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| 001 | 119376 | ||
| 005 | 20240228145436.0 | ||
| 024 | 7 | _ | |a 10.1038/leu.2016.299 |2 doi |
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| 024 | 7 | _ | |a 1476-5551 |2 ISSN |
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| 100 | 1 | _ | |a Schlenk, Richard |0 P:(DE-He78)d8a0e60e5e095f3161ee0de3712409bc |b 0 |e First author |
| 245 | _ | _ | |a Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. |
| 260 | _ | _ | |a Basingstoke |c 2017 |b Nature Publ. Group |
| 336 | 7 | _ | |a article |2 DRIVER |
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| 336 | 7 | _ | |a Journal Article |b journal |m journal |0 PUB:(DE-HGF)16 |s 1525081189_17856 |2 PUB:(DE-HGF) |
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| 520 | _ | _ | |a 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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| 700 | 1 | _ | |a Stegelmann, F. |b 1 |
| 700 | 1 | _ | |a Reiter, A. |b 2 |
| 700 | 1 | _ | |a Jost, E. |b 3 |
| 700 | 1 | _ | |a Gattermann, N. |b 4 |
| 700 | 1 | _ | |a Hebart, H. |b 5 |
| 700 | 1 | _ | |a Waller, C. |b 6 |
| 700 | 1 | _ | |a Hochhaus, A. |b 7 |
| 700 | 1 | _ | |a Platzbecker, U. |b 8 |
| 700 | 1 | _ | |a Schafhausen, P. |b 9 |
| 700 | 1 | _ | |a Blau, I. W. |b 10 |
| 700 | 1 | _ | |a Verbeek, W. |b 11 |
| 700 | 1 | _ | |a Heidel, F. H. |b 12 |
| 700 | 1 | _ | |a Werner, M. |b 13 |
| 700 | 1 | _ | |a Kreipe, H. |b 14 |
| 700 | 1 | _ | |a Teleanu, V. |b 15 |
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| 700 | 1 | _ | |a Döhner, H. |b 17 |
| 700 | 1 | _ | |a Grießhammer, M. |b 18 |
| 700 | 1 | _ | |a Döhner, K. |b 19 |
| 773 | _ | _ | |a 10.1038/leu.2016.299 |0 PERI:(DE-600)2008023-2 |n 4 |p 889-895 |t Leukemia |v 31 |y 2017 |x 1476-5551 |
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