Does Post-Transplant Maintenance Therapy Improve Survival for Patients with Amyloidosis?

Maintenance therapy following autologous hematopoietic cell transplantation (AHCT) did not significantly impact survival for patients with light-chain (AL) amyloidosis, according to a study from researchers at the Ohio State University (OSU).

AHCT is an effective treatment option for patients with the plasma cell disorder AL amyloidosis, which is marked by the deposition of malformed proteins in the organs. Maintenance therapy, which is designed to prevent disease from recurring following primary therapy, is standard following AHCT for multiple myeloma (MM), but the benefit to this approach for AL amyloidosis is understudied.

In this retrospective analysis, researchers identified at 50 patients with systemic AL amyloidosis who had undergone AHCT at OSU. Of this cohort, 28 patients received maintenance therapy and 22 did not. The team assessed the impact of fluorescent in situ hybridization (FISH) abnormalities, bone marrow plasma cell burden (BMPC), and the degree of organ involvement. Kaplan-Meier survival analysis was utilized to compare the effect of maintenance therapy on progression-free survival (PFS) and overall survival (OS).

Overall, there was no significant difference in PFS (P=0.66) and OS (P=0.32) between the two patient groups. These results were consistent upon further categorization by FISH, BMPC level, or two or more organ involvement.

“Despite the survival benefit seen in MM with lenalidomide, we did not observe a difference in PFS or OS in [patients with] AL amyloidosis exposed to maintenance therapy following transplant,” the authors concluded.

This study was published in the Journal of Clinical Medicine.