This study represents the first real-world report on the efficacy and safety of the BCMA-targeting bispecific antibody teclistamab in patients with relapsed/refractory systemic light chain amyloidosis (AL). Despite prior multiple treatment failures and progressive organ dysfunction, teclistamab achieved rapid and sustained hematological remission with favorable tolerability, offering novel therapeutic insights for this challenging patient population.
Literature Overview
This article, 'Safety and Efficacy Evaluation of Teclistamab in Relapsed/Refractory Amyloidosis: A Case Study', published in Frontiers in Oncology, reviews clinical responses and safety profiles of teclistamab in AL patients following multiple treatment failures. While current AL therapies exhibit high relapse rates and progressive organ deterioration, teclistamab—a BCMA-targeting bispecific antibody—has demonstrated remarkable efficacy in relapsed/refractory multiple myeloma but lacks clinical evidence in AL. This study fills this gap, providing clinicians with a potential therapeutic option.
Background Knowledge
Systemic light chain amyloidosis (AL) is a rare plasma cell disorder characterized by misfolded immunoglobulin light chains depositing in organs such as the heart, kidneys, liver, and nervous system, causing organ dysfunction. Standard treatments include daratumumab, bortezomib, and dexamethasone, yet relapses and treatment resistance remain common, with no unified subsequent therapy. Bispecific antibodies like teclistamab simultaneously target CD3 on T cells and BCMA on plasma cells, activating T-cell-mediated cytotoxicity. While extensively studied in multiple myeloma, AL efficacy lacks large-scale clinical validation. This study demonstrates rapid hematological response and cardiac improvement with teclistamab in a refractory AL case, highlighting its potential application while emphasizing the need for multi-center validation.
Research Methods and Experiments
The study evaluated a 64-year-old male diagnosed with AL amyloidosis, presenting with progressive renal and cardiac dysfunction after failing daratumumab, bortezomib, and dexamethasone therapies. Teclistamab treatment was initiated, and dynamic changes in hematological markers (lambda FLC), cardiac function (NTproBNP), and renal parameters (serum creatinine, proteinuria) were monitored. Treatment-related adverse events were documented.
Key Conclusions and Perspectives
Research Significance and Prospects
This real-world evaluation establishes teclistamab as a promising agent for relapsed/refractory AL. While hematological and cardiac benefits were evident, the lack of renal improvement suggests combination strategies may be necessary. Future multi-center trials should expand patient cohorts to confirm generalizability, optimize safety profiles, and explore synergistic regimens to enhance organ response rates, providing clinicians with robust evidence for treatment decision-making.
Conclusion
Systemic light chain amyloidosis (AL) is a severe plasma cell disorder causing multi-organ dysfunction with limited therapeutic options. This study reports a case of AL refractory to standard therapies achieving rapid hematological remission and cardiac improvement with teclistamab, demonstrating its favorable tolerability. Despite absent renal recovery, BCMA-targeting bispecific antibodies show potential for specific AL subpopulations. Further large-scale trials with extended follow-up are warranted to comprehensively assess efficacy, safety, and combination approaches, ultimately offering clinicians reliable therapeutic frameworks.
