Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis
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Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. / Schlenk, R F; 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; Döhner, H; Grießhammer, M; Döhner, K.
in: LEUKEMIA, Jahrgang 31, Nr. 4, 04.2017, S. 889-895.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis
AU - Schlenk, R F
AU - Stegelmann, F
AU - Reiter, A
AU - Jost, E
AU - Gattermann, N
AU - Hebart, H
AU - Waller, C
AU - Hochhaus, A
AU - Platzbecker, U
AU - Schafhausen, P
AU - Blau, I W
AU - Verbeek, W
AU - Heidel, F H
AU - Werner, M
AU - Kreipe, H
AU - Teleanu, V
AU - Benner, A
AU - Döhner, H
AU - Grießhammer, M
AU - Döhner, K
PY - 2017/4
Y1 - 2017/4
N2 - 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.
AB - 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.
U2 - 10.1038/leu.2016.299
DO - 10.1038/leu.2016.299
M3 - SCORING: Journal article
C2 - 27774990
VL - 31
SP - 889
EP - 895
JO - LEUKEMIA
JF - LEUKEMIA
SN - 0887-6924
IS - 4
ER -