Treatment of Patients with Myelofi brosis and Polyscythemia Vera with Constitutional Symptoms in Routine Clinical Practice in the Russian Federation

Introduction. In the Russian Federation, there is insuffi cient data on approaches to the treatment of polycythemia vera (PV) and myelofi brosis (MF). There is also a lack of information on the assessment of treatment effectiveness in routine clinical practice.

Aim: to study treatment approaches for patients with MF (primary myelofi brosis (PMF), post-polycythemic myelofi brosis (PPMF), post-thrombocythemic myelofi brosis (PTMF)) and PV in routine clinical practice.

Materials and methods. A multicenter, non-interventional, prospective observational study included 2005 patients from 49 centers: PV — 1019 (50.8 %), MF — 986 (49.2 %) patients. Gender, age, diagnosis, disease duration, mutation status of the JAK2 V617F gene, risk groups, and quality of life were analyzed.

Results. The median age of patients with PV was 57 years, MF — 55 years. In one third of PV patients, a histological examination of the bone marrow trepan biopsy was not performed; in MF, trepanobiopsy was not conducted in less than 10 % of cases. When assessing the risk of thrombotic complications in PV, only 9.0 % of patients were classifi ed as high-risk, yet thrombotic events were reported in 13.7 % of cases. According to the IPSS index, 39.8 % of MF patients were classifi ed as intermediate-2 and high-risk, while 27.0 % were classifi ed by the DIPSS index. In more than 80 % of cases, patients receivedhydroxycarabamide. 76.9 % of MF patients responded to ruxolitinib therapy for a period of 60 months, but only 30 MF patients received it as fi rst-line therapy. Ruxolitinib was used as second-line therapy in 183 (18.6 %) MF patients and 54 (5.3 %) PV patients in the entire study group. Analysis of survival in over 2,000 patients allowed for the evaluation of overall survival (OS) and event-free survival (EFS), which are close to epidemiological estimates, indicating the persistent complexity of treating these patients. The proportion of MF patients with progressive disease was comparable to the proportion of patients who responded to therapy for all drugs except ruxolitinib. The use of ruxolitinib made it possible to achieve remission in most patients.

Conclusion. The study characterized treatment outcomes and clinical-demographic features of patients with MPN. Discrepancies exist between real-world clinical practice and the diagnostic and therapeutic algorithms presented in the clinical guidelines.

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