The modern role of corticosteroids in immune thrombotic thrombocytopenic purpura

皮质类固醇在免疫性血栓性血小板减少性紫癜中的现代作用

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Abstract

Deficiency of ADAMTS13 caused by autoantibodies leads to the thrombotic microangiopathy immune thrombotic thrombocytopenic purpura (iTTP). Corticosteroids have long been utilized in the management of patients with iTTP, both in the acute setting and continued after clinical remission has been achieved, usually tapered over a period of weeks. However, with recent advances in the field, the landscape of steroid utilization is changing, particularly since the mid-2010s with the advent of caplacizumab and anti-cluster of differentiation 20 (CD20) directed therapy in iTTP. Though steroids are generally well tolerated, many patients are at risk of adverse events related to corticosteroid use, such as patients with diabetes mellitus, hypertension, and cardiovascular disease. There are no uniform guidelines regarding optimal steroid formulations, dosages, or duration of treatment. Modern treatment modalities target more specific aspects of the pathophysiology of iTTP, leading to speculation that steroid-free regimens may become the new standard of care. Despite this, because of the favorable adverse event profile of steroids and the modest benefit they provide, steroid use is still widespread for patients with iTTP. As such, there is a need for further studies regarding corticosteroid dosing; duration of treatment with corticosteroids; adverse events associated specifically with the use of corticosteroids; and whether, how, or in what context corticosteroids can safely be omitted or replaced with another treatment modality in the care of patients with iTTP. In this Mini-Review, we will explore the historical reasoning and data supporting the use of corticosteroids in iTTP and the shifting role of steroids in the modern era.

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