[Personalised treatment of patients with immune thrombotic thrombocytopenic purpura].

IF 0.3 4区医学 Q3 MEDICINE, GENERAL & INTERNAL

Terapevticheskii Arkhiv Pub Date : 2025-08-28 DOI:10.26442/00403660.2025.08.203326

G M Galstyan, E E Klebanova, S Y Mamleeva, P V Avdonin, Z T Fidarova, M Y Drokov, E N Parovichnikova

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引用次数: 0

Abstract

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.

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[免疫性血栓性血小板减少性紫癜患者的个性化治疗]。

背景：免疫性血栓性血小板减少性紫癜（iTTP）的治疗包括血浆置换（PEX）和免疫抑制（糖皮质激素和利妥昔单抗）。在治疗中加入卡普拉珠单抗改善了iTTP的治疗结果。然而，现有的治疗方法侧重于给药时间和临床反应，而不是ADAMTS13活性。目的：评价针对ADAMTS13活性的iTTP治疗的疗效。材料和方法：iTTP患者开始使用PEX、强的松龙（1mg /kg）和卡普拉珠单抗（10mg /天）治疗。血小板计数升高> 150×109/L后停用PEX。只有在PEX停止后，才开始用利妥昔单抗（每周375 mg/m2）治疗。当达到部分缓解（ADAMTS13 bb0 20%）时停用卡普拉珠单抗。当达到完全缓解（ADAMTS13 bb0 40%）时，停用利妥昔单抗和糖皮质激素。分析血小板计数、血吸虫细胞计数、血红蛋白、接触珠蛋白、乳酸脱氢酶活性、ADAMTS13、ADAMTS13抑制剂滴度、PEX次数、血浆置换量、血小板计数增加时间> 150×109/L、部分缓解和完全缓解情况。数据以中位数和四分位数范围表示。结果：从2021年到2025年，102例患者被确诊为TTP。研究纳入了35名患者。在4（3-4.5）天内进行4（3-5）次PEX手术后，血小板计数达到> 150×109/L。总共交换了11 395 (7241-16 343)ml血浆。100%的患者部分缓解，卡普拉珠单抗治疗持续时间为23（12-30）天。利妥昔单抗给药4 ~ 8次（中位4次），35例患者中有33例完全缓解，2例仅部分缓解，他们接受硼替佐米治疗，1例接受抗cd38单克隆抗体治疗。完全缓解的概率为97.1%。结论：iTTP患者卡普拉珠单抗、利妥昔单抗和糖皮质激素治疗的持续时间应由目标ADAMTS13活性的实现来确定。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Terapevticheskii Arkhiv 医学-医学：内科

CiteScore

1.40

自引率

33.30%

发文量

171

审稿时长

3-8 weeks

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.