浆细胞白血病临床特点及预后分析

目的 评估含硼替佐米或沙利度胺方案治疗浆细胞白血病(PCL)的疗效及对生存的影响。方法 回顾性分析北京大学人民医院2009年5月至2013年11月确诊并应用含硼替佐米或沙利度胺方案联合治疗20例PCL患者的临床资料，评估疗效及不良反应。应用Kaplan-Meier方法预估生存曲线。结果 40%的患者诊断时出现肾功能衰竭，85%的患者有溶骨病变。最常见的细胞遗传学异常是13q缺失，共有11例(55%)，其次是1q21(50%)和14q32异常(50%)，20%患者伴有17p缺失。50%的患者有3种以上染色体结构及数目异常。14例应用硼替佐米联合化疗，总有效率71.4%，其中5例完全缓解(CR)或接近完全缓解(nCR)，3例部分缓解(PR)，2例非常好的部分缓解(VGPR)，1例无反应；6例应用沙利度胺联合化疗，总有效率66.7%，其中1例CR，2例VGPR，1例PR；共有5例早期死亡，原因主要为心功能衰竭、肺部感染及本病进展。所有患者中位总生存期为10个月；原发性PCL和继发性PCL的中位生存期分别为20.6个月、3.8个月(P=0.048)；应用硼替佐米为基础的方案与沙利度胺为基础方案的中位生存期分别为18个月、3个月(P=0.178)。结论 含硼替唑米的联合化疗方案治疗PCL较传统方案提高了反应率，延长了生存期。

Clinical characteristics and prognosis of plasma cell leukemia.

Abstract：Objective To evaluate the outcomes of patients with plasma cell leukemia (PCL) in the era of novel agents (Bortezomib and Thalidomide).Methods We retrospectively reviewed the outcomes of patients with PCL managed at our institution from May 2009 to November 2013.Responses and side-effects were evaluated.We used SPSS software to calculate survival and the Kaplan-Meier method to estimate survival curves.Results We identified 20 patients with PCL (14 with primary PCL［pPCL］and 6 with secondary PCL［sPCL］) from our institution.The median age at diagnosis of PCL was 56 years (range：31 to 80 years).The median time from diagnosis of multiple myeloma (MM) to sPCL was 4 months (range：4-78 months).Forty percent of patients had renal failure at the time of diagnosis and 85% of patients presented with lytic bone lesions.The most common cytogenetic abnormality was deletion 13q，and 20% of patients had deletion 17p.Fifty percent of patients had more than 3 chromosomal abnormalities.Among the 14 patients treated with bortezomib，5 patients achieved complete remission (CR)，2 patients with very good partial remission (VGPR)，and 3 with partial remission (PR) and the overall response rates (ORR) were 71.4%.Six patients received Thalidomide-based regimens and the ORR were 66.7%，including 1 case of CR，2 cases of VGPR and 1 case of PR.The median overall survival for all patients was 10 months.The median overall survival for patients with pPCL was 20.6，significantly longer than that for patients with sPCL(P=0.048).Bortezomib-treated patients had a better median survival time than thalidomide-treated ones (P=0.178).Conclusion Plasma cell leukemia is an uncommon but aggressive malignancy associated with high-risk cytogenetic abnormalities and frequent organ dysfunction，ultimately resulting in poor prognosis.Bortezomib-containing combinations improve the response rate and prolong survivals of patients as compared with traditional regimens.