Long-term prognosis of AL amyloidosis in patients treated with bortezomib or thalidomide

Abstract Introduction AL amyloidosis has become a common secondary cause in elderly male patients presenting with nephrotic syndrome. Daratumumab-CyBorD regimen is approved as the standard first-line regimen for AL-amyloidosis. We aim to analyze the prognosis of patients treated with bortezomib-based regimen and other alternative regimens. Method We retrospectively collected the baseline and follow up data of newly diagnosed AL amyloidosis patients. Hematological and renal response rate were compared among different regimens and overall survival and renal survival were analyzed. Subgroup analysis of Mayo stage III patients was also performed. Results 72 cases were included, 48.6% of whom had cardiac involvement. Overall response rate in patients treated with bortezomib-based regimen was 67.4%, including 27.9% complete response (CR). Renal outcomes such as proteinuria response rate and incidence of end stage renal disease (ESRD) were not different between bortezomib and thalidomide-based therapy.Median follow up for whole cohort was 22 months,and 13(18.1%) patients died at the end of follow-up, while 7 patients(9.7%) progressed to dialysis. Median overall survival (OS) was not reached in both regimens and 1 year survival rates were 90.4% and 80.0% respectively in bortezomib and thalidomide based therapy(P = 0.127). Patients treated with bortezomib-based treatment had longer organ deterioration progression-free survival (MOD-PFS).There was no difference in response rate and OS between patients treated with cyclophosphamide, bortezomib,dexamethasone(CVD) and bortezomib, dexamethasone (VD). Mayo stage III patients who were treated with VD regimen had longer OS compared with TD or CTD regimen. Conclusion Bortezomib-based regimen had high efficacy in promoting rapid hematologic responses and reducing the risk of major organ deterioration in AL amyloidosis, which was also effective among patients with cardiac insufficiency. Addition of cyclophosphamide into VD regimen could not further improve the overall remission or survival of AL amyloidosis.