Extended survival and reduced risk of AML progression in erythroid-responsive lenalidomide-treated patients with lower-risk del(5q) MDS Journal Article


Authors: List, A. F.; Bennett, J. M.; Sekeres, M. A.; Skikne, B.; Fu, T.; Shammo, J. M.; Nimer, S. D.; Knight, R. D.; Giagounidis, A.
Article Title: Extended survival and reduced risk of AML progression in erythroid-responsive lenalidomide-treated patients with lower-risk del(5q) MDS
Abstract: Lenalidomide is the approved treatment for patients with red blood cell (RBC) transfusion-dependent lower-risk myelodysplastic syndromes (MDS) and chromosome 5q deletion (del(5q)). We report the long-term outcomes (median follow-up 3.2 years) in patients treated with lenalidomide in the MDS-003 trial. RBC transfusion independence (TI) ≥8 weeks was achieved in 97 of 148 treated patients (65.5%), with a median response duration of 2.2 years. Partial or complete cytogenetic response was achieved by 63 of 88 evaluable patients (71.6%). Median overall survival (OS) was longer in patients achieving RBC-TI ≥8 weeks (4.3 vs 2.0 years in non-responders; P<0.0001) or cytogenetic response (4.9 vs 3.1 years in non-responders; P=0.010). Time to acute myeloid leukemia (AML) progression was longer in patients achieving RBC-TI ≥8 weeks or any cytogenetic response versus non-responders (P=0.001 and P=0.0002, respectively). In a landmark multivariate analysis, RBC-TI ≥8 weeks was associated with prolonged OS (P<0.001) and a trend toward reduced relative risk of AML progression (P=0.080). Among these lower-risk MDS patients with del(5q), lenalidomide was associated with prolonged RBC-TI and cytogenetic responses, which were linked to improved OS and reduced risk of AML progression. © 2014 Macmillan Publishers Limited. All rights reserved.
Keywords: adult; cancer survival; controlled study; treatment response; aged; acute granulocytic leukemia; major clinical study; overall survival; lenalidomide; disease course; treatment duration; outcome assessment; follow up; multiple cycle treatment; neutrophil count; cytogenetics; hemoglobin; hemoglobin blood level; age; thrombocyte count; cell count; erythrocyte transfusion; karyotype; myelodysplastic syndromes; acute myeloid leukemia; masculinity; 5q- syndrome; international prognostic scoring system; human; male; female; priority journal; article; del(5q); transfusion independence; bone marrow blast count
Journal Title: Leukemia
Volume: 28
Issue: 5
ISSN: 0887-6924
Publisher: Nature Publishing Group  
Date Published: 2014-05-01
Start Page: 1033
End Page: 1040
Language: English
DOI: 10.1038/leu.2013.305
PROVIDER: scopus
PMCID: PMC4017258
PUBMED: 24150217
DOI/URL:
Notes: Leukemia -- Export Date: 2 June 2014 -- CODEN: LEUKE -- Source: Scopus
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  1. Stephen D Nimer
    347 Nimer