Molica S, De Rossi G, Luciani M, Levato D
Hematology Division, Regional Hospital A. Pugliese, Catanzaro, Italy.
Haematologica. 1995 Mar-Apr;80(2):176-93.
In the past few decades important progress has been made in the understanding of chronic lymphocytic leukemia (CLL). Indeed, systematic studies of natural history and prognostic factors have made it possible to predict the outcome of disease. Although clinical stage (i.e. Rai and Binet stages) is the strongest predictor of survival, additional prognostic parameters, including patterns of bone marrow (BM) infiltration, lymphocyte doubling time (LDT), immunophenotype and cytogenetics, have now been identified. Furthermore, criteria of smoldering CLL (i.e. stage A, low lymphocyte count, non-diffuse BM histology, relatively high hemoglobin level, LDT > 12 months) allow identification of a subgroup of patients with indolent course and good prognosis for whom treatment should be delayed, unless progression occurs. Recent meta-analysis of clinical trials has demonstrated no survival advantage for immediate versus referred treatment in low clinical stages. The same considerations apply when comparing combination versus single-drug regimens. Purine analogues like fludarabine, 2'-chlorodeoxyadenosine and 2'-deoxycoformicin are active in CLL. Data on these drugs come from uncontrolled clinical trials; randomized studies are in progress. In addition, some issues concerning the relationship between response and survival, cross-resistance between purine analogues and eradication of the CLL clone, remain still unresolved. There are also increasing data on bone marrow transplants in CLL, although the high treatment-related mortality suggests that this procedure may have some benefit only in selected refractory young CLL patients with adverse features. This review will focus on recent progress in the prognosis and therapy of CLL. Issues that remain controversial will be a matter of discussion.
在过去几十年里,人们对慢性淋巴细胞白血病(CLL)的认识取得了重要进展。的确,对自然病程和预后因素的系统研究使得预测疾病结局成为可能。尽管临床分期(即Rai分期和Binet分期)是生存的最强预测因素,但现在已经确定了其他预后参数,包括骨髓(BM)浸润模式、淋巴细胞倍增时间(LDT)、免疫表型和细胞遗传学。此外,惰性CLL的标准(即A期、低淋巴细胞计数、非弥漫性BM组织学、相对较高的血红蛋白水平、LDT>12个月)允许识别出一组病程惰性且预后良好的患者亚组,除非疾病进展,否则应推迟对其进行治疗。最近对临床试验的荟萃分析表明,在低临床分期中,立即治疗与延迟治疗相比并无生存优势。在比较联合治疗方案与单药治疗方案时,同样的考虑也适用。嘌呤类似物如氟达拉滨、2'-氯脱氧腺苷和2'-脱氧助间型霉素在CLL中具有活性。关于这些药物的数据来自非对照临床试验;随机研究正在进行中。此外,一些关于缓解与生存之间的关系、嘌呤类似物之间的交叉耐药性以及CLL克隆清除的问题仍未得到解决。关于CLL骨髓移植的数据也越来越多,尽管与治疗相关的高死亡率表明,这种治疗方法可能仅对某些具有不良特征的难治性年轻CLL患者有益。本综述将聚焦于CLL预后和治疗的最新进展。仍存在争议的问题将是讨论的主题。