Benz Rudolf, Arn Kornelius, Andres Martin, Pabst Thomas, Baumann Michael, Novak Urban, Hitz Felicitas, Hess Urs, Zenhaeusern Reinhard, Chalandon Yves, Mey Ulrich, Blum Sabine, Rauch Daniel, O'Meara Stern Alix, Cantoni Nathan, Bargetzi Mario, Bianchi-Papina Elena, Rossi Davide, Passweg Jakob, Lohri Andreas, Berardi Simona, Li Qiyu, Feller Anita, Stussi Georg
Division of Hematology and Oncology, Kantonsspital Muensterlingen, Muensterlingen, Switzerland.
Department of Hematology, Kantonsspital Luzern, Luzern, Switzerland.
Blood Adv. 2020 Aug 11;4(15):3699-3707. doi: 10.1182/bloodadvances.2020002160.
Hairy cell leukemia (HCL) remains an incurable disease. However, first-line treatment with either intravenous or subcutaneous cladribine generally leads to long-lasting remissions. Although there are excellent long-term data for intravenous application, similar data regarding subcutaneous administration are lacking. We therefore analyzed the long-term outcome of 3 prospective multicenter clinical trials on subcutaneous cladribine performed by the Swiss Group for Clinical Cancer Research (SAKK), which recruited 221 patients with classical HCL between 1993 and 2005. Median overall survival from start of treatment was not reached. Pretreatment anemia, higher Eastern Cooperative Oncology Group score, and higher age were associated with poorer overall survival in multivariable analysis, whereas early progression at 24 and 36 months had no significant impact on overall survival. Second-line treatment was necessary in 53 (23.7%) patients after a median of 5 (range, 0.2-20.4) years, and first retreatment was mainly monotherapy with cladribine (66%) or rituximab (15.1%) or a combination of these drugs (15.1%). A total of 44 (19.9%) patients developed second primary malignancies with a median time to occurrence of 5.7 (range, 0.01-17.5) years. Second primary malignancies were the main cause for death (14; 27.5%). Compared with a matched normal Swiss population, the incidence of second primary malignancies was not increased. However, survival of patients with HCL was slightly inferior by comparison (P = .036). In conclusion, the outcome of HCL patients treated with subcutaneous cladribine is excellent, and in most patients, 1 cycle of subcutaneous cladribine is sufficient for long-term disease control.
毛细胞白血病（HCL）仍然是一种无法治愈的疾病。然而，静脉或皮下注射克拉屈滨进行一线治疗通常会带来持久的缓解。虽然有关于静脉应用的出色长期数据，但缺乏关于皮下给药的类似数据。因此，我们分析了瑞士临床癌症研究组（SAKK）进行的3项关于皮下注射克拉屈滨的前瞻性多中心临床试验的长期结果，这些试验在1993年至2005年间招募了221例经典型HCL患者。从治疗开始的中位总生存期未达到。多变量分析显示，治疗前贫血、较高的东部肿瘤协作组（ECOG）评分和较高的年龄与较差的总生存期相关，而在24个月和36个月时的早期进展对总生存期没有显著影响。中位5年（范围0.2 - 20.4年）后，53例（23.7%）患者需要二线治疗，首次再治疗主要是克拉屈滨单药治疗（66%）或利妥昔单抗单药治疗（15.1%）或这两种药物联合治疗（15.1%）。共有44例（19.9%）患者发生了第二原发性恶性肿瘤，中位发病时间为5.7年（范围0.01 - 17.5年）。第二原发性恶性肿瘤是主要的死亡原因（14例；27.5%）。与匹配的瑞士正常人群相比，第二原发性恶性肿瘤的发病率没有增加。然而，相比之下，HCL患者的生存率略低（P = 0.036）。总之，皮下注射克拉屈滨治疗的HCL患者预后良好，在大多数患者中，1个周期的皮下注射克拉屈滨足以实现长期疾病控制。
