原发性浆细胞白血病：来自中国单中心研究的 46 例真实世界回顾性研究。
Primary Plasma Cell Leukemia: Real-World Retrospective Study of 46 Patients From a Single-Center Study in China.
机构信息
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Hematological Disorders, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China; Jerome Lipper Multiple Myeloma Center, LeBow Institute for Myeloma Therapeutics, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA.
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Hematological Disorders, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China.
出版信息
Clin Lymphoma Myeloma Leuk. 2020 Oct;20(10):e652-e659. doi: 10.1016/j.clml.2020.05.014. Epub 2020 May 27.
BACKGROUND
Primary plasma cell leukemia (PPCL) is a rare and aggressive plasma cell disorder. The use of novel agents, together with autologous stem cell transplantation, has improved survival outcome in PPCL. However, the prognosis is still very poor, and the optimal treatment remains an unmet clinical need.
PATIENTS AND METHODS
We studied the efficacy and prognostic impact of novel agents in 46 patients with PPCL patients at the Blood Diseases Hospital in China. We examined the impact of clinical and laboratory features, as well as therapies (bortezomib- and/or immunomodulatory drug-based therapies, chemotherapy) on survival and extent of clinical response, including progression-free survival and overall survival (OS). Progression-free survival and OS were assessed by the Kaplan-Meier method, and survival distributions were compared by log-rank test.
RESULTS
In our cohort of 46 PPCL patients, the median age at the time of diagnosis was 54 years. Overall response rate was 54% (25/46). The median (95% confidence interval) progression-free survival time was 6 (0-12.5) months, and OS time was 14 (4.6-23.4) months. The OS time was significantly longer in patients treated with bortezomib-based versus non-bortezomib-based therapies (median [95% confidence interval], 19 [9-28.9] vs. 5 [4-24] months; P = .019).
CONCLUSION
This large single-center study of PPCL supports the use of bortezomib-based therapies as frontline treatment in PPCL patients.
背景
原发性浆细胞白血病（PPCL）是一种罕见且侵袭性的浆细胞疾病。新型药物的应用，结合自体干细胞移植，已经改善了 PPCL 的生存预后。然而，预后仍然非常差，最佳治疗方法仍然是未满足的临床需求。
患者与方法
我们在中国血液疾病医院研究了 46 例 PPCL 患者中新型药物的疗效和预后影响。我们研究了临床和实验室特征以及治疗方法（硼替佐米和/或免疫调节药物治疗、化疗）对生存和临床反应程度（包括无进展生存期和总生存期）的影响。无进展生存期和总生存期通过 Kaplan-Meier 法评估，生存分布通过对数秩检验比较。
结果
在我们的 46 例 PPCL 患者队列中，诊断时的中位年龄为 54 岁。总缓解率为 54%（25/46）。中位（95%置信区间）无进展生存期为 6（0-12.5）个月，总生存期为 14（4.6-23.4）个月。接受硼替佐米为基础治疗与非硼替佐米为基础治疗的患者的总生存期有显著差异（中位[95%置信区间]，19[9-28.9] vs. 5[4-24]个月；P=0.019）。
结论
这项针对 PPCL 的大型单中心研究支持在 PPCL 患者中使用硼替佐米为基础的治疗方法作为一线治疗。
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