Quality of Life and Symptom Profile in Polycythemia Vera Patients on Ruxolitinib Therapy in a Real-World Setting: Results of a Multi-Center Prospective Observational Study

AIM. To assess the changes in quality of life and symptom profile in polycythemia vera (PV) patients during ruxolitinib treatment as well as to study the efficacy and safety of this drug in a real-world setting.

MATERIALS & METHODS. This multi-center prospective observational study enrolled 50 PV patients from 9 medical centers of the Russian Federation (median age 58 years, male/female ratio 1:1). All patients had indications for ruxolitinib therapy. The median disease duration was 48.3 months. Splenomegaly was observed in 86 % of cases; stratification of patients yielded an intermediate/high-risk group with 76 % and low-risk group with 24 % of them. Patients completed SF-36 and MPN10 questionnaires prior to treatment and then 1, 3, 6, 9, and 12 months after therapy onset along with a patient satisfaction checklist. Clinic response was assessed in accordance with the National Clinical Guidelines. The total MPN10 score decrease by ≥ 50 % during the treatment was regarded as a symptom-related response. The statistical analysis of the parameters changing over time was performed using generalized estimating equations (GEE).

RESULTS. Ruxolitinib therapy for 12 months resulted in pronounced positive changes in quality of life and symptom reduction in patients with both intermediate/high and low risk of thrombotic complications (p < 0.001). Therapy response with total MPN10 score decrease ≥ 50 % was observed in 59 % of patients after 1 month and in 70 % of patients in subsequent treatment periods. Nine months after therapy onset, a complete clinical and hematologic response was achieved in 57.8 % and a partial one in 37.8 % of patients; after 12 months it was reported in 59.2 % and 36.7 % of patients, respectively. Hematocrit improvement was achieved in more than 85 % of cases in different treatment periods. Adverse events (AEs), commonly grade 1/2, were identified in 11 (22 %) patients, and serious AEs (ischemic stroke and second tumor) were detected in 2 patients. During the follow-up period, the vast majority of PV patients reported improved well-being and satisfaction with ruxolitinib treatment outcomes.

CONCLUSION. In a real-world setting, ruxolitinib therapy showed good clinical efficacy, favorable safety profile as well as pronounced positive changes in quality of life and disease symptoms throughout 12 months after therapy onset.

• T.I. Ionova Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; Saint Petersburg State University Hospital, 154 Fontanki nab., Saint Petersburg, Russian Federation, 190103 https://orcid.org/0000-0002-9431-5286 (unauthenticated)
• Tatyana Pavlovna Nikitina Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; Saint Petersburg State University Hospital, 154 Fontanki nab., Saint Petersburg, Russian Federation, 190103 https://orcid.org/0000-0002-8279-8129 (unauthenticated)
• A.V. Proidakov Komi Republican Oncology Dispensary, 46 Nyuvchimskoe sh., Syktyvkar, Republic of Komi, Russian Federation, 167904 https://orcid.org/0000-0002-5013-6614 (unauthenticated)
• E.V. Lyurova Komi Republican Oncology Dispensary, 46 Nyuvchimskoe sh., Syktyvkar, Republic of Komi, Russian Federation, 167904 https://orcid.org/0009-0000-3413-3563 (unauthenticated)
• Е.M. Volodicheva Tula Regional Clinical Hospital, 1A korp. 1 Yablochkova ul., Tula, Russian Federation, 300053 https://orcid.org/0000-0002-3011-9729 (unauthenticated)
• L.A. Mkhitaryan Bashkir State Medical University Hospital, 2 Shafieva ul., Ufa, Republic of Bashkortostan, Russian Federation, 450083 https://orcid.org/0000-0003-2597-981X (unauthenticated)
• Yu.P. Martinova Clinical Oncology Dispensary No. 1, 146 Dimitrova ul., Krasnodar, Russian Federation, 350040 https://orcid.org/0009-0006-2045-7026 (unauthenticated)
• T.V. Chitanava Clinical Oncology Dispensary No. 1, 146 Dimitrova ul., Krasnodar, Russian Federation, 350040 https://orcid.org/0000-0002-9390-9154 (unauthenticated)
• N.B. Esefeva Ulyanovsk Regional Clinical Hospital, 7 III Internatsionala ul., Ulyanovsk, Russian Federation, 432017
• М.O. Ivanova Clinical and Diagnostic Center, Pavlov University, 6/8 korp. 5 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022 https://orcid.org/0000-0003-1272-3583 (unauthenticated)
• Т.I. Pospelova Municipal Clinical Hospital No 2, 21 Polzunova ul., Novosibirsk, Russian Federation, 630051 https://orcid.org/0000-0002-1261-5470 (unauthenticated)
• A.S. Lyamkina ГБУЗ НСО «Городская клиническая больница № 2», ул. Ползунова, д. 21, Новосибирск, Российская Федерация, 630051 https://orcid.org/0000-0002-2516-0778 (unauthenticated)
• E.A. Knyazeva Novgorod Regional Clinical Hospital, 14 Pavla Levitta ul., Velikiy Novgorod, Russian Federation, 173008
• Е.G. Lomaia VA Almazov National Medical Research Center, 2 Akkuratova ul., Saint Petersburg, Russian Federation, 197341 https://orcid.org/0000-0003-3290-7961 (unauthenticated)
• N.S. Lazorko VA Almazov National Medical Research Center, 2 Akkuratova ul., Saint Petersburg, Russian Federation, 197341 https://orcid.org/0000-0002-7199-3252 (unauthenticated)
• K.P. Novoselov Novgorod Regional Clinical Hospital, 14 Pavla Levitta ul., Velikiy Novgorod, Russian Federation, 173008
• D.A. Lipatova Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014
• N.M. Porfirieva Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014 https://orcid.org/0000-0003-3329-2743 (unauthenticated)
• А.D. Kulagin Pavlov University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022 https://orcid.org/0000-0002-9589-4136 (unauthenticated)

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