G M Galstyan, E E Klebanova, S Y Mamleeva, P V Avdonin, Z T Fidarova, M Y Drokov, E N Parovichnikova
{"title":"[免疫性血栓性血小板减少性紫癜患者的个性化治疗]。","authors":"G M Galstyan, E E Klebanova, S Y Mamleeva, P V Avdonin, Z T Fidarova, M Y Drokov, E N Parovichnikova","doi":"10.26442/00403660.2025.08.203326","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Treatment of immune thrombotic thrombocytopenic purpura (iTTP) includes plasma exchange (PEX) and immunosuppression (glucocorticoids and rituximab). The addition of caplacizumab to therapy has improved treatment outcomes in iTTP. However, the available therapies focus on the duration of drug administration and clinical response rather than ADAMTS13 activity.</p><p><strong>Aim: </strong>To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity.</p><p><strong>Materials and methods: </strong>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 > 150×10<sup>9</sup>/L. Only after PEX cessation treatment with rituximab (375 mg/m<sup>2</sup> 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 > 150×10<sup>9</sup>/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range.</p><p><strong>Results: </strong>From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts > 150×10<sup>9</sup>/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%.</p><p><strong>Conclusion: </strong>The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.</p>","PeriodicalId":22209,"journal":{"name":"Terapevticheskii Arkhiv","volume":"97 8","pages":"711-718"},"PeriodicalIF":0.3000,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Personalised treatment of patients with immune thrombotic thrombocytopenic purpura].\",\"authors\":\"G M Galstyan, E E Klebanova, S Y Mamleeva, P V Avdonin, Z T Fidarova, M Y Drokov, E N Parovichnikova\",\"doi\":\"10.26442/00403660.2025.08.203326\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Treatment of immune thrombotic thrombocytopenic purpura (iTTP) includes plasma exchange (PEX) and immunosuppression (glucocorticoids and rituximab). The addition of caplacizumab to therapy has improved treatment outcomes in iTTP. However, the available therapies focus on the duration of drug administration and clinical response rather than ADAMTS13 activity.</p><p><strong>Aim: </strong>To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity.</p><p><strong>Materials and methods: </strong>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 > 150×10<sup>9</sup>/L. Only after PEX cessation treatment with rituximab (375 mg/m<sup>2</sup> 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 > 150×10<sup>9</sup>/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range.</p><p><strong>Results: </strong>From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts > 150×10<sup>9</sup>/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%.</p><p><strong>Conclusion: </strong>The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.</p>\",\"PeriodicalId\":22209,\"journal\":{\"name\":\"Terapevticheskii Arkhiv\",\"volume\":\"97 8\",\"pages\":\"711-718\"},\"PeriodicalIF\":0.3000,\"publicationDate\":\"2025-08-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Terapevticheskii Arkhiv\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.26442/00403660.2025.08.203326\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"MEDICINE, GENERAL & INTERNAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Terapevticheskii Arkhiv","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.26442/00403660.2025.08.203326","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
[Personalised treatment of patients with immune thrombotic thrombocytopenic purpura].
Background: Treatment of immune thrombotic thrombocytopenic purpura (iTTP) includes plasma exchange (PEX) and immunosuppression (glucocorticoids and rituximab). The addition of caplacizumab to therapy has improved treatment outcomes in iTTP. However, the available therapies focus on the duration of drug administration and clinical response rather than ADAMTS13 activity.
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.
期刊介绍：
Терапевтический архив
The journal was founded by the prominent Russian therapists M.P. Konchalovsky and G.F. Lang in 1923. Then its editors-in-chief were Professors V.N. Vinogradov and A.G. Gukasyan. Since 1972, E.I. Chazov, Academician of the Russian Academy of Sciences, has been heading the editorial board of the journal.
Over 90 years, there have been more than 1000 issues where the authors and editorial staff have done their best for readers to keep abreast of current advances in medical science and practice and for physicians to master the advanced principles of recognition and treatment of a wide spectrum of visceral diseases.
The papers published in the journal (editorials, original articles, lectures, reviews, etc.) cover both current scientific achievements and practical experience in diagnosing, treating, and preventing visceral diseases. The authors of publications are not only Russian, but also foreign scientists and physicians. All papers are peer-reviewed by highly qualified Russian specialists.
The journal is published monthly. Traditionally, each issue has predominantly certain thematic areas covering individual therapy specializations. Every year, one of the issues is devoted to related problems in practical medicine (allergology and immunology, neurology and psychiatry, obstetrics, oncology, etc.). This all draws the attention of the reading public to the journal.
The journal is indexed in RSCI (Russian Science Citation Index), PubMed/Medline, Index Medicus, Scopus/EMBASE, Web of Science Core Collection (Science Citation Index Expanded), Web of Science (Russian Science Citation Index - RSCI, Current Contents Connect, BIOSIS Previews), Google Scholar, Ulrich''s Periodicals Directory.
The journal is included in the list of periodicals recommended by the Higher Attestation Committee for publishing the papers containing the basic materials of doctoral and candidate dissertations.
By the decision of the Presidium of the Russian Academy of Medical Sciences, the “Therapevticheskiy Arkhiv” was awarded the Botkin medal. It was admitted to the European Association of Sciences Editors (EASE).
The journal was honored with the Golden Press Fund decoration at the 13th International Press Professional Exhibition.
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