Hehlmann R
III. Medizinische Klinik, Klinikum Mannheim, Universität Heidelberg, Mannheim.
Ther Umsch. 1996 Feb;53(2):82-7.
The chronic myelogenous leukemia [CML] is a clonal disease of hematopoietic stem cells with unknown etiology. The incidence is around 2/100,000/year, the median age at diagnosis about 47 years. The course of CML is characterized by a chronic phase with few symptoms and good therapeutic response of about 4 to 5 years duration and by transition to a prognostically unfavourable blast phase of about 3 months duration. Therapy of choice, at present, is early allogenous bone marrow transplantation [BMT], which is curative in 40 to 80% of transplanted cases. In patients below 55 years, a donor search should be started at the earliest possible time after diagnosis. Drug therapy of choice are interferon alpha [IFN] and hydroxyurea, which are both superior to busulfan with regard to duration of chronic phase and survival. Complete cytogenetic remissions are observed in 5 to 9% of IFN-treated patients in randomized studies, but virtually all remain positive for bcr/abl by PCR. Whether and in how far IFN is superior to hydroxyurea appears, at least in part, to depend on the treatment intensity with hydroxyurea and on patients characteristics. In analyzing median survival times, the risk profiles of the patients have to be considered. In the future, intensive chemotherapy with or without autografting might play an important role in the therapy of chronic-phase CML. Forthcoming trials have to consider both, conventional and new experimental treatment modalities. An example is the treatment strategy of the ongoing randomized study of the German CML Study Group which compares allogenous BMT with the best available drug therapy and, in addition, analyses the influence of intensified drug therapy on survival.
慢性粒细胞白血病(CML)是一种造血干细胞的克隆性疾病,病因不明。发病率约为每年2/100,000,诊断时的中位年龄约为47岁。CML的病程特点是慢性期症状较少,治疗反应良好,持续约4至5年,然后转变为预后不良的急变期,持续约3个月。目前的首选治疗方法是早期异基因骨髓移植(BMT),在40%至80%的移植病例中可治愈。对于55岁以下的患者,诊断后应尽早开始寻找供体。首选的药物治疗是干扰素α(IFN)和羟基脲,在慢性期持续时间和生存率方面,两者均优于白消安。在随机研究中,5%至9%接受IFN治疗的患者可观察到完全细胞遗传学缓解,但通过聚合酶链反应(PCR)检测,几乎所有患者的bcr/abl仍为阳性。IFN是否优于羟基脲以及在多大程度上优于羟基脲,至少部分取决于羟基脲的治疗强度和患者特征。在分析中位生存时间时,必须考虑患者的风险特征。未来,强化化疗(无论是否进行自体移植)可能在慢性期CML的治疗中发挥重要作用。即将进行的试验必须同时考虑传统和新的实验性治疗方式。德国CML研究组正在进行的随机研究的治疗策略就是一个例子,该研究比较了异基因BMT与最佳可用药物治疗,并分析了强化药物治疗对生存的影响。