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脾肿大在 BCMA 靶向 CAR-T 细胞治疗复发骨髓瘤中的预后意义

英文原题:Prognostic Implications of Splenomegaly in BCMA-Directed CAR T-Cell Therapy for Relapsed Myeloma.

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Prognostic Implications of Splenomegaly in BCMA-Directed CAR T-Cell Therapy for Relapsed Myeloma.

PubMed 2025/07/16(内容时间) Transplant Cell Ther Q1 · IF 4.7(JCR 2025)

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研究概要

脾脏大小可能作为 RRMM 患者 CAR-T 细胞治疗中一个有前景的预后标志物,为增强风险分层提供一个简单且易于获取的工具。这一发现可为这些患者的监测策略提供信息,并优化医疗资源管理。

研究思路结论见上方概要

B细胞成熟抗原(BCMA)靶向嵌合抗原受体(CAR)T细胞疗法在复发/难治性多发性骨髓瘤(RRMM)患者中显示出显著前景。尽管其疗效显著,但治疗常伴随不良事件,如细胞因子释放综合征和血液学毒性,包括严重血小板减少症。识别可靠的预后标志物对于改善患者风险分层、优化治疗策略和有效管理并发症至关重要。虽然已探索了多种预后标志物,但脾脏大小在这一背景下尚未得到广泛研究。

本研究旨在评估脾脏大小作为接受CAR-T 细胞治疗的RRMM患者预后标志物的价值。具体而言,我们考察其与血小板减少症、代谢肿瘤体积、可溶性BCMA(sBCMA)水平、无进展生存期(PFS)和总生存期(OS)的关联。此外,我们将脾脏大小与已确立的预后标志物进行比较,包括基线sBCMA、EASIX和CAR-HEMATOTOX评分,以确定其预测价值。

对接受Idecabtagene vicleucel或Ciltacabtagen autoleucel治疗的RRMM患者(N = 73)数据进行了分析。评估了通过计算机断层扫描成像评估的脾脏大小与临床结局之间的关联。

脾肿大(脾脏大小 >340 cm)与严重且持续的血小板减少症、更高的代谢肿瘤体积以及升高的 sBCMA 水平显著相关。在我们的队列中,脾肿大成为 PFS 和 OS 的独立预后因素,其相关性比 sBCMA、EASIX 和 CAR-HEMATOTOX 评分等其他标志物更强。

展开英文摘要原文

B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell therapy has shown significant promise for patients with relapsed or refractory multiple myeloma (RRMM). Despite its efficacy, treatment is frequently complicated by adverse events such as cytokine release syndrome and hematologic toxicities, including severe thrombocytopenia. Identifying reliable prognostic markers is essential to improve patient risk stratification, optimizing treatment strategies, and managing complications effectively. While various prognostic markers have been explored, spleen size has not been extensively studied in this context.

This study aims to evaluate spleen size as a prognostic marker in RRMM patients receiving CAR T-cell therapy. Specifically, we examine its association with thrombocytopenia, metabolic tumor volume, soluble BCMA (sBCMA) levels, progression-free survival (PFS), and overall survival (OS). Additionally, we compare spleen size to established prognostic markers, including baseline sBCMA, EASIX, and CAR-HEMATOTOX scores, to determine its predictive value. STUDY DESIGN: Data from RRMM patients (N = 73) treated with either Idecabtagene vicleucel or Ciltacabtagen autoleucel were analyzed. The association of spleen size, assessed via computed tomography imaging, with clinical outcomes was evaluated.

Splenomegaly (spleen size >340 cm ) was found to be significantly associated with severe and prolonged thrombocytopenia, higher metabolic tumor volumes, and elevated sBCMA levels. In our cohort, splenomegaly emerged as an independent prognostic factor for both PFS and OS, showing stronger associations than other markers such as sBCMA, EASIX, and CAR-HEMATOTOX scores.

Spleen size may serve as a promising prognostic marker in CAR T-cell therapy for RRMM patients, providing a simple and readily accessible tool for enhancing risk stratification. This finding could inform monitoring strategies and optimize healthcare resource management for these patients.

论文信息

作者
Wiemers TC、Ferle M、Ader J、Sotikova V、Fandrei D、Grieb N、Fischer L、Born P
单位
Department of Hematology, Hemostaseology and Cellular Therapy, University Hospital Leipzig, Leipzig, Germany; Innovation Center Computer Assisted Surgery (ICCAS), University Hospital Leipzig, Leipzig, Germany. Electronic address: thomas.wiemers@medizin.uni-leipzig.de.Germany
期刊
Transplantation and cellular therapy2025 Oct
原文标识
PubMed 40680848 · DOI 10.1016/j.jtct.2025.07.003