Zheng X Long, Al-Housni Zainab, Cataland Spero R, Coppo Paul, Geldziler Brian, Germini Federico, Iorio Alfonso, Keepanasseri Arun, Masias Camila, Matsumoto Masanori, McCrae Keith R, McIntyre Jo, Mustafa Reem A, Peyvandi Flora, Russell Lene, Tarawneh Rawan, Vesely Sara K
Department of Pathology and Laboratory Medicine, The University of Kansas Medical Center, Kansas City, Kansas, USA; Institute of Reproductive Medicine and Developmental Sciences, The University of Kansas Medical Center, Kansas City, Kansas, USA.
Department of Medicine, McMaster University, Hamilton, Ontario, Canada; Department of Hematology, University Medical City, Muscat, Oman.
J Thromb Haemost. 2025 Jun 17. doi: 10.1016/j.jtha.2025.06.002.
Over the past few years, new information has emerged in the management of both immune thrombotic thrombocytopenic purpura (iTTP) and congenital (or hereditary) thrombotic thrombocytopenic purpura (cTTP).
In March 2024, the International Society on Thrombosis and Haemostasis (ISTH) formed a multidisciplinary panel comprising hematologists, intensivists, nephrologists, pathologists, patient representatives, and a methodology team. The panel discussed all treatment questions related to thrombotic thrombocytopenic purpura (TTP) using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) method to appraise evidence and formulate recommendations.
For patients with cTTP in remission, a new strong recommendation was issued for the use of recombinant ADAMTS-13 over fresh frozen plasma in the context of moderate certainty evidence. The panel also revised a previous recommendation and suggested using fresh frozen plasma over a watch-and-wait approach for patients with cTTP in remission based on very low certainty evidence should recombinant. The panel reviewed and referenced new publications supporting therapeutic efficacy, potential survival benefit, and cost considerations of adding caplacizumab to therapeutic plasma exchange, corticosteroids, and rituximab, but concluded that no change was warranted to the previous recommendations in the management of iTTP. Good practice statements on the concomitant use of antithrombotic agents were marginally modified.
For patients with iTTP, no change to 2020's recommendations. For patients with cTTP, the panel supports ADAMTS-13 replacement. Where accessible, recombinant ADAMTS-13 provides the most favorable balance of benefits and risks. Otherwise, fresh frozen plasma may still be effective. Shared decision-making should include the benefits, the potential harms, and the burden of care.
在过去几年中，免疫性血栓性血小板减少性紫癜（iTTP）和先天性（或遗传性）血栓性血小板减少性紫癜（cTTP）的管理方面出现了新信息。
2024年3月，国际血栓与止血学会（ISTH）组建了一个多学科小组，成员包括血液学家、重症监护专家、肾病学家、病理学家、患者代表和一个方法学团队。该小组使用推荐分级评估、制定和评价（GRADE）方法讨论了与血栓性血小板减少性紫癜（TTP）相关的所有治疗问题，以评估证据并制定建议。
对于处于缓解期的cTTP患者，在中等确定性证据的背景下，发布了一项新的强烈建议，即在使用重组ADAMTS-13和新鲜冰冻血浆方面，应选择前者。该小组还修订了先前的一项建议，基于极低确定性证据建议，对于处于缓解期的cTTP患者，若需使用重组药物，应选择新鲜冰冻血浆而非观察等待策略。该小组审查并参考了支持在治疗性血浆置换、皮质类固醇和利妥昔单抗中添加卡泊单抗的治疗效果、潜在生存益处和成本考虑的新出版物，但得出结论，iTTP管理中的先前建议无需改变。关于抗血栓药物联合使用的良好实践声明略有修改。
对于iTTP患者，2020年的建议无需改变。对于cTTP患者，该小组支持ADAMTS-13替代治疗。在可获得的情况下，重组ADAMTS-13提供了最有利的利益和风险平衡。否则，新鲜冰冻血浆可能仍然有效。共同决策应包括益处、潜在危害和护理负担。
