Background: Primary plasma cell leukemia (pPCL) represents the most aggressive plasma cell dyscrasia with a poor prognosis and survival of <3 years. The International Myeloma Working Group (IMWG) adopted more inclusive diagnostic criteria for pPCL in 2021, including patients with 5% or more circulating plasma cells (down from 20%). Most published studies of pPCL do not include patients who meet the criteria for pPCL based on the newer diagnostic guidelines, and the data on the optimal treatment of pPCL is scarce. In our multi-center retrospective analysis, we report data on treatment regimens used in 67 pPCL patients to characterize outcomes in this population. Methods: We included patients with newly diagnosed pPCL between 2010 and 2023 based on the 2021 IMWG definition at one of three academic centers. Results: Our results suggest significant improvement in overall response rate (ORR) and progression-free survival (PFS) with the use of autologous stem cell transplant, but without additional benefit for a tandem transplant. The presence of high-risk cytogenetics was an independent risk factor for progression in the cohort. Conclusions: Our dataset represents one of the largest cohorts to date using the expanded definition of pPCL adopted by the IMWG in 2021 and stresses the importance of taking pPCL patients to transplant. Unfortunately, our study was not powered to determine the efficacy of individual induction and maintenance regimens, and many patients diagnosed with pPCL are ineligible for transplant based on end-organ damage at diagnosis or from disease that is refractory to induction therapy, underscoring the need for early diagnosis and treatment in hopes of preserving transplant eligibility.
背景：原发性浆细胞白血病（pPCL）是最具侵袭性的浆细胞疾病，预后极差，生存期通常不足3年。国际骨髓瘤工作组（IMWG）于2021年采用了更具包容性的pPCL诊断标准，将外周血浆细胞比例≥5%（原标准为20%）的患者纳入诊断范围。目前多数已发表的pPCL研究未包含符合新诊断标准的患者，且关于pPCL最佳治疗方案的数据极为有限。本多中心回顾性研究通过分析67例pPCL患者的治疗方案，旨在揭示该群体的临床结局特征。 方法：我们根据2021年IMWG标准，纳入了2010年至2023年间在三家学术中心新确诊的pPCL患者。 结果：研究显示自体干细胞移植能显著提高总体缓解率（ORR）并延长无进展生存期（PFS），但序贯移植未带来额外获益。高危细胞遗传学是该队列中疾病进展的独立危险因素。 结论：本研究是迄今采用2021年IMWG扩展诊断标准的最大规模pPCL队列之一，强调了将pPCL患者纳入移植治疗的重要性。遗憾的是，本研究未能评估具体诱导及维持治疗方案的有效性。许多pPCL患者因确诊时存在终末器官损伤或对诱导治疗耐药而丧失移植机会，这凸显了早期诊断与治疗对维持移植可行性的重要意义。
肿瘤(癌症)患者之家