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Abstract：
Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its appropriate treatment.
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 immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations.
The panel agreed on 11 recommendations based on evidence ranging from very low to moderate certainty. For first acute episode and relapses of iTTP, the panel made a strong recommendation for adding corticosteroids to therapeutic plasma exchange (TPE) and a conditional recommendation for adding rituximab and caplacizumab. For asymptomatic iTTP with low plasma ADAMTS13 activity, the panel made a conditional recommendation for the use of rituximab outside of pregnancy, but prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, and 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 effects of an individual component of various treatment approaches, including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing the formation of ADAMTS13 autoantibody. There was insufficient evidence for further comparing different treatment approaches (eg, TPE, corticosteroids, rituximab, and caplacizumab, etc.), for which high quality studies are needed.
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 immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations.
The panel agreed on 11 recommendations based on evidence ranging from very low to moderate certainty. For first acute episode and relapses of iTTP, the panel made a strong recommendation for adding corticosteroids to therapeutic plasma exchange (TPE) and a conditional recommendation for adding rituximab and caplacizumab. For asymptomatic iTTP with low plasma ADAMTS13 activity, the panel made a conditional recommendation for the use of rituximab outside of pregnancy, but prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, and 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 effects of an individual component of various treatment approaches, including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing the formation of ADAMTS13 autoantibody. There was insufficient evidence for further comparing different treatment approaches (eg, TPE, corticosteroids, rituximab, and caplacizumab, etc.), for which high quality studies are needed.
摘要：
尽管血栓性血小板减少性紫癜（TTP）的治疗方案取得了进展，仍有有限的高质量数据告知临床医生关于其适当的治疗.
2018年6月，ISTH成立了一个多学科指南小组，发布有关TTP治疗的建议。小组讨论了与免疫介导的TTP（iTTP）和遗传性或先天性TTP（cTTP）相关的12个治疗问题。小组使用了建议评估的分级，发展,和评估方法，包括决策证据框架，评估证据并提出建议。
专家组根据从极低到中等确定性的证据，就11项建议达成了一致。对于iTTP的首次急性发作和复发，专家组强烈建议在治疗性血浆置换(TPE)中加入皮质类固醇,并有条件地推荐加入利妥昔单抗和卡普拉斯单抗.对于血浆ADAMTS13活性低的无症状iTTP，专家组对妊娠以外使用利妥昔单抗提出了有条件的建议，但在怀孕期间预防性TPE。对于无症状的cTTP，专家组强烈建议在怀孕期间预防性输注血浆，和有条件的建议血浆输注或等待和观察怀孕以外的方法。
小组的建议是基于各种治疗方法的单个组成部分的影响的所有可用证据，包括抑制炎症,阻断血小板凝集,替换缺失和/或抑制ADAMTS13，并抑制ADAMTS13自身抗体的形成。没有足够的证据进一步比较不同的治疗方法（例如，TPE,皮质类固醇,利妥昔单抗,和卡普拉珠单抗,等。),需要高质量的研究。
2018年6月，ISTH成立了一个多学科指南小组，发布有关TTP治疗的建议。小组讨论了与免疫介导的TTP（iTTP）和遗传性或先天性TTP（cTTP）相关的12个治疗问题。小组使用了建议评估的分级，发展,和评估方法，包括决策证据框架，评估证据并提出建议。
专家组根据从极低到中等确定性的证据，就11项建议达成了一致。对于iTTP的首次急性发作和复发，专家组强烈建议在治疗性血浆置换(TPE)中加入皮质类固醇,并有条件地推荐加入利妥昔单抗和卡普拉斯单抗.对于血浆ADAMTS13活性低的无症状iTTP，专家组对妊娠以外使用利妥昔单抗提出了有条件的建议，但在怀孕期间预防性TPE。对于无症状的cTTP，专家组强烈建议在怀孕期间预防性输注血浆，和有条件的建议血浆输注或等待和观察怀孕以外的方法。
小组的建议是基于各种治疗方法的单个组成部分的影响的所有可用证据，包括抑制炎症,阻断血小板凝集,替换缺失和/或抑制ADAMTS13，并抑制ADAMTS13自身抗体的形成。没有足够的证据进一步比较不同的治疗方法（例如，TPE,皮质类固醇,利妥昔单抗,和卡普拉珠单抗,等。),需要高质量的研究。
