Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis

Angela Dispenzieri, Francis Buadi, Kristina Laumann, Betsy LaPlant, Suzanne R. Hayman, Shaji K. Kumar, David Dingli, Steven R. Zeldenrust, Joseph R. Mikhael, Robert Hall, S. Vincent Rajkumar, Craig Reeder, Rafael Fonseca, P. Lief Bergsagel, A. Keith Stewart, Vivek Roy, Thomas E. Witzig, John A. Lust, Stephen J. Russell, Morie A. GertzMartha Q. Lacy

Research output: Contribution to journalArticlepeer-review

103 Scopus citations

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 was a discordance between the hematologic response and the N-terminal probrain natriuretic peptide response. The most common grade 3-5 adverse events, regardless of attribution, were neutropenia and fatigue. We conclude that pomalidomide appears to be a valuable drug covering an unmet clinical need in patients with previously treated AL. The trial is registered at www.clinicaltrials.gov as NCT00558896.

Original languageEnglish (US)
Pages (from-to)5397-5404
Number of pages8
JournalBlood
Volume119
Issue number23
DOIs
StatePublished - Jun 7 2012

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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