Singh Shagun, Peshin Supriya, Wertheim Betsy C, Larsen Ashley, Chineke Iloabueke, Sborov Douglas W, Green Damian, Liedtke Michaela, Okoniewski Marie, Wazir Mohammed, Nadeem Omar, Schachter Levanto G, DeGraaff Dominique, Vardell Victoria A, Coffey David G, Gowin Krisstina
Internal Medicine, Banner University Medical Center.
Norton Community Hospital, Ballad Health.
Haematologica. 2025 Sep 1;110(9):2129-2138. doi: 10.3324/haematol.2024.287158. Epub 2025 Apr 17.
Plasma cell leukemia (PCL) is a rare and aggressive plasma cell disorder with a short survival duration despite the use of novel therapeutics. PCL remains an understudied disease for which there is no current standard of care treatment. Knowledge on optimal novel drug sequencing, including the role and timing of hematopoietic stem cell transplant as primary and salvage therapy is needed. We conducted a large retrospective analysis of 153 PCL patients, using the new definition of ≥5% circulating plasma cells, including clinical features and treatment outcomes across seven academic centers in the United States. This is, to the best of our knowledge, the largest multicenter study conducted in the US of this rare disease. Disease presentation, clinical and genetic characteristics, and treatment patterns of 93 patients with primary PCL and 57 with secondary PCL are described. Additionally, associations between patient characteristics and mortality were investigated using Cox proportional hazards regression models. Secondary PCL was associated with a worse prognosis, with a median overall survival of 3.2 months compared to 36.6 months for primary PCL (P<0.001). Receipt of a transplant was associated with a survival advantage in both primary PCL (Hazard Ratio [HR] 0.16, P<0.001) and secondary PCL (HR 0.20, P=0.001). No significant difference in outcomes was observed between proteasome inhibitor-based triplet regimens and the VTD-PACE like regimen. The presence of extramedullary disease and high-risk cytogenetics was not associated with survival in the primary PCL group.
浆细胞白血病（PCL）是一种罕见且侵袭性强的浆细胞疾病，尽管使用了新型疗法，其生存时间仍很短。PCL仍然是一种研究不足的疾病，目前尚无标准的治疗方案。需要了解最佳的新型药物序贯，包括造血干细胞移植作为一线和挽救治疗的作用及时机。我们对153例PCL患者进行了一项大型回顾性分析，采用≥5%循环浆细胞的新定义，涵盖了美国七个学术中心的临床特征和治疗结果。据我们所知，这是美国针对这种罕见疾病开展的最大规模多中心研究。描述了93例原发性PCL患者和57例继发性PCL患者的疾病表现、临床和基因特征以及治疗模式。此外，使用Cox比例风险回归模型研究了患者特征与死亡率之间的关联。继发性PCL的预后较差，中位总生存期为3.2个月，而原发性PCL为36.6个月（P<0.001）。接受移植在原发性PCL（风险比[HR]0.16，P<0.001）和继发性PCL（HR 0.20，P=0.001）中均与生存优势相关。基于蛋白酶体抑制剂的三联方案与VTD-PACE类似方案在疗效上未观察到显著差异。原发性PCL组中髓外疾病和高危细胞遗传学的存在与生存无关。
