Zkušenosti s léčbou ruxolitinibem u pacientu s myelofibrózou a pravou polycytemií na českých hematologických pracovištích

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Title in English Experience with ruxolitinib in the treatment of myelofibrosis and polycythaemia vera at Czech haematological institutions
Authors

WEINBERGEROVÁ Barbora ČIČÁTKOVÁ Petra PALOVÁ M. STEJSKAL L. BĚLOHLÁVKOVÁ P. KISSOVÁ Jarmila WALTEROVÁ L. FRAŇKOVÁ H. ČERNÁ O. LAKOMÁ L. BREJCHA Martin PELKOVÁ J. SCHÜTZOVÁ M. OBERNAUEROVÁ J. NECHVÍLOVÁ D. BOGOCZOVÁ E. HLUŠÍ A. FABER E. PENKA Miroslav BRYCHTOVÁ Yvona ČERVINEK Libor DOUBEK Michael ŽÁK P. MAYER Jiří RÁČIL Zdeněk

Year of publication 2017
Type Article in Periodical
Magazine / Source Transfuze a hematologie dnes
MU Faculty or unit

Faculty of Medicine

Citation
Field Oncology and hematology
Keywords Drug toxicity; Myelofibrosis; Polycythaemia vera; Ruxolitinib; Treatment outcome
Description Ruxolitinib, a Janus kinase 1 and 2 inhibitor, demonstrated efficacy in patients with myelofibrosis and polycythaemia vera in the randomized COMFORT-I, COMFORT-II and RESPONSE studies. Ruxolitinib demonstrated superior durable reduction of splenomegaly and disease-associated symptoms, maintenance of haematocrit values, improvement in quality of life and overall survival compared to placebo or best available therapy. Material and Methods: A retrospective analysis evaluated efficacy and tolerability of ruxolitinib in a cohort of unselected myelofibrosis and polycythaemia vera patients treated in routine clinical practice at 14 Czech haematological centres from 2013 to 2016. Results: Myelofibrosis -a total of 62 patients with myelofibrosis treated with ruxolitinib were evaluated. The most frequent indication for treatment was concurrent splenomegaly and constitutional symptoms in 54 (87.1%) cases. ReductionASTERISK OPERATOR1/3 in palpable spleen length was achieved in 43 (72.9%) patients with baseline splenomegaly at a median of 4 weeks after starting therapy. Constitutional symptoms receded in 38 (92.7%) of 41 patients at a median of 4 weeks after starting therapy. While on ruxolitinib, eleven (18.0%) patients developed grade 3-4 anaemia and thirteen (21.3%) patients developed grade 3-4 thrombocytopenia. Forty six (74.2%) patients survived. Twenty five (40.3%) patients discontinued therapy, most frequently due to inefficacy (16.1% of patients) or haematological toxicity (8.1% of patients). Median duration of ruxolitinib therapy was 41 weeks. Polycythaemia vera -a total of 8 patients with polycythaemia vera treated with ruxolitinib because of resistance or intolerance of previous treatment was analysed. Six (75.0%) patients achieved complete remission. All patients experienced resolution of disease-associated symptoms. No patient developed grade 3 to 4 toxicity. At evaluation, all patients remained on ruxolitinib with a median duration of 32.5 weeks.
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