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).
journal_name
Leukemiajournal_title
Leukemiaauthors
Schlenk RF,Stegelmann F,Reiter A,Jost E,Gattermann N,Hebart H,Waller C,Hochhaus A,Platzbecker U,Schafhausen P,Blau IW,Verbeek W,Heidel FH,Werner M,Kreipe H,Teleanu V,Benner A,Döhner H,Grießhammer M,Döhner Kdoi
10.1038/leu.2016.299subject
Has Abstractpub_date
2017-04-01 00:00:00pages
889-895issue
4eissn
0887-6924issn
1476-5551pii
leu2016299journal_volume
31pub_type
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