Abstract：
BACKGROUND: Hairy cell leukemia (HCL) is a rare mature B-cell malignancy that is primarily treated with purine analogues. However, relapse remains a significant challenge, prompting the search for alternative therapies. The BRAF V600E mutation prevalent in HCL patients provides a target for treatment with vemurafenib.
METHODS: This multicenter retrospective study included nine patients with relapsed/refractory (R/R) HCL from six different centers. Patient data included demographics, prior treatments, clinical outcomes, and adverse events.
RESULTS: Patients received different treatment regimens between centers, including vemurafenib alone or in combination with rituximab. Despite the differences in protocols, all patients achieved at least a partial response, with seven patients achieving a complete response. Adverse events were generally mild with manageable side effects. The absence of myelotoxic effects and manageable side effects make BRAF inhibitors attractive, especially for patients ineligible for purine analogues or those with severe neutropenia.
CONCLUSIONS: Single agent vemurafenib or in combination with rituximab appears to be a promising therapeutic option for R/R HCL. Further research is needed to establish standardized treatment protocols and to investigate long-term outcomes.
METHODS: This multicenter retrospective study included nine patients with relapsed/refractory (R/R) HCL from six different centers. Patient data included demographics, prior treatments, clinical outcomes, and adverse events.
RESULTS: Patients received different treatment regimens between centers, including vemurafenib alone or in combination with rituximab. Despite the differences in protocols, all patients achieved at least a partial response, with seven patients achieving a complete response. Adverse events were generally mild with manageable side effects. The absence of myelotoxic effects and manageable side effects make BRAF inhibitors attractive, especially for patients ineligible for purine analogues or those with severe neutropenia.
CONCLUSIONS: Single agent vemurafenib or in combination with rituximab appears to be a promising therapeutic option for R/R HCL. Further research is needed to establish standardized treatment protocols and to investigate long-term outcomes.
摘要：
背景：毛状细胞白血病（HCL）是一种罕见的成熟B细胞恶性肿瘤，主要用嘌呤类似物治疗。然而,复发仍然是一个重大挑战，促使人们寻找替代疗法。HCL患者中普遍存在的BRAFV600E突变为vemurafenib治疗提供了靶标。
方法：这项多中心回顾性研究包括来自六个不同中心的9例复发/难治性（R/R）HCL患者。患者数据包括人口统计学，先前的治疗，临床结果，和不良事件。
结果：患者在中心之间接受不同的治疗方案，包括vemurafenib单独或与利妥昔单抗联合使用。尽管协议不同,所有患者至少达到部分反应，7名患者达到完全反应。不良事件一般轻微，副作用可控。没有骨髓毒性作用和可控制的副作用使BRAF抑制剂具有吸引力，特别是对于不适合嘌呤类似物的患者或严重中性粒细胞减少症的患者。
结论：单药vemurafenib或联合利妥昔单抗似乎是R/RHCL的有希望的治疗选择。需要进一步的研究来建立标准化的治疗方案并调查长期结果。
方法：这项多中心回顾性研究包括来自六个不同中心的9例复发/难治性（R/R）HCL患者。患者数据包括人口统计学，先前的治疗，临床结果，和不良事件。
结果：患者在中心之间接受不同的治疗方案，包括vemurafenib单独或与利妥昔单抗联合使用。尽管协议不同,所有患者至少达到部分反应，7名患者达到完全反应。不良事件一般轻微，副作用可控。没有骨髓毒性作用和可控制的副作用使BRAF抑制剂具有吸引力，特别是对于不适合嘌呤类似物的患者或严重中性粒细胞减少症的患者。
结论：单药vemurafenib或联合利妥昔单抗似乎是R/RHCL的有希望的治疗选择。需要进一步的研究来建立标准化的治疗方案并调查长期结果。
