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

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, we assessed 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 4 mg/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 time to response was 1 month. PDex is a rapidly active regimen and improves survival in responding, heavily pretreated patients with AL amyloidosis. This trial was registered at www.clinicaltrials.gov as #NCT01510613.

• Authors: Giovanni Palladini, Paolo Milani, Andrea Foli, Marco Basset, Francesca Russo, Stefano Perlini, Giampaolo Merlini
• Journal: Blood (Blood) Vol. 129 Issue 15 Pg. 2120-2123 (04 13 2017) ISSN: 1528-0020 [Electronic] United States
• PMID: 28130212 (Publication Type: Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2017 by The American Society of Hematology.

Chemical References

• Immunoglobulin Light Chains
• Immunologic Factors
• Thalidomide
• Bortezomib
• Dexamethasone
• pomalidomide

Topics

• Adult
• Aged
• Aged, 80 and over
• Amyloidosis (drug therapy, mortality)
• Bortezomib (administration & dosage, adverse effects)
• Dexamethasone (administration & dosage, adverse effects)
• Disease-Free Survival
• Female
• Humans
• Immunoglobulin Light Chains
• Immunologic Factors (administration & dosage, adverse effects)
• Male
• Middle Aged
• Survival Rate
• Thalidomide (administration & dosage, adverse effects, analogs & derivatives)