Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis

Abstract

Immunoglobulin light-chain (AL) amyloidosis is a rare, incurable plasma cell disorder. Its therapy has benefited immensely from the expanding drug armamentarium available for multiple myeloma. Pomalidomide in combination with weekly dexamethasone (Pom/dex) is active among patients with relapsed myeloma. In the present study, we explored the Pom/dex combination in patients with previously treated AL. Patients were eligible for this prospective phase 2 trial if they had had at least one prior regimen and if they had reasonably preserved organ function. Patients were treated with oral Pom/dex. Thirty-three patients were enrolled. The median age was 66 years. Median time from diagnosis to on-study was 37 months. Eighty-two percent had cardiac involvement. The confirmed hematologic response rate was 48%, with a median time to response of 1.9 months. Organ improvement was documented in 5 patients. The median overall and progression-free survival rates were 28 and 14 months, respectively; the 1-year overall and progression-free survival rates were 76% and 59%, respectively. There

Daratumumab yields rapid and deep hematologic responses in patients with heavily pretreated AL amyloidosis

“…15 Frequently, these responses are of limited duration (14.1-month median progression-free survival in the pomalidomide study and not yet reported in those receiving carfilzomib) and therapeutic options for relapsed patients remain limited.…”

Pomalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 1 and 2 trial

“…Although pomalidomide/dexamethasone has already been shown to have activity in AL amyloidosis by Dispenzieri et al, 14 our trial is different in that it includes a dose-escalation format to arrive at an MTD. This study design was prompted by the experience of lenalidomide dosing in AL amyloidosis.…”

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

“…7,8 Two independent trials showed efficacy of pomalidomide in AL amyloidosis; hematologic response occurred in 48% to 50% of patients. 9,10 However, these studies could not show an impact of response on survival. We designed the present phase 2 trial to assess the efficacy, safety, and impact on survival of the combination of pomalidomide and dexamethasone (PDex) in patients with AL amyloidosis treated upfront with bortezomib and an alkylating agent.…”