Key Information
AL Amyloidosis: A Real-World Experience with CAR-T Cell Therapy
Melinda SY Tan1 , Rahma Warsame1 , Ricardo Parrondo2 , Eli Muchtar1 , Taxiarchis Kourelis1 , Francis Buadi1 , Wilson Gonsalves1 , Joselle Cook1 , Prashant Kapoor1 , Suzanne Hayman1 , David Dingli1 , Moritz Binder1 , Nelson Leung1 , Vivek Roy2 , Taimur Sher2 , Erin Wiedmeier-Nutor3 , Saurabh Chhabra, MD MS3 , Rafael Fonseca3 , Nadine Abdallah1 , Vincent Rajkumar1 , Shaji Kumar1 , Morie Gertz1 , Yi Lin1 , Angela Dispenzieri1*
1Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN. 2Division of Hematology and Oncology, Department of Medicine, Mayo Clinic, Jacksonville, FL, USA 3Division of Hematology and Oncology, Department of Medicine, Mayo Clinic, Phoenix, AZ, USA *corresponding author: Angela Dispenzieri, [email protected]
Data Sharing Statement: The data that support the findings of this study are available from the corresponding author upon request. De-identified patient data access will be shared with qualified researches after approval of a written proposal and data use agreement.
There is no current established standard-of-care for patients with relapsed/refractory (RR) light chain (AL) amyloidosis. While the introduction of daratumumab combined with bortezomib, cyclophosphamide, and dexamethasone (Dara-VCD) 1 as frontline therapy has significantly improved outcomes, a subset of patients fail to achieve adequate responses or relapse, posing a therapeutic challenge.
Given the demonstrated efficacy of chimeric antigen receptor T-cell (CAR-T) therapy, particularly in multiple myeloma (MM)2,3, their potential application in AL amyloidosis is under active investigation. However, organ dysfunction, especially cardiac and renal, frequently precludes trial eligibility, limiting available data.
In this real-world study, we describe the clinical course of patients with systemic AL amyloidosis who underwent CAR-T therapy. The primary objective was to evaluate the safety and efficacy of this approach in a high-risk patient population.
We retrospectively identified nine patients with histologically confirmed systemic AL amyloidosis who underwent commercial B-cell maturation antigen (BCMA)-directed CAR-T at Mayo Clinic between March 1, 2021, and March 31, 2025. We included two patients previously reported by Das et al.4 with updated follow-up, and one who underwent CAR-T externally. The study was approved by the institutional review board. AL amyloidosis was confirmed via tissue biopsy with Congo red staining and amyloid typing using mass spectrometry. Four additional patients with amyloid limited to bone marrow and/or fat pad were excluded.