Development and Validation Results of the Russian MPN10 Form for Symptom Assessment in Patients with Myeloproliferative Neoplasms Compliant with International Recommendations

TI Ionova1,2, OYu Vinogradova3,4,5, EV Efremova6, AE Kersilova6, TP Nikitina1,2, MM Pankrashkina3, NM Porfir’eva2, A-PA Poshivai6, MS Fominykh6,7, DI Shikhbabaeva3, VA Shuvaev6

1 NI Pirogov Clinic for High Medical Technology, Saint Petersburg State University, 7-9 Universitetskaya nab., Saint Petersburg, Russian Federation, 199034

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

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

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

5 NI Pirogov Russian National Research Medical University, 1 Ostrovityanova str., Moscow, Russian Federation, 117997

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

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

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

For citation: Ionova TI, Vinogradova OYu, Efremova EV, et al. Development and Validation Results of the Russian MPN10 Form for Symptom Assessment in Patients with Myeloproliferative Neoplasms Compliant with International Recommendations. Clinical oncohematology. 2020;13(2):176–84 (In Russ).

DOI: 10.21320/2500-2139-2020-13-2-176-184


ABSTRACT

Aim. To develop a Russian version of MPN10 form for patients with myeloproliferative neoplasms (MPN) compliant with international recommendations.

Materials & Methods. The trial included 57 patients treated in 2019 at the Moscow Municipal Center for Hematology of the SP Botkin Clinical Hospital (n = 30) and the Russian Research Institute of Hematology and Transfusiology (n = 27). Among them there were 36 myelofibrosis, 9 polycythemia vera, and 12 essential thrombocythemia patients. Mean age of the patients was 54.6 years (standard deviation 15.9 years; age range 20–79 years). The male/female ratio was 23/34 (40.4 %/59.6 %). Underlying disease duration was from 1 month to 33 years (mean duration 7 years; standard deviation 8.6 years).

Results. A stable structure and a high inner consistency of the form as well as its reproducibility as a tool were demonstrated. The trial also confirmed its convergent and discriminant validity and satisfactory sensitivity to changes in a patient’s status.

Conclusion. The Russian MPN10 version can be used for symptom assessment in MPN patients in clinical practice and scientific research.

Keywords: symptom assessment form, myeloproliferative neoplasms, psychometric properties of the form, validation.

Received: January 15, 2020

Accepted: March 29, 2020

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Biology of Myeloproliferative Malignancies

AL Melikyan, IN Subortseva

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

For correspondence: Irina Nikolaevna Subortseva, PhD, 4а Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167; Tel.: +7(495)612-44-71; e-mail: soubortseva@yandex.ru

For citation: Melikyan AL, Subortseva IN. Biology of Myeloproliferative Malignancies. Clinical oncohematology. 2016;9(3):326-35 (In Russ).

DOI: 10.21320/2500-2139-2016-9-3-314-325


ABSTRACT

Chronic myeloproliferative diseases (WHO, 2001), or myeloproliferative neoplasms/malignancies (MPN) (WHO, 2008), are clonal diseases characterized by proliferation of one or more myelopoietic cell line in the bone marrow with signs of unimpaired terminal differentiation and is normally associated with changes in peripheral blood characteristics. The group of classical Ph-negative MPNs consists of polycythemia vera, essential thrombocythemia, primary myelofibrosis and unclassified MPNs. Acquired somatic mutations contributing to the pathogenesis of Ph-negative MPNs include JAK2 (V617F, exon 12), MPL, CALR gene mutations found in about 90 % of patients. However, these molecular events are not unique in the pathogenesis of the diseases. Mutations of other genes (ТЕТ2, ASXL1, CBL, IDH1/IDH2, IKZF1, DNMT3A, SOCS, EZH2, TP53, RUNX1, and HMGA2) are involved in formation of the disease phenotype. This review describes current concepts concerning the molecular biology of MPNs.


Keywords: chronic myeloproliferative diseases, myeloproliferative neoplasms, polycythemia vera, essential thrombocythemia, primary myelofibrosis, genes JAK2, CALR, and MPL.

Received: April 11, 2016

Accepted: April 11, 2016

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Polycythemia Vera: Literature Review and Own Data

I.N. Subortseva, T.I. Kolosheinova, E.I. Pustovaya, E.K. Egorova, A.M. Kovrigina, Yu.V. Pliskunova, T.V. Makarik, A.O. Abdullaev, A.L. Melikyan

Hematology Research Center under the Ministry of Health of the Russian Federation, 4а Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167

For correspondence: Irina Nikolaevna Subortseva, PhD, 4а Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167; Tel.: +7(495)612-44-72; e-mail: soubortseva@yandex.ru

For citation: Subortseva IN, Kolosheinova TI, Pustovaya EI, et al. Polycythemia Vera: Literature Review and Own Data. Clinical oncohematology. 2015;8(4):397–412 (In Russ).

DOI: 10.21320/2500-2139-2015-8-4-397-412


ABSTRACT

Background & Aims. Polycythemia vera (PV) refers to a group of classic Ph-negative myeloproliferative neoplasms characterized by panmyelosis, pancytosis, high risk of thrombotic and hemorrhagic complications, poor quality of life due to symptoms of tumor proliferation. Low-dose acetylsalicylic acid and phlebotomy/erythrocytapheresis are recommended for patients at low risk of complications, while the cytoreductive therapy (hydroxyurea or interferon alpha) is recommended for patients at high risk of thrombotic and hemorrhagic complications. At present, much attention is paid to the problems of diagnosis and treatment PV.

Methods. The article presents a brief description of the condition, a review of modern methods of treatment, results of observation over 100 PV patients who have been treated in the outpatient department of the Hematology Research Center. The observation period ranged from 6 to 262 months (median follow-up is 14 months).

Results. Patients’ age ranged from 23 to 80 years (median 56 years); 67 % of them were women, and 33 % were men. PV was diagnosed according to 2008 WHO guidelines. JAK2V617F mutation was detected in 100 % of cases. Splenomegaly was found in 70 % of patients. Plethoric symptoms (such as facial and hand hyperemia, and scleral injection) were found in 65 % of patients. All patients complained of headaches and dizziness, and 25 % of patients complained of itching. All patients received symptomatic therapy, antiaggregants, medications improving microcirculation, or antihypoxants. Patients were treated according to clinical guidelines: 49 % of them received hydroxyurea, 14 % IFN a-2b, 14 % a combination therapy (hydroxyurea and phlebotomy or IFN a-2b and phlebotomy), and 23 % of patients phlebotomy alone. Response to treatment was evaluated according to 2009 European LeukemiaNet criteria. Among all patients, the frequency of complete remission was 48 %, the partial response rate was 41 %, and no effect was achieved in 11 % of patients regardless they received therapy or not. The therapy was changed, if the treatment had proved to be ineffective, or in case of intolerance or complications. Complete remission was not achieved after switching treatment from one method to another.

Conclusion. Treatment of PV is mainly symptomatic. The effectiveness of first line therapy (complete remission) ranged from 14.5 to 71 %. It is necessary to conduct clinical trials designed to evaluate the effectiveness and safety of new targeted therapies.


Keywords: myeloproliferative neoplasms, polycythemia vera, JAK2V617F, target therapy.

Received: June 30, 2015

Accepted: November 5, 2015

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