B -Cell Maturation Antigen-CD19 Dual-Targeted Chimeric Antigen Receptor- T -Cell Therapy for Relapsed or Refractory AL Amyloidosis.
Xianghua Huang, Xiaomei Wu, Wencui Chen et al.
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In 30 seconds
This single-centre exploratory phase I clinical trial assessed BCMA-CD19 dual-targeted CAR T-cell therapy in six patients with relapsed or refractory AL amyloidosis after at least two prior treatment lines; all had kidney involvement. After a median follow-up of 640 (range, 563-745) days, all six achieved hematologic complete response and renal response (100% for each; 95% confidence interval, 54% to 100%). The treatment was reported as well tolerated, with no dose-limiting toxicity. One patient relapsed at month 6; the other five remained in remission.
Key findings
- All six patients achieved hematologic complete response (100%, 95% confidence interval, 54% to 100%).
- All six patients achieved renal response (100%, 95% confidence interval, 54% to 100%).
- Grade 1 cytokine release syndrome occurred in two patients; no immune effector cell-associated neurotoxicity syndrome was identified.
- Pneumonia occurred in two patients; one patient developed acute promyelocytic leukemia 15 months after CAR T-cell therapy.
Why it matters
AL amyloidosis can involve major organs, and this small trial reports hematologic and renal responses after dual-targeted CAR T-cell therapy in heavily pretreated patients. The findings provide early clinical evidence of activity and describe observed toxicities, while the limited cohort means the response rates and safety profile require confirmation in larger studies.
What to keep in mind
This was a single-centre exploratory trial with only six patients, limiting the precision and generalisability of its efficacy and safety findings.