Eketorp Sylvan Sandra, Hansson Lotta, Karlsson Claes, Norin Stefan, Lundin Jeanette, Osterborg Anders
Departments of Hematology and Oncology, Karolinska University Hospital , Stockholm , Sweden.
Leuk Lymphoma. 2014 Aug;55(8):1774-80. doi: 10.3109/10428194.2013.827786. Epub 2014 Feb 4.
Patients with fludarabine-refractory (FR) chronic lymphocytic leukemia (CLL) receive novel agents in pivotal, non-randomised phase-2 trials. Understanding outcome of FR-CLL in health-care may provide important contextual information. Records from 1301 patients (Stockholm-Cancer-Registry 1991-2010) identified 92 FR-patients; bulky lymph-nodes (BFR-group), double-refractory (DR-group), or Others'-group for outcome-analysis. Median age was 69 years 67% had Rai-stage III/IV with median 3 prior therapies. Overall response-rate was 20%; significantly lower in BFR (8%, p = 0.01) and DR (20%, p = 0.01) than in 'Others' (31%). Time-to-treatment-failure (months) was significantly longer in 'Others' (9.2) than in BFR/DR (5.3/4.4) (p < 0.01) and significantly longer (p < 0.05) in antibody-treated patients (9.1) compared to other regimens (5.2). Early-death occurred in 5%, ≥ grade III-infections in 20%. Median overall-survival (OS) was 18 months; 29 in BFR vs. 13 in DR (p = 0.054). Male sex was the only prognostic factor on OS (p = 0.01, HR 2.2, multivariate-Cox-regression). Our results, without external referrals, facilitate interpretation of non-randomised trials/novel drugs in advanced-stage-CLL.
氟达拉滨难治性（FR）慢性淋巴细胞白血病（CLL）患者在关键的非随机2期试验中接受新型药物治疗。了解医疗保健中FR-CLL的治疗结果可能会提供重要的背景信息。对1301例患者（斯德哥尔摩癌症登记处1991 - 2010年）的记录进行分析，确定了92例FR患者，分为有巨大淋巴结组（BFR组）、双重难治组（DR组）或其他组进行预后分析。中位年龄为69岁，67%的患者处于Rai分期III/IV期，之前的中位治疗次数为3次。总缓解率为20%；BFR组（8%，p = 0.01）和DR组（20%，p = 0.01）的缓解率显著低于“其他组”（31%）。“其他组”的治疗失败时间（月）（9.2）显著长于BFR/DR组（5.3/4.4）（p < 0.01），与其他治疗方案（5.2）相比，接受抗体治疗的患者治疗失败时间显著更长（p < 0.05）（9.1）。5%的患者早期死亡，20%的患者发生≥III级感染。中位总生存期（OS）为18个月；BFR组为29个月，DR组为13个月（p = 0.054）。男性是总生存期的唯一预后因素（p = 0.01，HR 2.2，多变量Cox回归）。我们的研究结果无需外部转诊，有助于解释晚期CLL的非随机试验/新型药物。
