Hairy cell leukemia (HCL) is a rare lymphoproliferative disease with an excellent prognosis after treatment with cladribine (2CDA), although relapse may occur during follow-up. The aim of the study is to review the efficacy, safety, long-term remission rate, and overall survival (OS) in those patients who received 2CDA as first-line treatment. We retrospectively reviewed data of HCL patients treated with 2CDA between March 1991 and May 2019 at 18 Italian Hematological centers: 513 patients were evaluable for study purpose. The median age was 54 years (range 24–88) and ECOG was 0 in 84.9% of cases. A total of 330 (64.3%) patients received 2CDA intravenously and 183 (35.7%) subcutaneously. ORR was 91.8%: CR was obtained in 335 patients (65.3%), PR in 96 (18.7%), and hematological response in 40 (7.8%) patients; in 42 (8.2%) no response was observed. Hemoglobin value (p = 0.044), frequency of circulating hairy cells (p = 0.039), recovery of absolute neutrophil count (p = 0.006), and normalization of spleen (p ≤ 0.001) were associated with CR compared to PR in univariable analysis. At a median follow-up of 6.83 years (range 0.04–28.52), the median time to relapse was 12.2 years. A significant difference in duration of response was identified between patients that obtained a CR and PR (19.4 years versus 4.8 years, p < 0.0001). Non-hematological grade 3 or higher early toxicity was reported in 103 (20.1%) patients. Median OS was not reached: 95.3%, 92.4%, and 81.8% of patients were estimated to be alive at 5, 10, and 15 years, respectively. Forty-nine patients died (9.5%), following an infection in 14 cases (2.7%), natural causes in 14 (2.7%), cardiovascular events in 13 (2.5%), a second neoplasm in 6 (1.2%), and progression of HCL in 2 cases (0.4%). Following treatment of HCL with 2CDA, 80% of patients are estimated to be alive 15 years after diagnosis.
毛细胞白血病（HCL）是一种罕见的淋巴增殖性疾病，经克拉屈滨（2CDA）治疗后预后良好，尽管随访期间可能出现复发。本研究的目的是评估接受2CDA作为一线治疗患者的疗效、安全性、长期缓解率及总生存期（OS）。我们回顾性分析了1991年3月至2019年5月期间在18个意大利血液学中心接受2CDA治疗的HCL患者数据：共有513例患者可进行评估。中位年龄为54岁（范围24-88岁），84.9%的病例ECOG评分为0。总共330例（64.3%）患者接受静脉注射2CDA，183例（35.7%）接受皮下注射。总缓解率（ORR）为91.8%：335例（65.3%）患者达到完全缓解（CR），96例（18.7%）达到部分缓解（PR），40例（7.8%）达到血液学缓解；42例（8.2%）未观察到缓解。单变量分析显示，与PR相比，CR与血红蛋白值（p=0.044）、循环毛细胞频率（p=0.039）、绝对中性粒细胞计数恢复（p=0.006）及脾脏正常化（p≤0.001）相关。中位随访时间为6.83年（范围0.04-28.52），中位复发时间为12.2年。达到CR和PR的患者在缓解持续时间上存在显著差异（19.4年 vs 4.8年，p<0.0001）。103例（20.1%）患者报告了非血液学3级或以上早期毒性。中位OS未达到：估计5年、10年和15年生存率分别为95.3%、92.4%和81.8%。49例患者死亡（9.5%），其中14例（2.7%）因感染，14例（2.7%）因自然原因，13例（2.5%）因心血管事件，6例（1.2%）因第二肿瘤，2例（0.4%）因HCL进展。经2CDA治疗后，估计80%的患者在诊断后15年仍存活。
肿瘤(癌症)患者之家