{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Dispenzieri A"],"funding":["NCI NIH HHS"],"pagination":["5397-404"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC3369677"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["119(23)"],"pubmed_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."],"journal":["Blood"],"pubmed_title":["Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis."],"pmcid":["PMC3369677"],"funding_grant_id":["P30 CA015083","P30 CA 15083"],"pubmed_authors":["Zeldenrust SR","Roy V","Fonseca R","Rajkumar SV","Lust JA","Russell SJ","Hayman SR","Mikhael JR","Hall R","Witzig TE","Dingli D","Dispenzieri A","Buadi F","Gertz MA","LaPlant B","Lacy MQ","Stewart AK","Kumar SK","Laumann K","Reeder C","Bergsagel PL"],"additional_accession":[]},"is_claimable":false,"name":"Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis.","description":"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.","dates":{"release":"2012-01-01T00:00:00Z","publication":"2012 Jun","modification":"2020-11-19T10:02:17Z","creation":"2019-03-27T00:54:15Z"},"accession":"S-EPMC3369677","cross_references":{"pubmed":["22493299"],"doi":["10.1182/blood-2012-02-413161"]}}