Galstyan G M, Klebanova E E, Mamleeva S Y, Avdonin P V, Fidarova Z T, Drokov M Y, Parovichnikova E N
National Medical Research Center for Hematology.
Koltsov Institute of Developmental Biology.
Ter Arkh. 2025 Aug 28;97(8):711-718. doi: 10.26442/00403660.2025.08.203326.
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.
To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity.
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/L. Only after PEX cessation treatment with rituximab (375 mg/m 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/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range.
From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts > 150×10/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%.
The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.
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）。在治疗中添加卡泊单抗改善了iTTP的治疗效果。然而，现有的治疗方法侧重于药物给药持续时间和临床反应，而非ADAMTS13活性。
评估以ADAMTS13活性为靶点治疗iTTP的疗效。
iTTP患者的治疗起始采用PEX、泼尼松龙（1mg/kg）和卡泊单抗（10mg/天）。血小板计数增加>150×10⁹/L后停止PEX。仅在停止PEX后开始用利妥昔单抗（375mg/m²每周）治疗。达到部分缓解（ADAMTS13>20%）时停用卡泊单抗。达到完全缓解（ADAMTS13>40%）时停用利妥昔单抗和糖皮质激素。分析血小板计数、裂体细胞计数、血红蛋白、触珠蛋白、乳酸脱氢酶活性、ADAMTS13、ADAMTS13抑制剂滴度、PEX次数、置换血浆量、血小板计数增加>150×10⁹/L的时间、部分缓解和完全缓解的达成情况。数据以中位数和四分位间距表示。
2021年至2025年，102例患者确诊为TTP。35例患者纳入研究。4（3 - 5）次PEX治疗后4（3 - 4.5）天血小板计数>150×10⁹/L。共置换血浆11395（7241 - 16343）ml。100%的患者实现部分缓解，卡泊单抗治疗持续时间为23（12 - 30）天。利妥昔单抗给药4至8次（中位数4次），35例患者中33例实现完全缓解，2例仅实现部分缓解，分别用硼替佐米和抗CD38单克隆抗体治疗。完全缓解概率为97.1%。
iTTP患者使用卡泊单抗、利妥昔单抗和糖皮质激素的治疗持续时间应由目标ADAMTS13活性的达成情况决定。
