Immunomodulatory drugs are active agents in light-chain (AL) amyloidosis. However, previous studies could not show a survival advantage for patients with AL amyloidosis responding to salvage treatment with pomalidomide. In this phase 2 trial,weassessed the safety and efficacy of pomalidomide and dexamethasone (PDex) in patients with AL amyloidosis who were previously exposed to bortezomib, alkylators, and other immunomodulatory drugs. Twenty-eight patients were enrolled. Three patients received pomalidomide 2 mg/d with no dose-limiting toxicity. The remaining patients received 4mg/d. Pomalidomide was administered continuously and dexamethasone was given once per week at a dose of 20 or 40 mg. Fifteen patients experienced grade 3 to 4 adverse events; the most common were fluid retention and infection. Hematologic response was observed in 68% of patients (very good partial response or complete response in 29%), as well as improved survival. Median timetoresponsewas1month.PDexisarapidly active regimen and improves survival in responding, heavily pretreated patients with AL amyloidosis.

A phase 2 trial of pomalidomide and dexamethasone rescue treatment in patients with AL amyloidosis

Palladini G.;Milani P.;Basset M.;Perlini S.;Merlini G.
2017-01-01

Abstract

Immunomodulatory drugs are active agents in light-chain (AL) amyloidosis. However, previous studies could not show a survival advantage for patients with AL amyloidosis responding to salvage treatment with pomalidomide. In this phase 2 trial,weassessed the safety and efficacy of pomalidomide and dexamethasone (PDex) in patients with AL amyloidosis who were previously exposed to bortezomib, alkylators, and other immunomodulatory drugs. Twenty-eight patients were enrolled. Three patients received pomalidomide 2 mg/d with no dose-limiting toxicity. The remaining patients received 4mg/d. Pomalidomide was administered continuously and dexamethasone was given once per week at a dose of 20 or 40 mg. Fifteen patients experienced grade 3 to 4 adverse events; the most common were fluid retention and infection. Hematologic response was observed in 68% of patients (very good partial response or complete response in 29%), as well as improved survival. Median timetoresponsewas1month.PDexisarapidly active regimen and improves survival in responding, heavily pretreated patients with AL amyloidosis.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11571/1367034
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