Journal Article

DKFZ-2017-00130

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis.

Schlenk, R. (First author)DKFZ* ; Stegelmann, F. ; Reiter, A. ; Jost, E. ; Gattermann, N. ; Hebart, H. ; Waller, C. ; Hochhaus, A. ; Platzbecker, U. ; Schafhausen, P. ; Blau, I. W. ; Verbeek, W. ; Heidel, F. H. ; Werner, M. ; Kreipe, H. ; Teleanu, V. ; Benner, A.DKFZ* ; Döhner, H. ; Grießhammer, M. ; Döhner, K.

2017
Nature Publ. Group Basingstoke

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Please use a persistent id in citations: doi:10.1038/leu.2016.299

Abstract: Myeloproliferative neoplasm (MPN)-associated myelofibrosis is a MPN characterized by bone marrow fibrosis, cytopenias, splenomegaly and constitutional symptoms. Pomalidomide, an immune-modifying drug, is reported to improve anaemia and thrombocytopenia in some patients with MPN-associated myelofibrosis. We designed a phase 2 study of pomalidomide in patients with MPN-associated myelofibrosis and anaemia and/or thrombocytopenia and/or neutropenia. Subjects received pomalidomide 2.0 mg/day in cohort 1 (n=38) or 0.5 mg/day in cohort 2 (n=58). Prednisolone was added if there was no response after 3 months in cohort 1 and based on up-front randomization in cohort 2 if there was no response at 3 or 6 months. Response rates were 39% (95% confidence interval (CI), 26-55%) in cohort 1 and 24% (95% CI, 15-37%) in cohort 2. In a multivariable logistic regression model pomalidomide at 2.0 mg/day (odds ratio (OR), 2.62; 95% CI, 1.00-6.87; P=0.05) and mutated TET2 (OR, 5.07; 95% CI, 1.16-22.17; P=0.03) were significantly associated with responses. Median duration of responses was 13.0 months (range 0.9-52.7). There was no significant difference in response rates or duration in subjects receiving or not receiving prednisolone. Clinical trial MPNSG 01-09 is registered at ClinicalTrials.gov (NCT00949364) and clinicaltrialsregister.eu (EudraCT Number: 2009-010738-23)Leukemia advance online publication, 29 November 2016; doi:10.1038/leu.2016.299.

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Contributing Institute(s):

1. Klinische Studienzentrale (G040)
2. Biostatistik (C060)

Research Program(s):

1. 317 - Translational cancer research (POF3-317) (POF3-317)

Appears in the scientific report 2017

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Database coverage:
; BIOSIS Previews ; Current Contents - Life Sciences ; Ebsco Academic Search ; IF >= 10 ; JCR ; NCBI Molecular Biology Database ; SCOPUS ; Science Citation Index ; Science Citation Index Expanded ; Thomson Reuters Master Journal List ; Web of Science Core Collection

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