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

Giovanni Palladini, Paolo Milani, Andrea Foli, Marco Basset, Francesca Russo, Stefano Perlini, Giampaolo Merlini

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)2120-2123
Number of pages4
Issue number15
Publication statusPublished - Apr 13 2017

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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