Journal
BLOOD
Volume 119, Issue 23, Pages 5397-5404Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2012-02-413161
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Funding
- Celgene
- Robert A. Kyle Hematologic Malignancies Program
- JABBS Foundation
- Predolin Foundation
- National Institutes of Health [P30 CA 15083]
- Millennium
- Merck
- Genzyme
- Novartis
- Cephalon
- Medtronic
- Otsuka
- BMS
- Lilly
- AMGEN
- Cylene
- Onyx
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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. (Blood. 2012;119(23):5397-5404)
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