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Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis.

Authors
  • Dispenzieri, Angela
  • Buadi, Francis
  • Laumann, Kristina
  • LaPlant, Betsy
  • Hayman, Suzanne R
  • Kumar, Shaji K
  • Dingli, David
  • Zeldenrust, Steven R
  • Mikhael, Joseph R
  • Hall, Robert
  • Rajkumar, S Vincent
  • Reeder, Craig
  • Fonseca, Rafael
  • Bergsagel, P Lief
  • Stewart, A Keith
  • Roy, Vivek
  • Witzig, Thomas E
  • Lust, John A
  • Russell, Stephen J
  • Gertz, Morie A
  • And 1 more
Type
Published Article
Journal
Blood
Publisher
American Society of Hematology
Publication Date
Jun 07, 2012
Volume
119
Issue
23
Pages
5397–5404
Identifiers
DOI: 10.1182/blood-2012-02-413161
PMID: 22493299
Source
Medline
License
Unknown

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 pro-brain 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.

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