靶向 BCMA 疗法在复发/难治性 AL 淀粉样变性中的疗效与安全性：一项描述性汇总分析

B-cell maturation antigen (BCMA)-targeted therapies have emerged as promising treatment options for patients with relapsed/refractory immunoglobulin light-chain (AL) amyloidosis.

However, comprehensive data on their efficacy and safety remain limited.

We conducted a systematic descriptive pooled analysis of published studies reporting outcomes of BCMA-directed therapies including chimeric antigen receptor T-cell (CAR-T) therapies, bispecific antibodies (BsAbs), and antibody-drug conjugate (ADC). The analysis included 256 patients. CAR-T therapy was administered to 89 patients (35%), BsAbs to 82 patients (32%), and ADCs to 85 patients (33%). The pooled overall response rate (ORR) was 83%, with CAR-T demonstrating 92% ORR, BsAbs 89%, and ADCs 68%. Deep hematologic responses ( VGPR) were achieved in approximately 70% of CAR-T and BsAb recipients and 64% of ADC recipients.

Minimal residual disease negativity was documented in 75% of evaluable CAR-T patients and in 86% evaluable BsAb patients with reported data. Organ responses were observed in 30% of evaluable patients, with cardiac responses in 41%. Cytokine release syndrome occurred in 74% of CAR-T and 49% of BsAb patients, predominantly grade 1-2. Grade 3 CRS was rare (9% CAR-T, 1% BsAb).

Ocular toxicity was the predominant adverse event in ADC recipients (84% any grade, 29% grade 3). Grade 3 infections occurred in 13% of CAR-T, 20% of BsAb, and 6% of ADC patients. BCMA-targeted therapies result in high response rates with manageable safety profiles in heavily pretreated AL amyloidosis patients, supporting continued investigation and clinical application of these approaches.