Harvard Medical School and CLL Center, Dana-Farber Cancer Institute, Boston, MA, USA.
Hematology Am Soc Hematol Educ Program. 2011;2011:110-8. doi: 10.1182/asheducation-2011.1.110.
Despite the widespread use of highly effective chemoimmunotherapy (CIT), fludarabine-refractory chronic lymphocytic leukemia (CLL) remains a challenging clinical problem associated with poor overall survival (OS). The traditional definition, which includes those patients with no response or relapse within 6 months of fludarabine, is evolving with the recognition that even patients with longer remissions of up to several years after CIT have poor subsequent treatment response and survival. Approved therapeutic options for these patients remain limited, and the goal of therapy for physically fit patients is often to achieve adequate cytoreduction to proceed to allogeneic stem cell transplantation (alloSCT). Fortunately, several novel targeted therapeutics in clinical trials hold promise of significant benefit for this patient population. This review discusses the activity of available and novel therapeutics in fludarabine-refractory or fludarabine-resistant CLL as well as recently updated data on alloSCT in CLL.
尽管广泛使用了高效的化疗免疫疗法（CIT），但氟达拉滨难治性慢性淋巴细胞白血病（CLL）仍然是一个具有挑战性的临床问题，与总体生存率（OS）差相关。传统的定义包括那些在氟达拉滨治疗后 6 个月内无反应或复发的患者，随着人们认识到即使是在 CIT 后长达数年的缓解期患者，其后续治疗反应和生存也较差，该定义正在不断发展。对于这些患者，批准的治疗选择仍然有限，对于身体状况良好的患者，治疗的目标通常是实现足够的细胞减少，以便进行异基因干细胞移植（alloSCT）。幸运的是，临床试验中的几种新型靶向治疗药物为这一患者群体带来了显著获益的希望。本文讨论了氟达拉滨难治性或氟达拉滨耐药性 CLL 中现有和新型治疗药物的活性，以及 CLL 中 alloSCT 的最新数据。
