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Персонализация лечения пациентов с иммунной тромботической тромбоцитопенической пурпурой - Журнал Терапевтический архив №8 Вопросы лечения 2025
Персонализация лечения пациентов с иммунной тромботической тромбоцитопенической пурпурой
Галстян Г.М., Клебанова Е.Е., Мамлеева С.Ю., Авдонин П.В., Фидарова З.Т., Дроков М.Ю., Паровичникова Е.Н. Персонализация лечения пациентов с иммунной тромботической тромбоцитопенической пурпурой. Терапевтический архив. 2025;97(8):711–718. DOI: 10.26442/00403660.2025.08.203326
© ООО «КОНСИЛИУМ МЕДИКУМ», 2025 г.
© ООО «КОНСИЛИУМ МЕДИКУМ», 2025 г.
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Аннотация
Обоснование. Лечение иммунной тромботической тромбоцитопенической пурпуры (иТТП) включает в себя терапевтические плазмообмены (ТПО) и иммуносупрессию. Добавление к терапии каплацизумаба позволило улучшить результаты лечения иТТП. Однако имеющиеся схемы терапии ориентированы на длительность применения препаратов и клинический ответ, а не на активность ADAMTS13.
Цель. Оценить эффективность терапии иТТП, ориентированной на активность ADAMTS13.
Материалы и методы. Лечение пациентов с иТТП начинали с ТПО, преднизолона (1 мг/кг) и каплацизумаба (10 мг/сут). ТПО прекращали после увеличения количества тромбоцитов крови >150×109/л. Лишь после этого начинали ритуксимаб (375 мг/м2 еженедельно). Каплацизумаб отменяли при достижении частичной ремиссии (ADAMTS13>20%), а ритуксимаб и глюкокортикоиды – после достижения полной ремиссии (ADAMTS13>40%). Анализировали число тромбоцитов, шистоцитов, концентрации гемоглобина, гаптоглобина, активности лактатдегидрогеназы, ADAMTS13, титр ингибитора ADAMTS13, количество процедур ТПО, объем замененной плазмы, количество тромбоцитов, время до их увеличения >150×109/л, достижения частичной и полной ремиссии. Данные представлены как медиана и межквартильный интервал.
Результаты. С 2021 по 2025 г. диагноз тромботической тромбоцитопенической пурпуры подтвержден у 102 пациентов. В исследование включены 35 пациентов. Количество тромбоцитов >150×109/л достигнуто после 4 (3–5) процедур ТПО через 4 (3–4,5) дня, при этом обменено 11 395 (7241–16 343) мл плазмы. Частичная ремиссия достигнута у 100% пациентов, длительность терапии каплацизумабом составила 23 (12–30) дня. Проведено от 4 до 8 введений ритуксимаба (медиана 4), полная ремиссия достигнута у 33 из 35 пациентов, у 2 получена только частичная ремиссия, им проведена терапия бортезомибом, в связи с неэффективностью последней у 1 пациентки проведена терапия моноклональным анти-CD38 антителом. Вероятность достижения полной ремиссии составила 97,1%.
Заключение. Длительность терапии каплацизумабом, ритуксимабом и глюкокортикоидами у пациентов с иТТП должна определяться достижением целевых значений активности ADAMTS13.
Ключевые слова: тромботическая тромбоцитопеническая пурпура, ADAMTS13, плазмообмен, глюкокортикоиды, каплацизумаб, ритуксимаб, бортезомиб
Aim. To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity.
Materials and methods. Treatment of patients with iTTP was started with PEX, prednisolone (1 mg/kg) and caplacizumab (10 mg/day). PEX was discontinued after an increase of platelet count >150×109/L. Only after PEX cessation treatment with rituximab (375 mg/m2 weekly) was started. Caplacizumab was discontinued when partial remission (ADAMTS13>20%) was achieved. Rituximab and glucocorticoids were discontinued when complete remission (ADAMTS13>40%) was achieved. Platelet count, schistocyte count, haemoglobin, haptoglobin, lactate dehydrogenase activity, ADAMTS13, ADAMTS13 inhibitor titre, number of PEX, plasma volume replaced, time to increase platelet count >150×109/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range.
Results. From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts >150×109/L were achieved after 4 (3–5) PEX procedures in 4 (3–4.5) days. In total, 11 395 (7241–16 343) ml of plasma were exchanged. Partial remission was achieved in 100% of patients, the duration of caplacizumab therapy was 23 (12–30) days. Rituximab was administered from 4 to 8 times (median 4), complete remission was achieved in 33 out of 35 patients, 2 patients achieved only partial remission, they were treated with bortezomib and 1 with anti-CD38 monoclonal antibody. The probability of complete remission was 97.1%.
Conclusion. The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.
Keywords: thrombotic thrombocytopenic purpura, ADAMTS13, plasma exchange, glucocorticoids, caplacizumab, rituximab, bortezomib
Цель. Оценить эффективность терапии иТТП, ориентированной на активность ADAMTS13.
Материалы и методы. Лечение пациентов с иТТП начинали с ТПО, преднизолона (1 мг/кг) и каплацизумаба (10 мг/сут). ТПО прекращали после увеличения количества тромбоцитов крови >150×109/л. Лишь после этого начинали ритуксимаб (375 мг/м2 еженедельно). Каплацизумаб отменяли при достижении частичной ремиссии (ADAMTS13>20%), а ритуксимаб и глюкокортикоиды – после достижения полной ремиссии (ADAMTS13>40%). Анализировали число тромбоцитов, шистоцитов, концентрации гемоглобина, гаптоглобина, активности лактатдегидрогеназы, ADAMTS13, титр ингибитора ADAMTS13, количество процедур ТПО, объем замененной плазмы, количество тромбоцитов, время до их увеличения >150×109/л, достижения частичной и полной ремиссии. Данные представлены как медиана и межквартильный интервал.
Результаты. С 2021 по 2025 г. диагноз тромботической тромбоцитопенической пурпуры подтвержден у 102 пациентов. В исследование включены 35 пациентов. Количество тромбоцитов >150×109/л достигнуто после 4 (3–5) процедур ТПО через 4 (3–4,5) дня, при этом обменено 11 395 (7241–16 343) мл плазмы. Частичная ремиссия достигнута у 100% пациентов, длительность терапии каплацизумабом составила 23 (12–30) дня. Проведено от 4 до 8 введений ритуксимаба (медиана 4), полная ремиссия достигнута у 33 из 35 пациентов, у 2 получена только частичная ремиссия, им проведена терапия бортезомибом, в связи с неэффективностью последней у 1 пациентки проведена терапия моноклональным анти-CD38 антителом. Вероятность достижения полной ремиссии составила 97,1%.
Заключение. Длительность терапии каплацизумабом, ритуксимабом и глюкокортикоидами у пациентов с иТТП должна определяться достижением целевых значений активности ADAMTS13.
Ключевые слова: тромботическая тромбоцитопеническая пурпура, ADAMTS13, плазмообмен, глюкокортикоиды, каплацизумаб, ритуксимаб, бортезомиб
________________________________________________
Aim. To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity.
Materials and methods. Treatment of patients with iTTP was started with PEX, prednisolone (1 mg/kg) and caplacizumab (10 mg/day). PEX was discontinued after an increase of platelet count >150×109/L. Only after PEX cessation treatment with rituximab (375 mg/m2 weekly) was started. Caplacizumab was discontinued when partial remission (ADAMTS13>20%) was achieved. Rituximab and glucocorticoids were discontinued when complete remission (ADAMTS13>40%) was achieved. Platelet count, schistocyte count, haemoglobin, haptoglobin, lactate dehydrogenase activity, ADAMTS13, ADAMTS13 inhibitor titre, number of PEX, plasma volume replaced, time to increase platelet count >150×109/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range.
Results. From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts >150×109/L were achieved after 4 (3–5) PEX procedures in 4 (3–4.5) days. In total, 11 395 (7241–16 343) ml of plasma were exchanged. Partial remission was achieved in 100% of patients, the duration of caplacizumab therapy was 23 (12–30) days. Rituximab was administered from 4 to 8 times (median 4), complete remission was achieved in 33 out of 35 patients, 2 patients achieved only partial remission, they were treated with bortezomib and 1 with anti-CD38 monoclonal antibody. The probability of complete remission was 97.1%.
Conclusion. The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.
Keywords: thrombotic thrombocytopenic purpura, ADAMTS13, plasma exchange, glucocorticoids, caplacizumab, rituximab, bortezomib
Полный текст
Список литературы
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21. Neupane N, Thapa S, Mahmoud A, et al. Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta-analysis based on revised criteria by the International Working Group for thromboti. eJHaem. 2024;5(1):17-90. DOI:10.1002/jha2.833
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26. Yates SG, Hofmann SL, Ibrahim IF, et al. Tailoring caplacizumab administration using ADAMTS13 activity for immune-mediated thrombotic thrombocytopenic purpura. Blood VTH. 2024;1(3):100010. DOI:10.1016/j.bvth.2024.100010
27. Volker LA, Kaufeld J, Miesbach W, et al. ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP. Blood Adv. 2020;4(13):3093-101. DOI:10.1182/bloodadvances.2020001987
28. Imada K, Miyakawa Y, Ichikawa S, et al. Frontline use of rituximab may prevent ADAMTS13 inhibitor boosting during caplacizumab treatment in patients with iTTP: post hoc analysis of a phase 2/3 study in Japan. Thromb J. 2024;22(1):1-8. DOI:10.1186/s12959-024-00642-3
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3. Zheng XL, Vesely SK, Cataland SR, et al. ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020;18(10):2496-502. DOI:10.1111/jth.15010
4. George JN, Woodson RD, Kiss JE, et al. Rituximab Therapy for Thrombotic Thrombocytopenic Purpura: A Proposed Study of the Transfusion Medicine/Hemostasis Clinical Trials Network With a Systematic Review of Rituximab Therapy for Immune-Mediated Disorders. J Clin Apher. 2006;21:49-56. DOI:10.1002/jca.20091
5. Yamada Y, Ohbe H, Yasunaga H, et al. Clinical Practice Pattern of Acquired Thrombotic Thrombocytopenic Purpura in Japan: A nationwide Inpatient Database Analysis. Blood. 2019;134(Suppl 1):2374. DOI:10.1182/blood-2019-125170
6. Knoebl P, Cataland S, Peyvandi F, et al. Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. J Thromb Haemost. 2020;18(2):479-84. DOI:10.1111/jth.14679
7. Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2016;374(6):511-22. DOI:10.1056/NEJMoa1505533
8. He J, Qi J, Han H, et al. Efficacy and safety of caplacizumab in the treatment of thrombotic thrombocytopenic purpura: a systematic review and meta-analysis. Expert Rev Hematol. 2023;16(5):377-85. DOI:0.1080/17474086.2023.2202850
9. Scully M, Cataland SR, Peyvandi F, et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019;380(4):335-46. DOI:10.1056/nejmoa1806311
10. Coppo P, Bubenheim M, Azoulay E, et al. A regimen with caplacizumab, immunosuppression, and plasma exchange prevents unfavorable outcomes in immune-mediated TTP. Blood. 2021;137(6):733-42. DOI:10.1182/blood.2020008021
11. Dolgov VV, Svirin PV. Laboratornaia diagnostika narushenii gemostasa. Moscow–Tver: Triada, 2005 (in Russian).
12. Mancini I. ADAMTS13-related assays in acquired thrombotic thrombocytopenic purpura. Università degli studi di Milano, 2012.
13. Vendramin C, Thomas M, Westwood JP, et al. Bethesda Assay for Detecting Inhibitory Anti-ADAMTS13 Antibodies in Immune-Mediated Thrombotic Thrombocytopenic Purpura. TH Open. 2018;2(3):e329-33. DOI:10.1055/s-0038-1672187
14. Shelat SG, Smith P, Ai I, et al. Inhibitory autoantibodies against ADAMTS-13 in patients with thrombotic thrombocytopenic purpura bind ADAMTS-13 protease and may accelerate its clearance in vivo. J Thromb Haemost. 2006;4(8):1707-17. DOI:10.1111/j.1538-7836.2006.02025.x
15. Cuker A, Cataland SR, Coppo P, et al. Redefining Outcomes in Immune TTP: an International Working Group Consensus Report. Blood. 2021;137(14):1855-61. DOI:10.1182/blood.2020009150
16. Agosti P, De Leo P, Capecchi M, et al. Caplacizumab use for immune thrombotic thrombocytopenic purpura: the Milan thrombotic thrombocytopenic purpura registry. Res Pr Thromb Haemost. 2023;7(6):1-5. DOI:10.1016/j.rpth.2023.102185
17. Kühne L, Knöbl P, Eller K, et al. Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange. Blood. 2024;144(14):1486-95. DOI:10.1182/blood.2023023780
18. Lee GM. iTTP loses TPE. Blood. 2024;144(14):1462-3. DOI:10.1182/blood.2024025574
19. Sarode R, Bandarenko N, Brecher ME, et al. Thrombotic thrombocytopenic purpura: 2012 American Society for Apheresis (ASFA) consensus conference on classification, diagnosis, management, and future research. J Clin Apher. 2014;29(3):148-67. DOI:10.1002/jca.21302
20. Sargentini-Maier ML, De Decker P, Tersteeg C, et al. Clinical pharmacology of caplacizumab for the treatment of patients with acquired thrombotic thrombocytopenic purpura. Exp Rev Clin Pharmacol. 2019;12(6):537-45. DOI:10.1080/17512433.2019.1607293
21. Neupane N, Thapa S, Mahmoud A, et al. Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta-analysis based on revised criteria by the International Working Group for thromboti. eJHaem. 2024;5(1):17-90. DOI:10.1002/jha2.833
22. Coppo P, Cuker A, George JN. Thrombotic thrombocytopenic purpura: Toward targeted therapy and precision medicine. Res Pr Thromb Haemost. 2018;3(1):26-37. DOI:10.1002/RTH2.12160
23. Mazepa M, Masias C, Chaturvedi S. How targeted therapy disrupts the treatment paradigm for acquired TTP: the risks, benefits, and unknowns. Blood. 2019;134(5):415-20. DOI:10.1182/blood.2019000954
24. Völker LA, Brinkkoetter PT, Cataland SR, et al. Five years of caplacizumab – lessons learned and remaining controversies in immune-mediated thrombotic thrombocytopenic purpura. J Thromb Haemost. 2023;21(10):2718-25. DOI:10.1016/j.jtha.2023.07.027
25. Hughes M, Prescott C, Elliott N, et al. NICE guidance on caplacizumab for treating acute acquired thrombotic thrombocytopenia purpura. Lancet Haematol. 2021;8(1):e14-5. DOI:10.1016/S2352-3026(20)30406-3
26. Yates SG, Hofmann SL, Ibrahim IF, et al. Tailoring caplacizumab administration using ADAMTS13 activity for immune-mediated thrombotic thrombocytopenic purpura. Blood VTH. 2024;1(3):100010. DOI:10.1016/j.bvth.2024.100010
27. Volker LA, Kaufeld J, Miesbach W, et al. ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP. Blood Adv. 2020;4(13):3093-101. DOI:10.1182/bloodadvances.2020001987
28. Imada K, Miyakawa Y, Ichikawa S, et al. Frontline use of rituximab may prevent ADAMTS13 inhibitor boosting during caplacizumab treatment in patients with iTTP: post hoc analysis of a phase 2/3 study in Japan. Thromb J. 2024;22(1):1-8. DOI:10.1186/s12959-024-00642-3
29. Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011;118(7):1746-53. DOI:10.1182/blood-2011-03-341131
30. McDonald V, Manns K, Mackie IJ, et al. Rituximab pharmacokinetics during the management of acute idiopathic thrombotic thrombocytopenic purpura. J Thromb Haemost. 2010;8(6):1201-8. DOI:10.1111/j.1538-7836.2010.03818.x
31. Westwood JP, Thomas M, Alwan F, et al. Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: Outcome and evaluation of dosing regimens. Blood Adv. 2017;1(15):115966. DOI:10.1182/bloodadvances.2017008268
32. Lee NCJ, Yates S, Rambally S, et al. Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single-centre retrospective cohort and systematic literature review. Br J Haematol. 2024;204(2):63843. DOI:10.1111/bjh.19035
33. Katodritou E, Kastritis E, Dalampira D, et al. Improved survival of patients with primary plasma cell leukemia with VRd or daratumumab-based quadruplets: A multicenter study by the Greek myeloma study group. Am J Hematol. 2023;98(5):7308. DOI:10.1002/ajh.26891
2. Kremer Hovinga JA, Vesely SK, Terrell DR, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010;115(8):1500-11. DOI:10.1182/blood-2009-09-243790
3. Zheng XL, Vesely SK, Cataland SR, et al. ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020;18(10):2496-502. DOI:10.1111/jth.15010
4. George JN, Woodson RD, Kiss JE, et al. Rituximab Therapy for Thrombotic Thrombocytopenic Purpura: A Proposed Study of the Transfusion Medicine/Hemostasis Clinical Trials Network With a Systematic Review of Rituximab Therapy for Immune-Mediated Disorders. J Clin Apher. 2006;21:49-56. DOI:10.1002/jca.20091
5. Yamada Y, Ohbe H, Yasunaga H, et al. Clinical Practice Pattern of Acquired Thrombotic Thrombocytopenic Purpura in Japan: A nationwide Inpatient Database Analysis. Blood. 2019;134(Suppl 1):2374. DOI:10.1182/blood-2019-125170
6. Knoebl P, Cataland S, Peyvandi F, et al. Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. J Thromb Haemost. 2020;18(2):479-84. DOI:10.1111/jth.14679
7. Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2016;374(6):511-22. DOI:10.1056/NEJMoa1505533
8. He J, Qi J, Han H, et al. Efficacy and safety of caplacizumab in the treatment of thrombotic thrombocytopenic purpura: a systematic review and meta-analysis. Expert Rev Hematol. 2023;16(5):377-85. DOI:0.1080/17474086.2023.2202850
9. Scully M, Cataland SR, Peyvandi F, et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019;380(4):335-46. DOI:10.1056/nejmoa1806311
10. Coppo P, Bubenheim M, Azoulay E, et al. A regimen with caplacizumab, immunosuppression, and plasma exchange prevents unfavorable outcomes in immune-mediated TTP. Blood. 2021;137(6):733-42. DOI:10.1182/blood.2020008021
11. Долгов В.В, Свирин П.В. Лабораторная диагностика нарушений гемостаза. Москва–Тверь: Триада, 2005 [Dolgov VV, Svirin PV. Laboratornaia diagnostika narushenii gemostasa. Moscow–Tver: Triada, 2005 (in Russian)].
12. Mancini I. ADAMTS13-related assays in acquired thrombotic thrombocytopenic purpura. Università degli studi di Milano, 2012.
13. Vendramin C, Thomas M, Westwood JP, et al. Bethesda Assay for Detecting Inhibitory Anti-ADAMTS13 Antibodies in Immune-Mediated Thrombotic Thrombocytopenic Purpura. TH Open. 2018;2(3):e329-33. DOI:10.1055/s-0038-1672187
14. Shelat SG, Smith P, Ai I, et al. Inhibitory autoantibodies against ADAMTS-13 in patients with thrombotic thrombocytopenic purpura bind ADAMTS-13 protease and may accelerate its clearance in vivo. J Thromb Haemost. 2006;4(8):1707-17. DOI:10.1111/j.1538-7836.2006.02025.x
15. Cuker A, Cataland SR, Coppo P, et al. Redefining Outcomes in Immune TTP: an International Working Group Consensus Report. Blood. 2021;137(14):1855-61. DOI:10.1182/blood.2020009150
16. Agosti P, De Leo P, Capecchi M, et al. Caplacizumab use for immune thrombotic thrombocytopenic purpura: the Milan thrombotic thrombocytopenic purpura registry. Res Pr Thromb Haemost. 2023;7(6):1-5. DOI:10.1016/j.rpth.2023.102185
17. Kühne L, Knöbl P, Eller K, et al. Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange. Blood. 2024;144(14):1486-95. DOI:10.1182/blood.2023023780
18. Lee GM. iTTP loses TPE. Blood. 2024;144(14):1462-3. DOI:10.1182/blood.2024025574
19. Sarode R, Bandarenko N, Brecher ME, et al. Thrombotic thrombocytopenic purpura: 2012 American Society for Apheresis (ASFA) consensus conference on classification, diagnosis, management, and future research. J Clin Apher. 2014;29(3):148-67. DOI:10.1002/jca.21302
20. Sargentini-Maier ML, De Decker P, Tersteeg C, et al. Clinical pharmacology of caplacizumab for the treatment of patients with acquired thrombotic thrombocytopenic purpura. Exp Rev Clin Pharmacol. 2019;12(6):537-45. DOI:10.1080/17512433.2019.1607293
21. Neupane N, Thapa S, Mahmoud A, et al. Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta-analysis based on revised criteria by the International Working Group for thromboti. eJHaem. 2024;5(1):17-90. DOI:10.1002/jha2.833
22. Coppo P, Cuker A, George JN. Thrombotic thrombocytopenic purpura: Toward targeted therapy and precision medicine. Res Pr Thromb Haemost. 2018;3(1):26-37. DOI:10.1002/RTH2.12160
23. Mazepa M, Masias C, Chaturvedi S. How targeted therapy disrupts the treatment paradigm for acquired TTP: the risks, benefits, and unknowns. Blood. 2019;134(5):415-20. DOI:10.1182/blood.2019000954
24. Völker LA, Brinkkoetter PT, Cataland SR, et al. Five years of caplacizumab – lessons learned and remaining controversies in immune-mediated thrombotic thrombocytopenic purpura. J Thromb Haemost. 2023;21(10):2718-25. DOI:10.1016/j.jtha.2023.07.027
25. Hughes M, Prescott C, Elliott N, et al. NICE guidance on caplacizumab for treating acute acquired thrombotic thrombocytopenia purpura. Lancet Haematol. 2021;8(1):e14-5. DOI:10.1016/S2352-3026(20)30406-3
26. Yates SG, Hofmann SL, Ibrahim IF, et al. Tailoring caplacizumab administration using ADAMTS13 activity for immune-mediated thrombotic thrombocytopenic purpura. Blood VTH. 2024;1(3):100010. DOI:10.1016/j.bvth.2024.100010
27. Volker LA, Kaufeld J, Miesbach W, et al. ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP. Blood Adv. 2020;4(13):3093-101. DOI:10.1182/bloodadvances.2020001987
28. Imada K, Miyakawa Y, Ichikawa S, et al. Frontline use of rituximab may prevent ADAMTS13 inhibitor boosting during caplacizumab treatment in patients with iTTP: post hoc analysis of a phase 2/3 study in Japan. Thromb J. 2024;22(1):1-8. DOI:10.1186/s12959-024-00642-3
29. Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011;118(7):1746-53. DOI:10.1182/blood-2011-03-341131
30. McDonald V, Manns K, Mackie IJ, et al. Rituximab pharmacokinetics during the management of acute idiopathic thrombotic thrombocytopenic purpura. J Thromb Haemost. 2010;8(6):1201-8. DOI:10.1111/j.1538-7836.2010.03818.x
31. Westwood JP, Thomas M, Alwan F, et al. Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: Outcome and evaluation of dosing regimens. Blood Adv. 2017;1(15):115966. DOI:10.1182/bloodadvances.2017008268
32. Lee NCJ, Yates S, Rambally S, et al. Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single-centre retrospective cohort and systematic literature review. Br J Haematol. 2024;204(2):63843. DOI:10.1111/bjh.19035
33. Katodritou E, Kastritis E, Dalampira D, et al. Improved survival of patients with primary plasma cell leukemia with VRd or daratumumab-based quadruplets: A multicenter study by the Greek myeloma study group. Am J Hematol. 2023;98(5):7308. DOI:10.1002/ajh.26891
________________________________________________
2. Kremer Hovinga JA, Vesely SK, Terrell DR, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010;115(8):1500-11. DOI:10.1182/blood-2009-09-243790
3. Zheng XL, Vesely SK, Cataland SR, et al. ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020;18(10):2496-502. DOI:10.1111/jth.15010
4. George JN, Woodson RD, Kiss JE, et al. Rituximab Therapy for Thrombotic Thrombocytopenic Purpura: A Proposed Study of the Transfusion Medicine/Hemostasis Clinical Trials Network With a Systematic Review of Rituximab Therapy for Immune-Mediated Disorders. J Clin Apher. 2006;21:49-56. DOI:10.1002/jca.20091
5. Yamada Y, Ohbe H, Yasunaga H, et al. Clinical Practice Pattern of Acquired Thrombotic Thrombocytopenic Purpura in Japan: A nationwide Inpatient Database Analysis. Blood. 2019;134(Suppl 1):2374. DOI:10.1182/blood-2019-125170
6. Knoebl P, Cataland S, Peyvandi F, et al. Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. J Thromb Haemost. 2020;18(2):479-84. DOI:10.1111/jth.14679
7. Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2016;374(6):511-22. DOI:10.1056/NEJMoa1505533
8. He J, Qi J, Han H, et al. Efficacy and safety of caplacizumab in the treatment of thrombotic thrombocytopenic purpura: a systematic review and meta-analysis. Expert Rev Hematol. 2023;16(5):377-85. DOI:0.1080/17474086.2023.2202850
9. Scully M, Cataland SR, Peyvandi F, et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019;380(4):335-46. DOI:10.1056/nejmoa1806311
10. Coppo P, Bubenheim M, Azoulay E, et al. A regimen with caplacizumab, immunosuppression, and plasma exchange prevents unfavorable outcomes in immune-mediated TTP. Blood. 2021;137(6):733-42. DOI:10.1182/blood.2020008021
11. Dolgov VV, Svirin PV. Laboratornaia diagnostika narushenii gemostasa. Moscow–Tver: Triada, 2005 (in Russian).
12. Mancini I. ADAMTS13-related assays in acquired thrombotic thrombocytopenic purpura. Università degli studi di Milano, 2012.
13. Vendramin C, Thomas M, Westwood JP, et al. Bethesda Assay for Detecting Inhibitory Anti-ADAMTS13 Antibodies in Immune-Mediated Thrombotic Thrombocytopenic Purpura. TH Open. 2018;2(3):e329-33. DOI:10.1055/s-0038-1672187
14. Shelat SG, Smith P, Ai I, et al. Inhibitory autoantibodies against ADAMTS-13 in patients with thrombotic thrombocytopenic purpura bind ADAMTS-13 protease and may accelerate its clearance in vivo. J Thromb Haemost. 2006;4(8):1707-17. DOI:10.1111/j.1538-7836.2006.02025.x
15. Cuker A, Cataland SR, Coppo P, et al. Redefining Outcomes in Immune TTP: an International Working Group Consensus Report. Blood. 2021;137(14):1855-61. DOI:10.1182/blood.2020009150
16. Agosti P, De Leo P, Capecchi M, et al. Caplacizumab use for immune thrombotic thrombocytopenic purpura: the Milan thrombotic thrombocytopenic purpura registry. Res Pr Thromb Haemost. 2023;7(6):1-5. DOI:10.1016/j.rpth.2023.102185
17. Kühne L, Knöbl P, Eller K, et al. Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange. Blood. 2024;144(14):1486-95. DOI:10.1182/blood.2023023780
18. Lee GM. iTTP loses TPE. Blood. 2024;144(14):1462-3. DOI:10.1182/blood.2024025574
19. Sarode R, Bandarenko N, Brecher ME, et al. Thrombotic thrombocytopenic purpura: 2012 American Society for Apheresis (ASFA) consensus conference on classification, diagnosis, management, and future research. J Clin Apher. 2014;29(3):148-67. DOI:10.1002/jca.21302
20. Sargentini-Maier ML, De Decker P, Tersteeg C, et al. Clinical pharmacology of caplacizumab for the treatment of patients with acquired thrombotic thrombocytopenic purpura. Exp Rev Clin Pharmacol. 2019;12(6):537-45. DOI:10.1080/17512433.2019.1607293
21. Neupane N, Thapa S, Mahmoud A, et al. Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta-analysis based on revised criteria by the International Working Group for thromboti. eJHaem. 2024;5(1):17-90. DOI:10.1002/jha2.833
22. Coppo P, Cuker A, George JN. Thrombotic thrombocytopenic purpura: Toward targeted therapy and precision medicine. Res Pr Thromb Haemost. 2018;3(1):26-37. DOI:10.1002/RTH2.12160
23. Mazepa M, Masias C, Chaturvedi S. How targeted therapy disrupts the treatment paradigm for acquired TTP: the risks, benefits, and unknowns. Blood. 2019;134(5):415-20. DOI:10.1182/blood.2019000954
24. Völker LA, Brinkkoetter PT, Cataland SR, et al. Five years of caplacizumab – lessons learned and remaining controversies in immune-mediated thrombotic thrombocytopenic purpura. J Thromb Haemost. 2023;21(10):2718-25. DOI:10.1016/j.jtha.2023.07.027
25. Hughes M, Prescott C, Elliott N, et al. NICE guidance on caplacizumab for treating acute acquired thrombotic thrombocytopenia purpura. Lancet Haematol. 2021;8(1):e14-5. DOI:10.1016/S2352-3026(20)30406-3
26. Yates SG, Hofmann SL, Ibrahim IF, et al. Tailoring caplacizumab administration using ADAMTS13 activity for immune-mediated thrombotic thrombocytopenic purpura. Blood VTH. 2024;1(3):100010. DOI:10.1016/j.bvth.2024.100010
27. Volker LA, Kaufeld J, Miesbach W, et al. ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP. Blood Adv. 2020;4(13):3093-101. DOI:10.1182/bloodadvances.2020001987
28. Imada K, Miyakawa Y, Ichikawa S, et al. Frontline use of rituximab may prevent ADAMTS13 inhibitor boosting during caplacizumab treatment in patients with iTTP: post hoc analysis of a phase 2/3 study in Japan. Thromb J. 2024;22(1):1-8. DOI:10.1186/s12959-024-00642-3
29. Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011;118(7):1746-53. DOI:10.1182/blood-2011-03-341131
30. McDonald V, Manns K, Mackie IJ, et al. Rituximab pharmacokinetics during the management of acute idiopathic thrombotic thrombocytopenic purpura. J Thromb Haemost. 2010;8(6):1201-8. DOI:10.1111/j.1538-7836.2010.03818.x
31. Westwood JP, Thomas M, Alwan F, et al. Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: Outcome and evaluation of dosing regimens. Blood Adv. 2017;1(15):115966. DOI:10.1182/bloodadvances.2017008268
32. Lee NCJ, Yates S, Rambally S, et al. Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single-centre retrospective cohort and systematic literature review. Br J Haematol. 2024;204(2):63843. DOI:10.1111/bjh.19035
33. Katodritou E, Kastritis E, Dalampira D, et al. Improved survival of patients with primary plasma cell leukemia with VRd or daratumumab-based quadruplets: A multicenter study by the Greek myeloma study group. Am J Hematol. 2023;98(5):7308. DOI:10.1002/ajh.26891
Авторы
Г.М. Галстян*1, Е.Е. Клебанова1, С.Ю. Мамлеева1, П.В. Авдонин2, З.Т. Фидарова1, М.Ю. Дроков1, Е.Н. Паровичникова1
1ФГБУ «Национальный медицинский исследовательский центр гематологии» Минздрава России, Москва, Россия;
2ФГБУН «Институт биологии развития им. Н.К. Кольцова» РАН, Москва, Россия
*gengalst@gmail.com
1National Medical Research Center for Hematology, Moscow, Russia;
2Koltzov Institute of Developmental Biology, Moscow, Russia
*gengalst@gmail.com
1ФГБУ «Национальный медицинский исследовательский центр гематологии» Минздрава России, Москва, Россия;
2ФГБУН «Институт биологии развития им. Н.К. Кольцова» РАН, Москва, Россия
*gengalst@gmail.com
________________________________________________
1National Medical Research Center for Hematology, Moscow, Russia;
2Koltzov Institute of Developmental Biology, Moscow, Russia
*gengalst@gmail.com
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