AUTOLOGOUS STEM CELL TRANSPLANTATION FOR LIGHT CHAIN AMYLOIDOSIS: A SINGLE CENTER REPORT

• Published in: Hematology, Transfusion and Cell Therapy Vol. 46; p. S975
• Main Authors: JM Pessoa, AD Americo, ISP Pittol, HTR Figueroa, FL Ayoub, GGM Lima, PL Zenero, NPC Zing, BM Gusmão, FR Kerbauy, JUA Filho, P Scheinberg
• Format: Journal Article
• Language: English
• Published: Elsevier 01-10-2024
• DOI: 10.1016/j.htct.2024.09.1658
• ISSN: 2531-1379

Context: High dose intravenous melphalan followed by Autologous Hematopoietic Stem Cell Transplant (ASCT) has been as essential part of light chain (AL) amyloidosis since 1990s. Patients who achieve complete hematologic response following ASCT have better Overall Survival (OS). However Transplant-Related Mortality (TRM) and clinical complications during ASCT continue to be a challenge and a major concern. Objective/Methods: We retrospectively reviewed AL patients who underwent ASCT between 2016 and 2024 at Beneficência Portuguesa de São Paulo (BP) to assess the procedure's safety and long-term outcomes. We analyzed patient baseline characteristics, Progression-Free Survival (PFS), ICU admission rates and Overall Survival (OS). Results: Twenty seven patients met criteria for this retrospective study. The median age was 60 y/o (95% CI 38‒70), 17 (63%) had an ECOG score 0, 11 (40%) had a mayo score of 1, 11 of which had cardiac involment and 23 renal invovement. Amog them, 81% (95% CI 61%‒92%) received ASCT as part of a first line therapy, five (18%) patients received no therapy prior to ASCT. Twelve (44%) received CyBorDex and nine (33%) Dara-based quadruplet regimens. The most commoly used conditioning regimen was Melphalan 200 mg/m2 (55.6%) with the remainder of patients received 140 mg/m2. During hospitalization, 11 patients experienced congestive heart failure de-compensation (4 profile ‘B’, 7 profile ‘C’), no patients were on dialysis at baseline, but 4 required hemodialysis during hospital follow-up. There were no in-hospital deaths in this cohort, however 33% of patients were admitted to the ICU within 30 days of follow-up (95% CI 16‒510). The OS and progression free survival rates, as estimated at 2 years by Kaplan Meier, were 88% (95% CI 73‒100) and 89% (95% CI 76‒100) respectively, without any mortality within 100 days from ASCT. Conclusion: In the present experience ASCT was safe and resulted in favorable outcomes, without TRM demonstrated.