AUTOIMMUNE THROMBOCYTOPENIA

Efficacy Predictors of the Third-Line Tyrosine Kinase Inhibitor Therapy in Patients with Chronic Phase of Chronic Myeloid Leukemia: Results of a Multi-Center Study

AUTOIMMUNE THROMBOCYTOPENIA , , , ,

EG Lomaia1, VA Shuvaev2,3, TV Chitanava1, YuD Matvienko1, IS Martynkevich2, SV Voloshin2, EV Efremova2, ES Mileeva2, MS Fominykh4, AE Kersilova3, EV Karyagina5, NV Il’ina5, NV Dorofeeva6, NV Medvedeva6, AV Klimovich6, TV Shneider7, SA Stepanova7, NF Polezhaikovskaya7, NT Siordiya1, EI Sbityakova1, NS Lazorko1, EN Tochenaya1, DV Motorin1, NA Shnalieva1, YuA Alekseeva1, DB Zammoeva1, AYu Zaritskey1

1 VA Almazov National Medical Research Center, 2 Akkuratova ul., Saint Petersburg, Russian Federation, 197341

2 Russian Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya ul., Saint Petersburg, Russian Federation, 191024

3 VV Veresaev Municipal Clinical Hospital, 10 Lobnenskaya ul., Moscow, Russian Federation, 127644

4 AVA-PETER, Multispecialty Clinic “Skandinaviya”, 55A Liteinyi pr-t, Saint Petersburg, Russian Federation, 191014

5 Municipal Hospital No. 15, 4 Avangardnaya ul., Saint Petersburg, Russian Federation, 198205

6 Municipal Clinical Hospital No. 31, 3 Dinamo pr-t, Saint Petersburg, Russian Federation, 197110

7 Leningrad Regional Clinical Hospital, 45 korp. 2A Lunacharskogo pr-t, Saint Petersburg, Russian Federation, 194291

For correspondence: Tamara Vangelevna Chitanava, 2 Akkuratova ul., Saint Petersburg, Russian Federation, 197341; e-mail: chitanava.tamara@yandex.ru

For citation: Lomaia EG, Shuvaev VA, Chitanava TV, et al. Efficacy Predictors of the Third-Line Tyrosine Kinase Inhibitor Therapy in Patients with Chronic Phase of Chronic Myeloid Leukemia: Results of a Multi-Center Study. Clinical oncohematology. 2022;15(3):271–81. (In Russ).

DOI: 10.21320/2500-2139-2022-15-3-271-281

ABSTRACT

Background. The introduction of tyrosine kinase inhibitors (TKIs) into real-world clinical practice considerably improved the prognosis for patients with chronic myeloid leukemia (CML). However, during long-term follow-up, almost 1/2 and 2/3 of patients in the chronic phase (CP) discontinue TKI therapy of the first or second line, respectively. According to the Russian and International clinical guidelines, the third-line therapy should include allogeneic hematopoietic stem cell transplantation (allo-HSCT). And yet, some patients on the third-line therapy achieve and sustain optimal response on long-term TKI administration. Up to now, no clear-cut prognostic factors of TKI efficacy in the third-line therapy have been identified. This creates a challenge for treatment decision making after the failures of two lines of TKI therapy.

Aim. To assess the efficacy of the third-line TKI therapy in real-world clinical practice and to identify the factors affecting the long-term therapy outcomes in CML-CP.

Materials & Methods. The retrospective study enrolled 73 CML-CP patients aged ≥ 18 years, treated with TKIs in the third-line at 5 specialized institutions in Saint Petersburg and Leningrad Region. Among the patients there were 26 men (35 %). The median age of the patients was 51 years (range 25–88 years).

Results. With the median (range) third-line TKI therapy duration of 14 (1–120) months, the rate of complete cytogenetic response (CCR) was 30 % (n = 22) in the total cohort. The median time before achieving CCR was 9 (4–25) months. With the median follow-up time from the beginning of third-line TKI therapy till the last visit of 25 (3–136) months, progression to accelerated phase or blast crisis was observed only in 13 (17 %) out of 73 patients. Death was reported in 26 % (n = 19) of cases, among them 5 patients whose death was not CML-associated. At the last visit, 13/73 (18 %) patients were still on third-line TKI therapy. Direct and long-term therapy outcomes, including achievement of CCR and assessment of overall and progression-free survivals, were significantly better in patients with any cytogenetic response (CR) than in those without it or without complete hematologic response.

Conclusion. The implementation of TKIs in the third-line CML-CP therapy seems to be suitable for patients with at least some CR, especially if an optimal donor of hematopoietic stem cells is unavailable or if the risk of severe allo-HSCT complications is too high.

Keywords: chronic myeloid leukemia, chronic phase, complete cytogenetic response, tyrosine kinase inhibitors, third-line therapy, allogeneic hematopoietic stem cell transplantation, minimal residual disease.

Received: April 7, 2022

Accepted: June 20, 2022

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Stratification of Patients with Multiple Myeloma: State-of-the-Art and Prospects

AUTOIMMUNE THROMBOCYTOPENIA ,

AYu Aksenova1, AS Zhuk2, EI Stepchenkova1,3, SV Gritsaev4

1 Saint Petersburg State University, 7/9 Universitetskaya nab., Saint Petersburg, Russian Federation, 199034

2 ITMO National Research University, 49 lit. A Kronverkskii pr-t, Saint Petersburg, Russian Federation, 197101

3 NI Vavilov Institute of General Genetics, Saint Petersburg branch, 7/9 Universitetskaya nab., Saint Petersburg, Russian Federation, 199034

4 Russian Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya ul., Saint Petersburg, Russian Federation, 191024

For correspondence: Anna Yurevna Aksenova, PhD in Biology, 17 Botanicheskaya ul., Saint Petersburg, Russian Federation, 198504; Tel.: +7(812)428-40-09; e-mail: a.aksenova@spbu.ru; Sergei Vasilevich Gritsaev, MD, PhD, 16 2-ya Sovetskaya ul., Saint Petersburg, Russian Federation, 191024; Tel.: +7(812)717-54-68; e-mail: gritsaevsv@mail.ru

For citation: Aksenova AYu, Zhuk AS, Stepchenkova EI, Gritsaev SV. Stratification of Patients with Multiple Myeloma: State-of-the-Art and Prospects. Clinical oncohematology. 2022;15(3):259–70. (In Russ).

DOI: 10.21320/2500-2139-2022-15-3-259-270

ABSTRACT

In recent years, there has been a substantial progress in improving progression-free survival (PFS) and quality of life of multiple myeloma (MM) patients. This has become possible through implementation of novel drugs into clinical practice which were developed on the basis of multiomic molecular genetic studies in MM. The results of these studies also enabled to assess genetic heterogeneity of tumor cells in MM. That allowed to identify types and prevalence of single-nucleotide variations, structural chromosomal aberrations, and abnormal copy numbers of chromosomes in the genome of malignant plasma cells. It was shown that MM patients can have quite different spectra of detected genetic defects in the tumor. High genetic disease heterogeneity is one of the major causes of differences in drug efficacy and PFS. The present review comprehensively discusses the value of some chromosomal aberrations in risk stratification of MM patients. It describes the most prevalent aberrations, also those associated with high and low risk of early MM progression which have already been included in different international prognostic scores. Besides, the additional aberrations were determined which are potentially applicable in clinical practice. Special attention was paid to risk assessment in case a number of different chromosome rearrangements are identified in a patient. The review outlines challenges and prospects of dealing with the information on chromosome rearrangements in choosing the most optimal treatment strategy and assessing of its efficacy. In this context, emphasis is laid on integrating genetic data and such clinical parameters as age, comorbidity, renal failure, bone lesions, indications for autologous hematopoietic stem cell transplantation, etc.

Keywords: multiple myeloma, international staging systems, chromosome rearrangements, R-ISS, R2-ISS, mSMART, MASS.

Received: March 28, 2022

Accepted: June 5, 2022

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Prognostic Value of the Degree of Tumor Tissue Infiltration by CD15-Positive Granulocytes in Nodular Sclerosis Classical Hodgkin’s Lymphoma

AUTOIMMUNE THROMBOCYTOPENIA

EA Perfilova, DA D’yakonov, MS Minaev

Kirov Research Institute of Hematology and Transfusiology, 72 Krasnoarmeiskaya ul., Kirov, Russian Federation, 610027

For correspondence: Elena Aleksandrovna Perfilova, PhD in Veterinary Medicine, 72 Krasnoarmeiskaya ul., Kirov, Russian Federation, 610027; Tel.: +7(996)896-08-67; e-mail: lperf78@gmail.com

For citation: Perfilova EA, D’yakonov DA, Minaev MS. Prognostic Value of the Degree of Tumor Tissue Infiltration by CD15-Positive Granulocytes in Nodular Sclerosis Classical Hodgkin’s Lymphoma. Clinical oncohematology. 2022;15(3):253–8. (In Russ).

DOI: 10.21320/2500-2139-2022-15-3-253-258

ABSTRACT

Classical Hodgkin’s lymphoma (cHL) nodular sclerosis type is one of the most common malignant lymphoproliferative diseases among younger people. The tumor is considered to be potentially curable. However, despite successful application of standard treatment methods, primary resistance and relapses occur. At present, many researchers focus on studying the value of tumor microenvironment in the prognosis of the course and progression of cHL, aiming at identifying new therapeutic targets. The present paper shows that the relative count of CD15-positive granulocytes in patients with favorable course of the disease is significantly lower than in therapy-refractory patients. The cut-off of tumor microenvironment cells expressing CD15 was 8 %. The data obtained provide the basis for determining prognostic value of CD15-positive granulocytes in nodular sclerosis cHL and presenting this cell pool as a potential therapeutic target.

Keywords: classical Hodgkin’s lymphoma, nodular sclerosis, CD15 granulocytes, tumor microenvironment.

Received: April 8, 2022

Accepted: June 17, 2022

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Статистика Plumx английский

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Quality of Life and Efficacy of Triplet IxaRd Therapy in Relapsed/Refractory Multiple Myeloma: Results of a Multi-Center Pilot Real-World Study

AUTOIMMUNE THROMBOCYTOPENIA ,

TI Ionova1,2, OYu Vinogradova3,4,5, YuB Kochkareva3, EE Markova3, KD Kaplanov6, MN Shirokova6,7, TV Shelekhova8, AN Levanov8, AV Kopylova9, OYu Li10, TA Mitina11, OA Rukavitsyn12, PI Simashova12, LV Anchukova13, EN Babich14, SA Volkova15, DB Dasheeva16, MV Demchenkova17, SK Dubov18, TV Esenina19, LE Ivanova17, TL Kravchuk20, EV Rimashevskaya21, MT Savinova22, NO Saraeva23, NM Porfirieva1, TP Nikitina1,2, VV Ptushkin3

1 Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014

2 Saint Petersburg State University Hospital, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103

3 Moscow Municipal Center for Hematology, SP Botkin City Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284

4 NI Pirogov Russian National Research Medical University, 1 Ostrovityanova ul., Moscow, Russian Federation, 117997

5 Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, 1 Samory Mashela ul., Moscow, Russian Federation, 117997

6 SP Botkin City Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284

7 Volgograd Regional Clinical Oncology Dispensary, 78 Zemlyachki ul., Volgograd, Russian Federation, 400138

8 VI Razumovskii Saratov State Medical University, 112 Bolshaya Kazach’ya ul., Saratov, Russian Federation, 410012

9 Lipetsk Municipal Hospital No. 3 “Svobodnyi sokol”, 10 Ushinskogo ul., Lipetsk, Russian Federation, 398007

10 Sakhalin Regional Clinical Hospital, 430 Mira pr-t, Yuzhno-Sakhalinsk, Russian Federation, 693004

11 MF Vladimirskii Moscow Regional Research Clinical Institute, 61/2 Shchepkina ul., Moscow, Russian Federation, 129110

12 NN Burdenko Main Military Clinical Hospital, 3 Gospital’naya pl., Moscow, Russian Federation, 105094

13 Vologda Regional Clinical Hospital, 17 Lechebnaya ul., Vologda, Russian Federation, 160002

14 Yugry District Clinical Hospital, 40 Kalinina ul., Khanty-Mansiisk, Russian Federation, 628011

15 Privolzhsky Research Medical University, 10/1 Minina i Pozharskogo pl., Nizhny Novgorod, Russian Federation, 603005

16 Zabaikalsky Krai Oncology Dispensary, 104 Leningradskaya ul., Chita, Russian Federation, 672027

17 Irkutsk Regional Cancer Center, 32 Frunze ul., Irkutsk, Russian Federation, 664035

18 Krai Clinical Hospital No. 2, 55 Russkaya ul., Vladivostok, Russian Federation, 690105

19 Amurskaya Regional Clinical Hospital, 26 Voronkova ul., Blagoveshchensk, Russian Federation, 675000

20 Tomsk National Research Medical Center, 5 Kooperativnyi per., Tomsk, Russian Federation, 634009

21 Russian Medical Academy of Postgraduate Education, 38 Smolnaya ul., Moscow, Russian Federation, 125445

22 Municipal Clinical Hospital No. 16, 121 Gagarina ul., Kazan, Russian Federation, 420039

23 Irkutsk Regional Clinical Hospital, 100 Yubileinyi mikroraion, Irkutsk, Russian Federation, 664049

For correspondence: Tatyana Pavlovna Nikitina, MD, PhD, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; Tel.: +7(962)710-17-12; e-mail: qolife@mail.ru

For citation: Ionova TI, Vinogradova OYu, Kochkareva YuB, et al. Quality of Life and Efficacy of Triplet IxaRd Therapy in Relapsed/Refractory Multiple Myeloma: Results of a Multi-Center Pilot Real-World Study. Clinical oncohematology. 2022;15(3):240–52. (In Russ).

DOI: 10.21320/2500-2139-2022-15-3-240-252

ABSTRACT

Aim. To study quality of life (QoL) indicators and symptom profile as well as treatment satisfaction of patients with relapsed/refractory multiple myeloma (r/r MM) on triplet therapy based on ixazomib combined with lenalidomide and dexamethasone (IxaRd); to assess efficacy and safety of IxaRd protocol in real-world clinical practice.

Materials & Methods. The study enrolled 40 patients with confirmed r/r MM diagnosis, aged > 18 years, at 18 Russian health care institutions. They received at least one line of prior therapy and were IxaRd-eligible. Clinical and QoL indicators were assessed according to the RAND SF-36, and symptoms were evaluated using the ESAS-R questionnaire prior to IxaRd therapy and in 1, 3, 6, 9, 12, 15, and 18 months after its start. Besides, patients filled out checklists for assessment of treatment satisfaction at all time-points after therapy onset. The analysis of clinical IxaRd efficacy included assessment of treatment response by IMWG 2011 criteria, as well as response duration, overall survival (OS), and progression-free survival (PFS). The analysis of IxaRd safety was based on reporting adverse events (AEs), including severe ones (SAEs). To analyze patient-reported QoL and symptom changes during follow-up, GEE was used. To determine clinically meaningful changes, an effect size was calculated.

Results. The study included 40 r/r MM patients (mean age 63 ± 9 years, 65 % women). Median disease duration before IxaRd therapy onset was 55 months (range 2–99 months). 60 % of patients had IIIA/IIIB Durie-Salmon stage. With the median IxaRd duration of 7.5 months, clinical benefit rate was 71.8 %. Complete response was reported in 7.7 % of patients, stringent complete response in 2.6 % of patients, very good partial response in 5.1 % of patients, partial response in 30.8 % of patients, and minor response was achieved in 25.6 % of patients. Stable disease was reported in 15.4 % of patients, and disease progression was identified in 10.3 % patients, including immunochemical relapse in 1 patient. The median response duration was 16.3 months (95% confidence interval [95% CI] 15.4–17.3 months), the median PFS was 10.6 months (95% CI 6.3–16.3 months). The median OS was not reached; the 1-year OS after IxaRd therapy onset was 85.2 % (95% CI 71–99 %). AEs on IxaRd therapy were reported in 55 % of patients, SAEs were reported in 3 (7.5 %) patients. Positive QoL changes were observed on IxaRd therapy. QoL improvement was meaningful in terms of physical functioning, role-physical functioning, general health, vitality, and mental health, compared to baseline. Moreover, a considerable decrease of pain, fatigue, and nausea was revealed. On the whole, 87.5 % of patients were satisfied with the triplet IxaRd therapy.

Conclusion. The results of the present pilot study demonstrate efficacy and safety of the triplet IxaRd therapy (all per os) in real-world clinical practice from r/r MM patients’ and physicians’ perspective. Our data testify to the importance of patients’ feedback in the evaluation of therapy efficacy.

Keywords: multiple myeloma, relapsed/refractory, ixazomib, quality of life, real-world clinical practice.

Received: March 16, 2022

Accepted: June 6, 2022

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Efficacy and Safety of Pola-BR Combination in Relapsed/Refractory Aggressive В-Cell Non-Hodgkin’s Lymphomas: A Russian Multi-Center Study

AUTOIMMUNE THROMBOCYTOPENIA

OG Smykova1, AA Semenova2, YuB Chernykh3, TA Mitina3, AV Kildyushevskii3, SK Kravchenko4, AE Misyurina4, AU Magomedova4, EA Baryakh5, SV Samarina6, NP Volkov1, VV Markelov1, PV Kotselyabina1, LV Fedorova1, KV Lepik1, EV Kondakova1, LV Stelmakh1, VV Baykov1, NB Mikhailova1, IS Moiseev1, GS Tumyan2, EA Osmanov2, AD Kulagin1

1 RM Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation; IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022

2 NN Blokhin National Medical Cancer Research Center, 24 Kashirskoye sh., Moscow, Russian Federation, 115478

3 MF Vladimirskii Moscow Regional Research Clinical Institute, 61/2 Shchepkina ul., Moscow, Russian Federation, 129110

4 National Research Center for Hematology, 4 Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167

5 Municipal Clinical Hospital No. 52, 3 Pekhotnaya ul., Moscow, Russian Federation, 123182

6 Kirov Research Institute of Hematology and Transfusiology, 72 Krasnoarmeiskaya ul., Kirov, Russian Federation, 610027

For correspondence: Olesya Gennadevna Smykova, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022; Tel.: +7(981)144-67-95; e-mail: olesya.gen@gmail.com

For citation: Smykova OG, Semenova AA, Chernykh YuB, et al. Efficacy and Safety of Pola-BR Combination in Relapsed/Refractory Aggressive В-Cell Non-Hodgkin’s Lymphomas: A Russian Multi-Center Study. Clinical oncohematology. 2022;15(3):232–9. (In Russ).

DOI: 10.21320/2500-2139-2022-15-3-232-9

ABSTRACT

Aim. To analyze the first experience of administering polatuzumab vedotin combined with bendamustine and rituximab (Pola-BR) in clinical practice at some specialized institutions in the Russian Federation.

Materials & Methods. The prospective multi-center study enrolled 39 patients with relapsed/refractory aggressive В-cell non-Hodgkin’s lymphomas (B-NHLs): 31 (79 %) patients with diffuse large B-cell lymphoma, 7 (18 %) patients with primary mediastinal (thymic) large B-cell lymphoma, and 1 (3 %) patient with gray zone lymphoma. There were 20 men and 19 women aged 19–69 years (median 43 years). All the patients were treated with Pola-BR protocol: bendamustine 90 mg/m2 on Days 1 and 2, rituximab 375 mg/m2 on Day 1, and polatuzumab vedotin 1.8 mg/kg on Day 1 of each 21-day cycle. Full treatment with 6 cycles was completed by 19 patients. PET-CT was performed prior to therapy and after the 2nd, 4th, and 6th Pola-BR cycles. The tumor response was evaluated according to the Lugano 2014 criteria. The toxicity profile was assessed by means of reporting adverse events according to the NCI CTCAE, version 5.0.

Results. Objective response to the therapy, according to the Lugano 2014 criteria, was identified in 24 (61.5 %) patients: 19 (48.7 %) of them showed the complete response, and 5 (12.8 %) of them showed the partial one. Stable disease as best response to the therapy was reported in 3 (7.7 %) patients, disease progression was observed in 12 (30.8 %) patients. By the time of data analysis, the median follow-up duration was 16.8 months (range 5.3–24.2 months). The 2-year overall survival (OS) was 44 % (95% confidence interval [95% CI] 24–62 %), the median OS was 20.8 months. The 2-year progression-free survival (PFS) was 27 % (95% CI 12–43 %), the median PFS was 7.3 months. Adverse events of grade 3/4 included anemia (n = 4; 10.3 %), neutropenia (n = 15; 38.5 %), thrombocytopenia (n = 3; 7.7 %), and febrile neutropenia (n = 2; 5.1 %). In 2 patients with history of hepatitis B, the virus reactivation was identified on Pola-BR therapy. No cases of peripheral neuropathy were observed.

Conclusion. Results obtained in real-world clinical practice correspond to the previously published data and demonstrate that polatuzumab vedotin therapy (Pola-BR protocol) has a controllable toxicity profile and is, therefore, a promising chemotherapy method of relapsed/refractory aggressive B-NHL treatment.

Keywords: polatuzumab vedotin, aggressive B-NHLs, relapsed/refractory, treatment outcomes.

Received: March 13, 2022

Accepted: June 8, 2022

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  9. Dunleavy K, Wilson WH. Primary mediastinal B-cell lymphoma and mediastinal gray zone lymphoma: do they require a unique therapeutic approach? 2015;125(1):33–9. doi: 10.1182/blood-2014-05-575092.
  10. Dornan D, Bennett F, Chen Y, et al. Therapeutic potential of an anti-CD79b antibody-drug conjugate, anti-CD79b-vc-MMAE, for the treatment of non-Hodgkin lymphoma. 2009;114(13):2721–9. doi: 10.1182/blood-2009-02-205500.
  11. Pfeifer M, Zheng B, Erdmann T, et al. Anti-CD22 and anti-CD79B antibody drug conjugates are active in different molecular diffuse large B-cell lymphoma subtypes. 2015;29(7):1578–86. doi: 10.1038/leu.2015.48.
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In Memory of Professor A.Yu. Zaritskey

AUTOIMMUNE THROMBOCYTOPENIA

16 октября 2021 г. после продолжительной болезни скончался Андрей Юрьевич Зарицкий, один из ведущих ученых России в области гематологии, выдающийся клиницист, доктор медицинских наук, профессор, директор института онкологии и гематологии Национального медицинского исследовательского центра им. В.А. Алмазова.

Андрей Юрьевич Зарицкий родился 11 марта 1950 г. в Ленинграде. В 1973 г. окончил Первый Ленинградский медицинский институт им. акад. И.П. Павлова.

Его профессиональный путь начался на кафедре внутренних болезней этого же института, где он успешно закончил обучение по специальности «Гематология» в 1975 г. в ординатуре и 1978 г. в аспирантуре. Будучи одним из любимых учеников академика РАМН Владимира Андреевича Алмазова, Андрей Юрьевич под его руководством занимался прорывными для своего времени исследованиями микроокружения лейкозных клеток. В аспирантуре он работал над темой «Колониеобразующая способность костного мозга, колониестимулирующая активность лейкоцитов периферической крови и плазмы у гематологически здоровых лиц и больных хроническими лимфопролиферативными заболеваниями». В 1979 г. Андрей Юрьевич Зарицкий успешно защищает диссертацию по данной теме и становится кандидатом медицинских наук. Продолжая изучать биологию лейкозных клеток и их микроокружения, в 1996 г. он заканчивает работу над докторской диссертацией по теме: «Исследование фибробластных клеток костного мозга у больных с заболеваниями системы крови». В 2001 г. ему присвоено ученое звание профессора.

Андрей Юрьевич Зарицкий посвятил гематологии всю свою жизнь, активно занимался научной, лечебной и преподавательской деятельностью в избранной им области. В течение многих лет он был научным руководителем отделения гематологии Городской больницы № 31 г  Санкт-Петербурга, был профессором кафедры факультетской терапии Первого Санкт-Петербургского медицинского университета им. акад. И.П. Павлова. В 2008 г. Андрей Юрьевич организовал гематологическую службу в ФГБУ «НМИЦ им. В.А. Алмазова» Минздрава РФ и возглавил Институт гематологии НМИЦ. В институте проводились и проводятся работы в области фундаментальных, прикладных и трансляционных исследований злокачественных опухолей системы крови.

В Институте гематологии, в настоящее время переименованном в Институт онкологии и гематологии ФГБУ «НМИЦ им. В.А. Алмазова», под руководством проф. А.Ю. Зарицкого появились и успешно развивались научные и практические сферы гематологии. Созданы новые клинические отделения онкогематологии для детей и взрослых, где были внедрены инновационные методы химио- и иммунотерапии онкогематологических заболеваний, а также современные методы диагностики и мониторинга результатов терапии.

Андрей Юрьевич Зарицкий свободно владел английским языком, активно развивал международное сотрудничество с зарубежными коллегами. Он был активным членом рабочей группы по хроническому миелолейкозу и хроническому лимфолейкозу Европейской сети по изучению лейкозов (European LeukemiaNet), был экспертом международного уровня, соавтором рекомендаций European LeukemiaNet 2020 г. по лечению хронического миелоидного лейкоза. Благодаря его организаторским способностям и известности как ученого ему было предложено участие в нескольких международных широкомасштабных исследовательских проектах European LeukemiaNet, а также в научной работе, проводившейся Международным консорциумом в рамках 7-рамочной программы ЕС (SystemAge, WorkPackage 4).

Он принимал активное участие в развитии молекулярной диагностики и мониторинга результатов терапии хронического миелолейкоза в России, в т. ч. в организации международной стандартизации методики количественного определения гена BCR-ABL. Андрей Юрьевич Зарицкий был избран Послом в России крупнейшего онкологического центра США MD Anderson Cancer Centre, был представителем International Chronic Myeloid Leukemia Foundation (Международного фонда по ХМЛ) в России. В рамках программы клинического наставничества фонда iCMLf Андрей Юрьевич делился опытом и знаниями с коллегами из стран СНГ. Он был приглашенным спикером на многих международных конференциях, проводимых не только в России, но и в Украине, Республике Беларусь, Узбекистане, Казахстане.

Проф. А.Ю. Зарицкий вел регулярную просветительскую работу в рамках семинаров, региональных и всероссийских конференций, делился своими научными и клиническими знаниями с коллегами из разных регионов России.

Андрей Юрьевич также вел просветительскую деятельность среди пациентов, проводил школы в рамках программы «Право жить» и при поддержке Всероссийского общества онкогематологии «Содействие», был главным исследователем более чем в 40 клинических исследованиях инновационных лекарственных препаратов для лечения пациентов онкогематологического профиля.

Профессор Андрей Юрьевич Зарицкий был членом редакционной коллегии журналов «Трансляционная медицина», «Вестник гематологии».

За долгие годы работы он воспитал плеяду учеников, которые продолжают лечебную, исследовательскую и педагогическую работу как в НМИЦ им. В.А. Алмазова, так и в ведущих гематологических центрах нашей страны и мира. На его счету более 360 научных публикаций, несколько монографий и патентов. За заслуги в области здравоохранения Андрей Юрьевич Зарицкий награжден нагрудным знаком «Отличник здравоохранения».

Андрей Юрьевич поражал и заражал своей неисчерпаемой энергией, был человеком пытливого и проницательного ума, обширной эрудиции и колоссального трудолюбия. Он был искренним и открытым человеком, при этом очень принципиальным и требовательным как к себе, так и к своим ученикам и коллегам. Умел находить единомышленников, быть настоящим другом. Таким его помнят многочисленные коллеги и друзья.

Из воспоминаний коллег

I first met Professor Andrey Zaritskey during last year of medical school, he was assigned as our teacher in Hematology course. I owe him my choice of Hematology as my future specialty. At that time, we had very limited options for therapy of acute leukemia patients, with limited supportive care options, and I saw how difficult and acutely ill patients with hematologic malignancies are. AYu told me at that time: “These are very sick patients. Someone must treat these patients. And I think this should be you”. With those words, I continue to treat leukemia patients now at MD Anderson Cancer Center, for more than 25 years in my career. AYu was mentoring me in my laboratory research when I started my work at hematology hospital and later in clinic. He had unrelenting passion in science and always asked “out-of-the box” questions that could lead to discoveries. I inherited for life his enthusiasm and curiosity in laboratory and clinical research, that shaped my career, leading me to what I am now, physician-scientist. Professor Zaritskey was that rare hybrid of excellent clinician, thoughtful and forward-looking researcher, outstanding mentor and a wonderful human being. He has helped in many ways his multiple trainees, their relatives and friends, and was highly respected and loved by his colleagues and mentees. Professor Zaritskey was highly regarded nationally and internationally for his efforts in treatment of CML patients in Russia. He was integral part of multiple hematology schools disseminating the knowledge of CML therapy across multiple regions of the country. Dr. Zaritskey attended ASH and other international meetings, visited several times MD Anderson and became an ambassador at SOHO hematology meeting. AYu worked till the very last minute. Even being very ill confined to the hospital bed, he continued consulting responsibilities on his clinical unit, guided laboratory research and chaired international virtual conference. His dedication, passion to science and hematology and his nature of a human being with big heart will be always inspiring me in my life and professional career.

Marina Konopleva, MD, PhD, Professor, Member of the clinical faculty in the Departments of Leukemia and Stem Cell Transplantation of the MD Anderson Cancer Center (Houston, USA)

Я впервые встретила профессора Андрея Юрьевича Зарицкого на последнем курсе медицинского факультета, он был назначен нашим преподавателем гематологии. Именно благодаря ему я выбрала гематологию своей будущей специальностью. В то время у нас были очень ограниченные возможности лечения пациентов с острыми лейкозами, ограниченные варианты поддерживающей терапии, и я видела, насколько тяжелыми бывают пациенты с онкогематологическими заболеваниями. Андрей Юрьевич сказал мне тогда: «Эти пациенты очень больны. Кто-то должен их лечить. И я думаю, что это должны быть Вы». С этими словами я продолжаю лечить пациентов с лейкозом в онкологическом центре им. М.Д. Андерсона (MD Anderson Cancer Center) на протяжении всей своей карьеры, более 25 лет. Андрей Юрьевич был моим наставником в лабораторных исследованиях, когда я начинала работать в гематологической больнице, а затем — в клинике. Он обладал неослабевающей страстью к науке и всегда задавал нестандартные вопросы, которые могли привести к открытиям. Я на всю жизнь унаследовала его энтузиазм и жажду знаний в лабораторных и клинических исследованиях, которые определили мою карьеру и позволили мне стать тем, кем я сейчас являюсь, — врачом-ученым. Профессор Зарицкий сочетал в себе качества прекрасного клинициста, вдумчивого и прогрессивного исследователя, выдающегося наставника и замечательного человека, что встречается так редко. Он во многом помогал своим многочисленным ученикам, их родственникам и друзьям, его очень уважали и любили коллеги и подопечные. Профессор Зарицкий пользовался большим уважением на национальном и международном уровнях за приложенные усилия в области лечения пациентов с хроническим миелоидным лейкозом (ХМЛ) в России. Он был неотъемлемой частью нескольких гематологических школ, обеспечив распространение знаний о терапии ХМЛ во многих регионах страны. Доктор Зарицкий участвовал в конференциях ASH и других международных встречах, несколько раз посетил онкологический центр им. М.Д. Андерсона и стал послом на гематологической конференции SOHO. Андрей Юрьевич работал до последней минуты. Даже будучи очень больным и прикованным к больничной койке, он продолжал консультировать свое клиническое отделение, руководил лабораторными исследованиями и был председателем на Международной виртуальной конференции. Его преданность делу, страсть к науке и гематологии и качества человека с большим сердцем всегда будут вдохновлять меня в моей жизни и профессиональной карьере.

Марина Юрьевна Коноплева, д-р мед. наук, профессор, член клинического факультета отделения лейкоза и трансплантации стволовых клеток онкологического центра им. М.Д. Андерсона (Хьюстон, США)

European LeukemiaNet

Coordinator:

Prof. Dr. Dr. h. c. R. Hehlmann

ELN Foundation

Im Langgewann 45

69469 Weinheim

E-mail: hehlmann.eln@gmail.com

Date: 29.01.2022

The hematologic community lost a leader and international communicator, I lost a friend. Professor Dr. Andrey Zaritskey died in St. Petersburg after fighting a malicious disease for 4 years.

I met Andrey Zaritskey around 2005 when he on behalf of Paul’s University and the Almazov National Research Center joined the European LeukemiaNet (ELN) highlighting his comprehensive research interests and his cooperative spirit. Upon his invitation, I visited St. Petersburg in September 2006 starting 15 years of fruitful cooperation in the field of leukemia, particularly chronic myeloid leukemia (CML).

Promoting international cooperation, Andrey Zaritskey organized participation of young coworkers in international training and research activities around the world such as Dmitry Motorin in Bologna, Marina Konopleva in Houston and others, including attending the Annual ELN Symposia in Heidelberg and Mannheim/Germany and the Annual Workshops of the European Investigators on CML (EI-CML) across Europe. With Anthony Ho from Heidelberg University he embarked on an official Russian-German cooperation project on stem cells, and with Hagop Kantarjian and Elias Jabbour from MD Anderson Cancer Center (MDACC) in Houston on a Russian-American cooperation on clinical CML research. With the help of Natalya Lazorko of his team, he succeeded hosting in St. Petersburg an EI-CML Workshop in 2013, and an international workshop in cooperation with MDACC and ELN in 2017.

For promoting European integration of research in the field of leukemia Andrey Zaritskey was awarded the ELN Merit Award 2010. He was invited by ELN to join the international panel of experts to help develop the ELN recommendations for treating CML published in “Leukemia” in 2020.

An important aspect of Andrey Zaritskey’s promotional activity was familiarizing international cooperators with the scientific landscape and cultural heritage of St. Petersburg and Russia. Because of his scientific standing and outgoing, but always modest, personality he was well respected and invited to lectures and seminars all over Russia. By taking me along to some of these events he made me feel at home in Russia thereby deepening the spirit of cooperation.

Since 2008, Andrey Zaritskey established as director the new Institute of Hematology at the Almazov National Medical Research Center with research facilities and a transplantation unit. During the 13 years of his directorship he developed the institute into a well working institution with a dedicated, future-oriented team of young doctors and researchers and with international visibility.

The institute is well prepared for continuing top-level research and patient care in international cooperation under his successor, his long-term associate Professor Elza Lomaia.

Профессор Рудигер Хельманн, координатор Европейской сети по изучению лейкозов

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Pharmacoeconomic Analysis of CAR-T Cell Therapy in Diffuse Large B-Cell Lymphoma and B-Lineage Acute Lymphoblastic Leukemias

AUTOIMMUNE THROMBOCYTOPENIA

IV Gribkova, AA Zavyalov

Research Institute of Healthcare and Medical Management, 9 Sharikopodshipnikovskaya ul., Moscow, Russian Federation, 115088

For correspondence: Irina Vladimirovna Gribkova, PhD in Biology, 9 Sharikopodshipnikovskaya ul., Moscow, Russian Federation, 115088; Tel.: +7(916)078-73-90; e-mail: igribkova@yandex.ru

For citation: Gribkova IV, Zavyalov AA. Pharmacoeconomic Analysis of CAR-T Cell Therapy in Diffuse Large B-Cell Lymphoma and B-Lineage Acute Lymphoblastic Leukemias. Clinical oncohematology. 2022;15(2):205–12. (In Russ).

DOI: 10.21320/2500-2139-2022-15-2-205-212

ABSTRACT

Genetically modified Т-lymphocytes with chimeric antigen receptors (CAR-T cells) represent a new treatment strategy in relapsed/refractory B-cell malignant neoplasms. In 2017–2018 two CAR-T cell drugs, tisagenlecleucel and axicabtagene ciloleucel, were approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for clinical use in patients with refractory acute lymphoblastic leukemia and relapsed/refractory B-cell lymphomas. Due to its high efficacy, CAR-T cell therapy is increasingly becoming an integral part of clinical practice. However, this method of chemotherapy is very expensive. The mean cost of tisagenlecleucel is $475,000 and that of axicabtagene ciloleucel is $373,000. It is worth noting that these are only the drug prices which exclude other therapy-related costs. In the studies of 2018–2020 groups of researchers attempted to estimate the CAR-T cell therapy-associated costs. The aim of the present review is to analyze these studies and to assess the total treatment cost and expense structure, as well as to discuss the factors underlying the increasing costs and to explore opportunities to improve availability of the CAR-T technology, on the whole. The results showed that the mean cost of tisagenlecleucel therapy in B-cell lymphoma was $515,150 and that of axicabtagene ciloleucel therapy was $503,955. The treatment cost in acute lymphoblastic leukemia was $580,459. The major factors affecting the total therapy cost were CAR-T cell drug prices, severity of adverse events, and high tumor load prior to CAR-T cell drug infusion. It is agreed that the main opportunities to rise affordability of the CAR-T cell therapy lie in reducing the drug prices (for example, by means of medical facility-based production at its own expense), further therapy improvement aimed at less toxicity, and its implementation at earlier stages of tumor disease.

Keywords: B-cell lymphoma, acute lymphoblastic leukemia, CAR-T cell therapy, chimeric antigen receptor, tisagenlecleucel, axicabtagene ciloleucel, costs, review.

Received: October 29, 2021

Accepted: February 15, 2022

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Статистика Plumx английский

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Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Myelodysplastic Syndromes with Trisomy 8 and/or Monosomy 7

AUTOIMMUNE THROMBOCYTOPENIA

MV Latypova, NN Mamaev, TL Gindina, AI Shakirova, OV Paina, AA Osipova, TV Rudakova, EV Morozova, SN Bondarenko, LS Zubarovskaya

RM Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation; IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022

For correspondence: Prof. Nikolai Nikolaevich Mamaev, MD, PhD, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022; e-mail: nikmamaev524@gmail.com

For citation: Latypova MV, Mamaev NN, Gindina TL, et al. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Myelodysplastic Syndromes with Trisomy 8 and/or Monosomy 7. Clinical oncohematology. 2022;15(2):198–204. (In Russ).

DOI: 10.21320/2500-2139-2022-15-2-198-204

ABSTRACT

The study assessed the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in 34 patients with cytogenetically verified variants of myelodysplastic syndrome (MDS) with trisomy 8 and/or monosomy 7, who were treated at the RM Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation from 2013 to 2020. Both adult and pediatric MDS were analyzed without excluding the variants with two additional chromosomal abnormalities or complex karyotype. The study revealed that а) allo-HSCT should be performed in the treatment of both MDS variants; b) the outcomes of trisomy 8 treatment appeared to be better; c) children with monosomy 7 showed a higher rate of toxic complications in allo-HSCT.

Keywords: myelodysplastic syndromes, cytogenetic variants, trisomy 8, monosomy 7, allo-HSCT.

Received: October 2, 2021

Accepted: March 6, 2022

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Статистика Plumx английский

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Current Quality-of-Life Aspects in Patients with Classical Ph-Negative Myeloproliferative Neoplasms in the Russian Federation: Results and Discussion of the National Observational Program MPN-QoL-2020

AUTOIMMUNE THROMBOCYTOPENIA , , ,

TI Ionova1,2,3,*, EA Andreevskaya4,*, EN Babich5,*, NB Bulieva6,7,*, OYu Vinogradova8,9,10,*, EM Volodicheva11,*, SV Voloshin12,13,14,*, NN Glonina15,*, SK Dubov16,*, NB Esef’eva17,*, AYu Zaritskey18,*, EE Zinina19,*, MO Ivanova20,*, TYu Klitochenko21,*, AV Kopylova22,*, AD Kulagin23,*, GB Kuchma24,25,*, OYu Li26,*, EG Lomaia18,*, AL Melikyan27,*, VYa Melnichenko3,*, SN Menshakova28,*, NV Minaeva29,*, TA Mitina30,*, EV Morozova23,*,TP Nikitina1,2,*, OE Ochirova31,*, AS Polyakov13,*, TI Pospelova32,*, AV Proidakov33,*, OA Rukavitsyn34,*, GSh Safuanova35,36,*, IN Subortseva27,*, MS Fominykh37,*, MV Frolova38,*, TV Shelekhova39,*, DG Sherstnev39,*, TV Shneider40,*, VA Shuvaev12,41,*, ZK Abdulkhalikova23,†, LV Anchukova38,†, IA Apanaskevich15,†, AN Arnautova22,†, MV Barabanshchikova23,†, NV Berlina34,†, AP Bityukov34,†, EA Gilyazitdinova27,†, VI Gilmanshina36,†, EK Egorova27,†, EV Efremova12,†, EB Zhalsanova31,†, EN Kabanova19,†, OB Kalashnikova20,†, AE Kersilova41,†, TI Kolosheinova27,†, PM Kondratovskii16,†, EV Koroleva28,†, AN Kotelnikova34,†, NA Lazareva16,†, NS Lazorko18,†, EV Lyyurova33,†, AS Lyamkina32,†, YuN Maslova20,†, ES Mileeva12,†, NE Mochkin3,†, EK Nekhai16,†, YaA Noskov13,†, ES Osipova29,†, MM Pankrashkina8,†, EV Potanina16,†, OD Rudenko25,†, TYu Rozhenkova36,†, EI Sbityakova18,†, NT Siordiya18,†, AV Talko16,†, EI Usacheva42,†, YuB Chernykh30,†, TV Chitanava18,†, KS Shashkina27,†, DI Shikhbabaeva8,†, KS Yurovskaya23,†

1 Saint Petersburg State University Hospital, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103

2 Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014

3 NI Pirogov National Medical and Surgical Center, 70 Nizhnyaya Pervomaiskaya ul., Moscow, Russian Federation, 105203

4 Krai Clinical Hospital No. 1, 7 Kokhanskogo ul., Chita, Russian Federation, 672038

5 Yugry District Clinical Hospital, 40 Kalinina ul., Khanty-Mansiisk, Russian Federation, 628011

6 I Kant Baltic Federal University, 14 Aleksandra Nevskogo ul., Kaliningrad, Russian Federation, 236041

7 Clinical Hospital of Kaliningrad Region, 74 Klinicheskaya ul., Kaliningrad, Russian Federation, 236016

8 Moscow Municipal Center for Hematology, SP Botkin City Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284

9 NI Pirogov Russian National Research Medical University, 1 Ostrovityanova ul., Moscow, Russian Federation, 117997

10 Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, 1 Samory Mashela ul., Moscow, Russian Federation, 117997

11 Tula Regional Clinical Hospital, 1A korp. 1 Yablochkova ul., Tula, Russian Federation, 300053

12 Russian Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya ul., Saint Petersburg, Russian Federation, 191024

13 SM Kirov Military Medical Academy, 6 Akademika Lebedeva ul., Saint Petersburg, Russian Federation, 194044

14 II Mechnikov North-Western State Medical University, 47 Piskarevskii pr-t, Saint Petersburg, Russian Federation, 195067

15 SI Sergeev Krai Clinical Hospital No. 1, 9 Krasnodarskaya ul., Khabarovsk, Russian Federation, 680009

16 Krai Center of Hematology, Krai Clinical Hospital No. 2, 55 Russkaya ul., Vladivostok, Russian Federation, 690105

17 Ulyanovsk Regional Clinical Hospital, 7 III Internatsionala ul., Ulyanovsk, Russian Federation, 432017

18 VA Almazov National Medical Research Center, 2 Akkuratova ul., Saint Petersburg, Russian Federation, 197341

19 Surgut District Clinical Hospital, 14 Energetikov ul., Surgut, Russian Federation, 628408

20 Clinical and Diagnostic Center, IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022

21 Volgograd Regional Clinical Oncology Dispensary, 78 Zemlyachki ul., Volgograd, Russian Federation, 400138

22 Lipetsk Municipal Hospital No. 3 “Svobodnyi sokol”, 10 Ushinskogo ul., Lipetsk, Russian Federation, 398007

23 RM Gorbacheva Research Institute of Pediatric Oncology, Hematology and Transplantation, Pavlov University, 12 Rentgena ul., Saint Petersburg, Russian Federation, 197022

24 Orenburg State Medical University, 6 Sovetskaya ul., Orenburg, Russian Federation, 460000

25 Orenburg Regional Clinical Hospital, 23 Aksakova ul., Orenburg, Russian Federation, 460018

26 Sakhalin Regional Clinical Hospital, 430 Mira pr-t, Yuzhno-Sakhalinsk, Russian Federation, 693004

27 National Research Center for Hematology, 4 Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167

28 Regional Clinical Hospital, 105 Peterburgskoe sh., Tver, Russian Federation, 170036

29 Kirov Research Institute of Hematology and Transfusiology, 72 Krasnoarmeiskaya ul., Kirov, Russian Federation, 610027

30 MF Vladimirskii Moscow Regional Research Clinical Institute, 61/2 Shchepkina ul., Moscow, Russian Federation, 129110

31 NA Semashko Republican Clinical Hospital, 12 Pavlova ul., Ulan-Ude, Russian Federation, 670031

32 Novosibirsk State Medical University, 52 Krasnyi pr-t, Novosibirsk, Russian Federation, 630091

33 Komi Republican Oncology Dispensary, 46 Nyuvchimskoe sh., Syktyvkar, Republic of Komi, Russian Federation, 167904

34 NN Burdenko Main Military Clinical Hospital, 3 Gospital’naya pl., Moscow, Russian Federation, 105229

35 Bashkir State Medical University, 3 Lenina ul., Ufa, Republic of Bashkortostan, Russian Federation, 450008

36 GG Kuvatov Republican Clinical Hospital, 132 Dostoevskogo ul., Ufa, Republic of Bashkortostan, Russian Federation, 450005

37 Multispecialty Clinic “Skandinaviya”, AVA-PETER, 55A Liteinyi pr-t, Saint Petersburg, Russian Federation, 191014

38 Vologda Regional Clinical Hospital, 17 Lechebnaya ul., Vologda, Russian Federation, 160002

39 VI Razumovskii Saratov State Medical University, 6/9 53rd Strelkovoi Divizii ul., Saratov, Russian Federation, 410028

40 Leningrad Regional Clinical Hospital, 45 korp. 2A Lunacharskogo pr-t, Saint Petersburg, Russian Federation, 194291

41 VV Veresaev Municipal Clinical Hospital, 10 Lobnenskaya ul., Moscow, Russian Federation, 127644

42 SM Clinic, 19 korp. 1 Udarnikov pr-t, Saint Petersburg, Russian Federation, 195279

* Coordinators and members of Expert Panel.

† Program participants.

For correspondence: Tatyana Pavlovna Nikitina, MD, PhD, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; e-mail: qolife@mail.ru

For citation: Ionova TI, Andreevskaya EA, Babich EN, et al. Current Quality-of-Life Aspects in Patients with Classical Ph-Negative Myeloproliferative Neoplasms in the Russian Federation: Results and Discussion of the National Observational Program MPN-QoL-2020. Clinical oncohematology. 2022;15(2):176–97. (In Russ).

DOI: 10.21320/2500-2139-2022-15-2-176-197

ABSTRACT

Background. The National Observational Program MPN-QoL-2020 was aimed at collecting the data on QoL (quality of life) characteristics and symptoms as well as patient- and physician-related disease and treatment perceptions in classical Ph-negative myeloproliferative neoplasms (MPN) in the Russian Federation.

Aim. Using new standardized forms, to analyze the quality of life among patients with various MPNs, to characterize ubiquitous symptoms and their effect on quality of life among the myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) patients as well as to describe the perceptions of disease- and therapy-associated problems as reported by patients and hematologists treating them.

Materials & Methods. The study enrolled 1100 patients with Ph-negative MPNs (355 MF, 408 PV, and 337 ET patients at the mean age of 58 ± 14 years, 61 % women). The study also involved 100 hematologists (mean age of 42 ± 12 years, 85 % women) from 37 health and preventive facilities in 8 Federal districts of the Russian Federation. The patients contributed to the study by one-time completing a special MPN10 form for MPN symptom assessment, a special QoL questionnaire HM-PRO for hematological malignancy patients, as well as a patient checklist. The task of hematologists consisted in one-time filling out of a physician checklist and completing the medical records of all the enrolled MPN patients.

Results. For the first time in the Russian Federation, the real clinical practice yielded the data on the quality of life in Ph-negative MPN patients, symptom profiles in different MPNs, and the extent of their effect on everyday life. QoL impairments mostly relate to physical and emotional functioning of MPN patients and to feeding and drinking regime, but rarely to social functioning. More than 1/3 of patients with Ph-negative MPNs reported on considerable QoL impairments. Absolute majority of patients complain of weakness: 92.6 % in MF, 83.7 % in PV, and 82 % in ET. The profiles of relevant symptoms and their intensity differ in various MPNs. The study identified the symptoms which need most to be corrected, both in the view of patients and physicians. There were established differences between patient- and doctor-reported evaluations of the attitude to the disease and treatment as well as the aspects for improvement in physician-patient relationship.

Conclusion. The National Observational Program MPN-QoL-2020 has resulted in characterization of QoL impairments in MPN patients in Russia. It determined the spectrum of particular disease and treatment challenges specific to these patients. Moreover, their unmet needs were updated. The outcomes of MPN-QoL-2020 can serve as a basis for the guidelines for QoL improvement/maintenance in Ph-negative MPNs and for activities aimed at raising MPN patients’ awareness about the disease and its treatment.

Keywords: classical Ph-negative myeloproliferative neoplasms, polycythemia vera, essential thrombocythemia, primary myelofibrosis, quality of life, MPN10 form.

Received: October 12, 2021

Accepted: February 10, 2022

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Статистика Plumx английский

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