Madanat Yazan F, Rybicki Lisa, Radivoyevitch Tomas, Jagadeesh Deepa, Dean Robert, Pohlman Brad, Kalaycio Matt, Sekeres Mikkael A, Smith Mitchell R, Hill Brian T
Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH.
Quantitative Health Sciences, Cleveland Clinic, Cleveland, OH.
Clin Lymphoma Myeloma Leuk. 2017 Dec;17(12):857-862. doi: 10.1016/j.clml.2017.07.003. Epub 2017 Jul 14.
Hairy cell leukemia (HCL) is a rare hematologic malignancy with high response rates and long progression-free survival (PFS) after treatment with purine nucleoside analogs (PNAs; Pentostatin/Cladribine). However, treatment is not curative, and subsequent treatment at relapse is often required. Rechallenge with a purine analog is commonly implemented despite limited data regarding the efficacy of this approach. We retrospectively analyzed 61 consecutive patients with HCL diagnosed between 1995 and 2013 at Cleveland Clinic. Median follow-up was 72 months (3-193). Cladribine as first-line therapy was administered to 59 patients (97%). Overall response rate (ORR) was 97%, with 78% of patients achieving complete remission (CR). PFS after response was significantly improved for patients who achieved CR compared with those with a partial remission (PR) (5-year PFS 71% vs. 39%, respectively [P = .004]). Of the 19 patients who relapsed, 12 received PNAs as second-line treatment with an ORR (83%) comparable to what these patients had with first-line treatment (ORR 92%). Overall survival of all 61 patients was excellent and superior to that of age-, sex-, and race-matched controls from the general population, possibly due to selection bias. In an analysis of a larger cohort of unselected patients in the Surveillance, Epidemiology, and End Results (SEER) database, we found that mortality rates for patients with HCL were similar to those of the general population approximately 5 years after diagnosis. These data confirm the excellent prognosis for patients with HCL after first- and second-line PNA therapy.
毛细胞白血病（HCL）是一种罕见的血液系统恶性肿瘤，经嘌呤核苷类似物（PNA；喷司他丁/克拉屈滨）治疗后缓解率高且无进展生存期（PFS）长。然而，该治疗并非根治性的，复发后通常需要后续治疗。尽管关于这种方法疗效的数据有限，但嘌呤类似物再激发治疗仍普遍实施。我们回顾性分析了1995年至2013年在克利夫兰诊所确诊的61例连续的HCL患者。中位随访时间为72个月（3 - 193个月）。59例患者（97%）接受克拉屈滨作为一线治疗。总缓解率（ORR）为97%，78%的患者达到完全缓解（CR）。与部分缓解（PR）的患者相比，达到CR的患者缓解后的PFS显著改善（5年PFS分别为71%和39%[P = .004]）。19例复发患者中，12例接受PNA作为二线治疗，ORR（83%）与这些患者一线治疗时的ORR（92%）相当。61例患者的总生存期良好，优于来自一般人群的年龄、性别和种族匹配的对照组，这可能是由于选择偏倚。在对监测、流行病学和最终结果（SEER）数据库中一个更大的未选择患者队列的分析中，我们发现HCL患者的死亡率在诊断后约5年与一般人群相似。这些数据证实了HCL患者经一线和二线PNA治疗后的良好预后。
