Phase 1/2 trial shows venetoclax-dexamethasone achieves high response rates in relapsed/refractory systemic AL amyloidosis, indicating its strong potential as a treatment.
Key Points
This study evaluates the safety and efficacy of venetoclax-dexamethasone in relapsed/refractory systemic AL amyloidosis with t(11;14) translocation.
Phase 1/2 multicenter open-label trial
Investigated venetoclax with or without dexamethasone
Included patients with relapsed/refractory AL amyloidosis and prior anti-CD38 exposure
Maximally tolerated dose determined using Bayesian Optimal Interval Network design
12 out of 16 screened patients were enrolled
91.7% achieved hematologic overall response
75% reached ≥VGPR after treatment initiation
No dose limiting toxicities were reported during the first cycle
Substantial efficacy observed without significant safety issues