Vol. 16 No. 2 (2023), CLINICAL TRIALS
Vol. 16 No. 2 (2023)
CLINICAL TRIALS

Quality of Life Changes in Patients with Chronic Immune Thrombocytopenia in the Process of Romiplostim Therapy, its Efficacy and Safety in the Real-World Setting: Results of a Multi-Center Observational Study

Published 20.02.2024
T.I. Ionova
Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; NI Pirogov Clinic for High Medical Technology, Saint Petersburg State University, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103
O.Yu. Vinogradova
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284; NI Pirogov Russian National Research Medical University, 1 Ostrovityanova ul., Moscow, Russian Federation, 117997; Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, 1 Samory Mashela ul., Moscow, Russian Federation, 117997
T.V. Shelekhova
VI Razumovskii Saratov State Medical University, 112 Bol’shaya Kazach’ya ul., Saratov, Russian Federation, 410012
D.G. Sherstnev
VI Razumovskii Saratov State Medical University, 112 Bol’shaya Kazach’ya ul., Saratov, Russian Federation, 410012
A.V. Proidakov
Komi Republican Oncology Dispensary, 46 Nyuvchimskoe sh., Syktyvkar, Republic of Komi, Russian Federation, 167904
E.V. Lyyurova
Komi Republican Oncology Dispensary, 46 Nyuvchimskoe sh., Syktyvkar, Republic of Komi, Russian Federation, 167904
M.M. Pankrashkina
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284
L.A. Mukha
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284
E.E. Markova
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284
N.V. Novitskaya
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284
T.I. Pospelova
Novosibirsk State Medical University, 52 Krasnyi pr-t, Novosibirsk, Russian Federation, 630091
T.N. Babaeva
Novosibirsk State Medical University, 52 Krasnyi pr-t, Novosibirsk, Russian Federation, 630091
N.B. Bulieva
I Kant Baltic Federal University, 14 Aleksandra Nevskogo ul., Kaliningrad, Russian Federation, 236041
G.B. Kuchma
Orenburg State Medical University, 6 Sovetskaya ul., Orenburg, Russian Federation, 460000
E.A. Andreevskaya
Krai Clinical Hospital No. 1, 7 Kokhanskogo ul., Chita, Russian Federation, 672038
E.E. Zinina
Clinical and Diagnostic Center (Hematology), Surgut District Clinical Hospital, 14 Energetikov ul., Surgut, Russian Federation, 628408
M.V. Frolova
Vologda Regional Clinical Hospital, 17 Lechebnaya ul., Vologda, Russian Federation, 160002
K.B. Trizna
Tomsk Regional Clinical Hospital, 96 I. Chernykh ul., Tomsk, Russian Federation, 634063
I.L. Shestopalova
SI Sergeev Krai Clinical Hospital No. 1, 9 Krasnodarskaya ul., Khabarovsk, Russian Federation, 680009
T.V. Shneider
Leningrad Regional Clinical Hospital, 45 korp. 1 lit. A Lunacharskogo pr-t, Saint Petersburg, Russian Federation, 194291
S.A. Volkova
Privolzhsky Research Medical University, 10/1 Minina i Pozharskogo pl., Nizhny Novgorod, Russian Federation, 603005
S.G. Zakharov
MF Vladimirskii Moscow Regional Research Clinical Institute, 61/2 Shchepkina ul., Moscow, Russian Federation, 129110
I.I. Mulina
Republican Hospital No. 1, National Medical Center, 4 Sergelyakhskoe sh., Yakutsk, Republic of Sakha (Yakutiya), Russian Federation, 677008
I.E. Solov’eva
Republican Hospital No. 1, National Medical Center, 4 Sergelyakhskoe sh., Yakutsk, Republic of Sakha (Yakutiya), Russian Federation, 677008
A.A. Myasnikov
VA Baranov Republican Clinical Hospital, 3 Pirogova ul., Petrozavodsk, Republic of Karelia, Russian Federation, 185002
A.A. Kuchin
VA Baranov Republican Clinical Hospital, 3 Pirogova ul., Petrozavodsk, Republic of Karelia, Russian Federation, 185002
L.B. Khvorostenko
Regional Clinical Hospital No. 1, 55 Kotovskogo ul., Tyumen, Russian Federation, 625023
N.M. Porfirieva
Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014
Tatyana Pavlovna Nikitina
Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014; NI Pirogov Clinic for High Medical Technology, Saint Petersburg State University, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103
V.V. Ptushkin
Moscow Municipal Center of Hematology, SP Botkin Municipal Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284; NI Pirogov Russian National Research Medical University, 1 Ostrovityanova ul., Moscow, Russian Federation, 117997; Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, 1 Samory Mashela ul., Moscow, Russian Federation, 117997
S.V. Gritsaev
Russian Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya ul., Saint Petersburg, Russian Federation, 191024
2023-2
PDF_2023-16-2_154-165 (Russian)

Keywords

chronic immune thrombocytopenia
quality of life
romiplostim
efficacy
safety
data in the real-world setting

How to Cite

1.
Ionova TI, Vinogradova OY, Shelekhova TV, et al. Quality of Life Changes in Patients with Chronic Immune Thrombocytopenia in the Process of Romiplostim Therapy, its Efficacy and Safety in the Real-World Setting: Results of a Multi-Center Observational Study. Клиническая онкогематология. 2024;16(2):154-165. doi:10.21320/2500-2139-2023-16-2-154-165
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Abstract

Aim. To study the quality of life in patients with chronic immune thrombocytopenia (ITP) in the process of romiplostim therapy and to assess the efficacy and safety of this drug in real-world setting.

Materials & Methods. The study enrolled adult patients with the confirmed chronic ITP diagnosis and indications for romiplostim therapy. Clinical parameters, RAND SF-36 and FACT-Th6 quality of life as well as FACIT-Fatigue scores were evaluated prior to romiplostim administration vs. 3, 6, and 12 months after the treatment onset. Patient satisfaction checklist was also administered at all study points after the start of therapy. The clinical efficacy of romiplostim was analyzed along with assessing response and time to response. To study the quality of life and fatigue changes, the Generalized Estimating Equation (GEE) method was used during the observation period. Significant fatigue changes were determined and compared in terms of the perception differences from patient’s and physician’s perspective.

Results. The study enrolled 60 chronic ITP patients treated with romiplostim in the real-world setting (mean age 51.9 years, 70 % women). The median thrombocyte count prior to romiplostim therapy was 18.5 × 109/L (interquartile range 10.8–22.3 × 109/л). On the enrollment date, 90 % of patients showed hemorrhagic syndrome. Overall response to romiplostim therapy was 98.3 % (complete response was achieved in 93.3 % of patients). After 6 months of therapy, 89.5 % of patients preserved response. After 3 months of therapy, hemorrhagic syndrome was eliminated in 81 % of patients, after 6 months the same was achieved in 93 % of patients. The median time to response was 4.4 weeks (95% confidence interval 3.6–5.3 weeks). Adverse events of grades 1/2 associated with romiplostim were reported in 6.7 % of patients. On romiplostim therapy, pronounced positive changes in quality of life were shown by all scales of the general questionnaire SF-36 and the targeted questionnaire FACT-Th6 (p < 0.001). The clearest improvements were observed in role-physical and role-emotional functioning. Already after 3 months of therapy, a considerable fatigue reduction was observed and sustained for the next 6 and 12 months of romiplostim administration (p < 0.001). During the therapy, the proportion of patients with fatigue impacting various aspects of functioning became considerably smaller. The vast majority of patients (85 %) were satisfied with the treatment. Discrepancies between patients’ and physicians’ evaluations of fatigue were also identified during the treatment.

Conclusion. The results of the present multi-center observational study demonstrate high efficacy and safety of romiplostim for chronic ITP patients in the real-world setting. Romiplostim therapy yields considerable quality of life improvement and fatigue reduction. To optimize the patient monitoring system and patient-centered ITP treatment in the real-world setting, it is advisable to use the standardized questionnaires assessing quality of life and fatigue.

PDF_2023-16-2_154-165 (Russian)

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