Zheng X Long, Vesely Sara K, Cataland Spero R, Coppo Paul, Geldziler Brian, Iorio Alfonso, Matsumoto Masanori, Mustafa Reem A, Pai Menaka, Rock Gail, Russell Lene, Tarawneh Rawan, Valdes Julie, Peyvandi Flora
Department of Pathology and Laboratory Medicine, University of Kansas Medical Center, Kansas City, KS, USA.
Hudson College of Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA.
J Thromb Haemost. 2020 Oct;18(10):2503-2512. doi: 10.1111/jth.15009. Epub 2020 Sep 11.
Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its management.
In June 2018, the ISTH formed a multidisciplinary guideline panel to issue recommendations about treatment of TTP. The panel discussed 12 treatment questions related to both immune-mediated TTP (iTTP) and hereditary/congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations.
The panel agreed on eleven recommendations based on evidence ranging from very low to moderate certainty. For first episode and relapses of acute iTTP, the panel made a strong recommendation for the addition of corticosteroids to therapeutic plasma exchange (TPE), and a conditional recommendation for addition of rituximab and caplacizumab. For asymptomatic iTTP with low ADAMTS13, the panel made a conditional recommendation for rituximab outside of pregnancy, and for prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, but a conditional recommendation for plasma infusion or a wait and watch approach outside of pregnancy.
The panel's recommendations are based on all the available evidence for the treatment effects of various approaches including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing ADAMTS13 antibody production. There was insufficient evidence for further comparison of different treatment approaches, for which future high-quality studies in iTTP (e.g., rituximab, corticosteroids, recombinant ADAMTS13, and caplacizumab) and in cTTP (eg, recombinant ADAMTS13) are needed.
尽管血栓性血小板减少性紫癜（TTP）的治疗选择有所进展，但仍缺乏高质量数据为临床医生提供有关其管理的信息。
2018年6月，国际血栓与止血学会（ISTH）成立了一个多学科指南小组，以发布关于TTP治疗的建议。该小组讨论了12个与免疫介导的TTP（iTTP）和遗传性/先天性TTP（cTTP）相关的治疗问题。该小组采用推荐分级评估、制定和评价（GRADE）方法，包括证据到决策框架，来评估证据并制定建议。
该小组基于从极低到中等确定性的证据达成了11项建议。对于急性iTTP的首次发作和复发，该小组强烈建议在治疗性血浆置换（TPE）中添加皮质类固醇，并建议有条件地添加利妥昔单抗和卡泊西单抗。对于ADAMTS13水平低的无症状iTTP患者，该小组建议有条件地在非孕期使用利妥昔单抗，孕期进行预防性TPE。对于无症状cTTP，该小组强烈建议孕期进行预防性血浆输注，但对于非孕期血浆输注或观察等待方法建议有条件使用。
该小组的建议基于各种治疗方法治疗效果的所有现有证据，包括抑制炎症、阻止血小板聚集、替代缺失和/或受抑制的ADAMTS13以及抑制ADAMTS13抗体产生。没有足够的证据对不同治疗方法进行进一步比较，为此需要未来在iTTP（例如利妥昔单抗、皮质类固醇、重组ADAMTS13和卡泊西单抗）和cTTP（例如重组ADAMTS13）方面开展高质量研究。
