文章：

毛细胞白血病：简短回顾，今天的建议和展望

Hairy cell leukemia: short review, today’s recommendations and outlook

原文发布日期：2014-02-14

DOI: 10.1038/bcj.2014.3

类型: Original Article

开放获取: 是

英文摘要：

Hairy cell leukemia (HCL) is part of the low-grade non-Hodgkin lymphoma family and represents approximately 2% of all leukemias. Treatment with splenectomy and interferon-α historically belonged to the first steps of therapeutic options, achieving partial responses/remissions (PR) in most cases with a median survival between 4 and 6 years in the 1980s. The introduction of the purine analogs (PA) pentostatin and cladribine made HCL a well-treatable disease: overall complete response rates (CRR) range from 76 to 98%, with a median disease-free survival (DFS) of 16 years a normal lifespan can be reached and HCL-related deaths are rare. However, insufficient response to PA with poorer prognosis and relapse rates of 30–40% after 5–10 years of follow-up may require alternative strategies. Minimal residual disease can be detected by additional examinations of bone marrow specimens after treatment with PA. The use of immunotherapeutic monoclonal antibodies (mAB) like rituximab as a single agent or in combination with a PA or more recently clinical trials with recombinant immunotoxins (RIT) show promising results to restrict these problems. Recently, the identification of the possible disease-defining BRAF V600E mutation may allow the development of new therapeutic targets.

摘要翻译： 

毛细胞白血病（HCL）属于低度恶性非霍金森淋巴瘤范畴，约占所有白血病病例的2%。历史上，脾切除术与α-干扰素治疗曾是首选治疗方案，在1980年代使大多数患者获得部分缓解，中位生存期达4至6年。嘌呤类似物喷司他丁与克拉屈滨的引入使HCL成为可有效控制的疾病：总完全缓解率达76%-98%，中位无病生存期达16年，患者可达到正常预期寿命，HCL相关死亡罕见。然而，对嘌呤类似物应答不足者预后较差，5-10年随访期间复发率达30%-40，需采用替代策略。通过嘌呤类似物治疗后骨髓标本的补充检查可检测微小残留病灶。免疫治疗单克隆抗体（如利妥昔单抗）的单药应用、与嘌呤类似物联用，以及近期重组免疫毒素的临床试验，均显示出解决这些问题的良好前景。近期研究中，对疾病定义性BRAF V600E突变的识别有望为开发新的治疗靶点提供方向。

原文链接：

Hairy cell leukemia: short review, today’s recommendations and outlook