Efficacy of Bortezomib-Based Regimens for Patients with Newly Diagnosed AL Amyloidosis Not Receiving ASCT Compared with Alkylators and Dexamethasone-Based Regimens

Background

For patients not receiving autologous stem cell transplantation (ASCT), bortezomib-based regimens such as CyBorD (bortezomib with cyclophosphamide and dexamethasone), BMDex (bortezomib with melphalan and dexamethasone), VD have been first-line treatment in AL amyloidosis over the last few years. Changing trends in first-line treatment (MDex to bortezomib-based regimens) significantly improved responses over time. However, several studies showed early death (<6 months) rate have been unchanged or rather higher in bortezomib-based group. The purpose of this study is to investigate the efficacy of bortezomib-based regimens for patients with newly diagnosed AL amyloidosis

Patients and methods

A total 115 patients with newly diagnosed AL amyloidosis who did not receive ASCT were analyzed. They were registered from two cohorts; retrospective cohort (April 1995 - November 2011, n=27) and prospective cohort (March 2012 - February 2017, n=88). Patients were classified into two groups according to first-line treatment; bortezomib-based regimens (VMP, n=39; Bortezomib and dexamethasone, n=12; CyBorD, n=8; BMDex, n=2; Bortezomib with thalidomide and dexamethasone, n=1) and alkylator and dexamethasone-based regimens (MDex, n=35; Cyclophosphamide and dexamethasone, n=18). The primary endpoint was early death rate and secondary endpoints were overall response rate, overall survival, and progression free survival.

Results

At baseline characteristics, bortezomib-based group showed significantly higher risk of cardiac involvement and more advanced stage. Although bortezomib-based group was in higher risk, there were no significant differences in early death rate (22.5% vs 18.8%, p=0.790). The rate of ≥ VGPR (very good partial response) and overall response rate were significantly higher in bortezomib-based group (50% vs 22.3% and 63.2% vs 28.9%, respectively). However overall survival and progression free survival represented no significant differences by two groups (p=0.916 and p=0.798).

Conclusion

In this retrospective analysis bortezomib-based regimens in newly diagnosed AL amyloidosis showed higher efficacy and similar early death rate and survival in spite of higher cardiac risks in comparison with alkylating agents with dexamethasone.

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