title: 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.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Giovanni
authors.identifier: 0000-0001-5994-5138
authors.initials: G
authors.lastname: Palladini
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Milani
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Foli
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Basset
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Russo
authors.affiliation: Internal Medicine, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy; and.|Department of Internal Medicine, University of Pavia, Pavia, Italy.
authors.forename: Stefano
authors.initials: S
authors.lastname: Perlini
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Giampaolo
authors.identifier: 0000-0001-7680-3254
authors.initials: G
authors.lastname: Merlini
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality; D000069286:Bortezomib / Q000008:administration & dosage / Q000009:adverse effects; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects; D008297:Male; D008875:Middle Aged; D015996:Survival Rate; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*
chemical_list: D007147:Immunoglobulin Light Chains; D007155:Immunologic Factors; D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; C467566:pomalidomide