Rescue therapy with pomalidomide in combination with dexamethasone is a rapidly active regimen among heavily pretreated patients with amyloid light-chain (AL) amyloidosis, including those who have previously been exposed to alkylators, proteasome inhibitors, and lenalidomide, according to a study published in Blood.1

Although immunomodulatory drugs like lenalidomide and pomalidomide are active in AL amyloidosis, previous studies were unable to demonstrate a survival benefit among patients with AL amyloidosis responding to salvage therapy with pomalidomide.

In this open-label, phase 2 trial ( Identifier: NCT01510613), investigators enrolled 28 heavily pretreated patients with AL amyloidosis to evaluate the safety and efficacy of pomalidomide plus dexamethasone rescue therapy. Patients received oral pomalidomide continuously and dexamethasone weekly.

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Results showed that 68% (95% CI, 49-83) of patients achieved a hematologic response, including 29% who had a very good partial or complete response. Median time to response was 1 month.

Investigators observed hematologic responses in 86% of the 7 patients who received prior lenalidomide and in 75% of the 4 patients exposed to ixazomib.

Median progression-free survival and overall survival were 16 months and 26 months, respectively. Hematologic response was associated with improved overall survival (P = .001).

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More than half of patients had grade 3 to 4 adverse events, with the most common being fluid retention, infection, atrial fibrillation, and deep vein thrombosis. Nearly 30% of patients discontinued treatment due to adverse events.

The findings suggest that pomalidomide plus dexamethasone is an efficacious rescue strategy in relapsed/refractory AL amyloidosis. Studies investigating pomalidomide in combination with other agents as upfront treatment are warranted.


  1. Palladini G, Milani P, Foli A, et al. A phase II trial of pomalidomide and dexamethasone rescue treatment in patients with AL amyloidosis. Blood. 2017 Jan 27. doi: 10.1182/blood-2016-12-756528 [Epub ahead of print]