文章：

泊马度胺：治疗多发性骨髓瘤的新型免疫调节剂

Pomalidomide: the new immunomodulatory agent for the treatment of multiple myeloma

原文发布日期：2013-09-06

DOI: 10.1038/bcj.2013.38

类型: Review

开放获取: 是

英文摘要：

In this report, we provide a comprehensive review on the preclinical and clinical investigations conducted in development of the next-generation immunomodulatory drug (IMiD) pomalidomide for the treatment of relapsed/refractory multiple myeloma (MM). We consulted PubMed, MEDLINE, ASH, ASCO annual symposium abstracts and http://clinicaltrials.gov/ for the purpose of this literature review. Twenty-six preclinical and 11 clinical studies were examined. These studies delineate the mechanisms of action of pomalidomide and attest to the robust clinical activity in relapsed/refractory MM. MM is the second most common hematological malignancy in the US. Despite availability of several therapeutic agents, MM remains incurable. Thus, the development of new therapies remains a priority. Pomalidomide is the newest member of the IMiDs class of drugs, and in preclinical and clinical investigations, it has demonstrated an improved efficacy and toxicity profile in comparison to its sister compounds, lenalidomide and thalidomide. Importantly, recent clinical studies have demonstrated its activity in relapsed or refractory myeloma, particularly in lenalidomide and bortezomib-refractory patients. Thus, the addition of pomalidomide to the anti-myeloma armamentarium is widely anticipated to have a significant impact on the overall clinical outcome of advanced stage relapsed and refractory MM patients.

摘要翻译： 

在本报告中，我们系统回顾了用于治疗复发/难治性多发性骨髓瘤的新一代免疫调节药物泊马度胺在临床前及临床研发阶段的相关研究。通过检索PubMed、MEDLINE、美国血液学会年会摘要、美国临床肿瘤学会年会摘要及ClinicalTrials.gov临床试验数据库，共纳入26项临床前研究和11项临床研究。这些研究阐明了泊马度胺的作用机制，并证实其在复发/难治性多发性骨髓瘤治疗中具有显著的临床活性。多发性骨髓瘤是美国发病率第二高的血液系统恶性肿瘤。尽管现有多种治疗手段，该疾病仍无法治愈，因此新疗法的研发仍是当务之急。作为免疫调节剂家族的最新成员，泊马度胺在临床前及临床研究中均显示出较其同类药物来那度胺和沙利度胺更优的疗效及更低的毒性特征。尤为重要的是，最新临床研究证实其在复发难治性骨髓瘤（特别是对来那度胺和硼替佐米耐药的病例）中具有治疗活性。因此，医学界普遍预期泊马度胺加入抗骨髓瘤药物体系后，将对晚期复发难治性多发性骨髓瘤患者的整体临床结局产生重大影响。

原文链接：

Pomalidomide: the new immunomodulatory agent for the treatment of multiple myeloma