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

Czech version

Authors: B. Weinbergerová ¹; P. Čičátková ¹; M. Palová ²; L. Stejskal ³; P. Bělohlávková ⁴; J. Kissová ⁵; L. Walterová ⁶; H. Fraňková ⁶; O. Černá ⁷; L. Lakomá ⁸; M. Brejcha ⁹; J. Pelková ¹⁰; M. Schutzova ¹¹; J. Obernauerová ¹²; D. Nechvílová ¹³; E. Bogoczová ¹⁴; A. Hluší ²; E. Faber ²; M. Penka ⁵; Y. Brychtová ¹; L. Červinek ¹; M. Doubek ¹; P. Žák ⁴; J. Mayer ¹; Z. Ráčil ¹
Authors‘ workplace: Interní hematologická a onkologická klinika LF MU a FN Brno ¹; Hemato-onkologická klinika FN Olomouc ²; Klinika hematoonkologie FN Ostrava a LF OU ³; IV. Interní hematologická klinika FN a LF UK, Hradec Králové ⁴; Oddělení klinické hematologie FN Brno ⁵; Oddělení klinické hematologie Krajské nemocnice Liberec ⁶; Interní hematologická klinika FNKV a LF UK, Praha ⁷; Hematologická ambulance Nemocnice Havlíčkův Brod ⁸; Hematologická ambulance Nemocnice Nový Jičín ⁹; Hematologické a transfúzní oddělení Nemocnice Vsetín ¹⁰; Hematologicko-onkologické oddělení FN Plzeň ¹¹; Hematologicko-transfúzní oddělení Nemocnice Mladá Boleslav ¹²; Hematologicko-transfúzní oddělení Orlickoústecké nemocnice, Ústí nad Orlicí ¹³; Hematologická ambulance Vítkovické nemocnice, Ostrava-Vítkovice ¹⁴
Published in: Transfuze Hematol. dnes,23, 2017, No. 1, p. 30-40.
Category: Comprehensive Reports, Original Papers, Case Reports

Overview

Backgrounds:
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. Reduction ≥ 1/3 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.

Conclusion:
Our analysis confirmed the very good treatment efficacy of ruxolitinib in patients with myelofibrosis and polycythaemia vera on reduction of splenomegaly and alleviation of disease-associated symptoms. Ruxolitinib additionally led to the correction of haematocrit values in patients with polycythaemia vera. Haematological toxicity was generally low.

KEY WORDS:
ruxolitinib – myelofibrosis – polycythaemia vera – treatment outcome – drug toxicity

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Labels

Haematology Internal medicine Clinical oncology

Prevention and therapy of venous thromboembolism in cancer patients

Modern therapy of acute myeloid leukaemia

Autoimmune haemolytic anaemia – diagnosis and treatment, including erythrocyte transfusions

Article was published in

Transfusion and Haematology Today

2017 Issue 1