Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis
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 myelofibro...
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| Main Authors: | , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
2017
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| In: |
Leukemia
Year: 2016, Volume: 31, Issue: 4, Pages: 889-895 |
| ISSN: | 1476-5551 |
| DOI: | 10.1038/leu.2016.299 |
| Online Access: | Verlag, Volltext: http://dx.doi.org/10.1038/leu.2016.299 Verlag, Volltext: https://www.nature.com/articles/leu2016299 
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| Author Notes: | R.F. Schlenk, F. Stegelmann, A. Reiter, E. Jost, N. Gattermann, H. Hebart, C. Waller, A. Hochhaus, U. Platzbecker, P. Schafhausen, I.W. Blau, W. Verbeek, F.H. Heidel, M. Werner, H. Kreipe, V. Teleanu, A. Benner, H. Döhner, M. Grießhammer and K. Döhner |
| Summary: | 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) |
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| Item Description: | Gesehen am 11.05.2018 Advance online publication, 29 November 2016 |
| Physical Description: | Online Resource |
| ISSN: | 1476-5551 |
| DOI: | 10.1038/leu.2016.299 |