinoic acid (ATRA) and chemotherapy, were studied. The results of the studies are as follows: (1) Among 65 patients investigated for the postremissional therapy, the 5-year survival probabilities were 0.20 +/- 0.13 (mean +/- SE) in the group treated with ATRA alone, 0.47 + 0.10 (mean +/- SE) in the group using chemotherapy alone and 0.42 +/- 0.09 (mean +/- SE) in the group treated with chemotherapy and ATRA. (2) The main severe adverse effects in the ATRA treatment include retinoic acid syndrome, renal failure, and thrombosis. These sequelae were observed more frequently in cases with persistent, marked elevation of white blood cell count without significant maturation of leukemic promyelocytes. (3) APL is not a homogeneous disease in that among 50 patients studied at the molecular level, although a PML-RARA fusion gene was detected in 45 cases, one had a variant translocation t(11;17) bearing fusion gene PLZF-RARA, one presented no obvious structural alteration of the PML gene while the RARA gene was rearranged, and three patients had no rearrangement of either PML or RARA genes. (4) Using RT/PCR to detect minimal residual disease, we found positive rates of 22%, 18.4%, and 11.5%, respectively, 12, 24, and 36 months after CR. This observation justifies the use of chemotherapy for at least 3 years after CR induced by ATRA. (5) It seems likely that the fusion gene PML-RARA plays an important role in APL leukemogenesis and in its response to the ATRA treatment.
对大量接受全反式维甲酸（ATRA）和化疗的急性早幼粒细胞白血病（APL）患者进行了研究。研究结果如下：（1）在65例接受缓解后治疗的患者中，单纯接受ATRA治疗组的5年生存概率为0.20±0.13（均值±标准误），单纯化疗组为0.47±0.10（均值±标准误），化疗联合ATRA治疗组为0.42±0.09（均值±标准误）。（2）ATRA治疗的主要严重不良反应包括维甲酸综合征、肾衰竭和血栓形成。在白血病早幼粒细胞无明显成熟且白细胞计数持续显著升高的病例中，这些后遗症更为常见。（3）APL并非同质疾病，在50例进行分子水平研究的患者中，虽然45例检测到PML-RARA融合基因，但1例有变异易位t(11;17)携带融合基因PLZF-RARA，1例PML基因无明显结构改变而RARA基因重排，3例患者PML和RARA基因均无重排。（4）使用RT/PCR检测微小残留病，我们发现在完全缓解（CR）后12、24和36个月时的阳性率分别为22%、18.4%和11.5%。这一观察结果证明在ATRA诱导CR后至少进行3年化疗是合理的。（5）融合基因PML-RARA似乎在APL白血病发生及其对ATRA治疗的反应中起重要作用。
