Development and validation of a prognostic staging system for primary plasma cell leukemia

作者：Mengru Tian, Gang An, Weijun Fu, Wenqiang Yan, Li Lu, Chunyan Sun, Zhenyu Li, Lijuan Chen, Aijun Liao, Guangxun Gao, Xiaoqi Qin, Mengyao Li, Chunrui Li, Hua Xue, Li Gao, Yi Wang, He Aili, Fan Zhou, Dongmei Guo, Yujun Dong, Zhihong Fang, Xiaoxia Chu, Jian‐Qing Mi, Chengcheng Fu, Hui Zeng, Shuling Hou, Xiaotao Wang, Hua Wang, Yongqiang Wei, Xinyue Liang, Xingcheng Yi, Yue Sun, Lugui Qiu, Yun Dai, Fengyan Jin · 发表于：Journal of Hematology & Oncology · 年份：2025 · DOI：10.1186/s13045-025-01723-0 · 被引用次数：3 · 研究领域：Multiple Myeloma Research and Treatments、Chronic Myeloid Leukemia Treatments、Acute Myeloid Leukemia Research

BACKGROUND: The existing risk models for multiple myeloma (MM) are suboptimal for the stratification of patients with primary plasma cell leukemia (pPCL), a rare and peculiar MM. In this study, we aimed to develop a staging system for pPCL defined as the presence of ≥ 5% circulating plasma cells (CPC) according to the new diagnostic criteria, utilizing one of the largest series of patients with pPCL. METHODS: This multicenter retrospective study included 340 patients with pPCL (the training cohort) from 25 centers nationwide in China. The prognostic impact of baseline characteristics and cytogenetic abnormalities was evaluated. Univariate and multivariate analyses were conducted to identify variables predicting overall survival (OS) to develop a staging system. Its performance was then validated in an independent cohort (n = 80). Genome-wide DNA and RNA sequencing were performed to explore the molecular basis for inter-stage clinical heterogeneity. RESULTS: Del(17p), t(4;14), and t(14;16), but not 1q+, were verified as high-risk cytogenetic abnormalities (HRCAs) of pPCL. HRCA, elevated LDH, and thrombocytopenia had the highest impact on OS and were used to create a simple algorithm, stratifying patients with pPCL into stages I, II, and III, with median OS of 54.1, 24.0, and 5.4 months (II vs. I: HR, 1.986; 95% CI, 1.034-3.814; P = 0.0394; III vs. II: HR, 3.206; 95% CI, 1.757-5.852; P = 0.0001) in the training cohort and 62.1, 31.6, and 21.8 months (II vs. I: HR, 2.013; 95% CI...