Mazzierli Tommaso, Allegretta Federica, Maffini Enrico, Allinovi Marco
Nephrology, Dialysis and Transplantation Unit, Careggi University Hospital, Florence, Italy.
Department of Biomedical Experimental and Clinical Sciences "Mario Serio", University of Florence, Florence, Italy.
Front Pharmacol. 2023 Jan 9;13:1088031. doi: 10.3389/fphar.2022.1088031. eCollection 2022.
Drug-induced thrombotic microangiopathy (DITMA) represents 10%-13% of all thrombotic microangiopathy (TMA) cases and about 20%-30% of secondary TMAs, just behind pregnancy-related and infection-related forms. Although the list of drugs potentially involved as causative for TMA are rapidly increasing, the scientific literature on DITMA is quite scarce (mostly as individual case reports or little case series), leading to poor knowledge of pathophysiological mechanisms and clinical management. In this review, we focused on these critical aspects regarding DITMA. We provided an updated list of TMA-associated drugs that we selected from a scientific literature review, including only those drugs with a definite or probable causal association with TMA. The list of drugs is heterogeneous and could help physicians from several different areas to be familiar with DITMA. We describe the clinical features of DITMA, presenting the full spectrum of clinical manifestations, from systemic to kidney-limited forms. We also analyze the association between signs/symptoms (i.e., malignant hypertension, thrombocytopenia) and specific DITMA causative drugs (i.e., interferon, ticlopidine). We highlighted their multiple different pathophysiological mechanisms, being frequently classified as immune-mediated (idiosyncratic) and dose-related/toxic. In particular, to clarify the role of the complement system and genetic deregulation of the related genes, we conducted a revision of the scientific literature searching for DITMA cases who underwent renal biopsy and/or genetic analysis for complement genes. We identified a complement deposition in renal biopsies in half of the patients (37/66; 57%), with some drugs associated with major deposits (i.e., gemcitabine and ramucirumab), particularly in capillary vessels (24/27; 88%), and other with absent deposits (tyrosine kinase inhibitors and intraocular anti-VEGF). We also found out that, differently from other secondary TMAs (such as pregnancy-related-TMA and malignant hypertension TMA), complement genetic pathological mutations are rarely involved in DITMA (2/122, 1.6%). These data suggest a variable non-genetic complement hyperactivation in DITMA, which probably depends on the causative drug involved. Finally, based on recent literature data, we proposed a treatment approach for DITMA, highlighting the importance of drug withdrawal and the role of therapeutic plasma-exchange (TPE), rituximab, and anti-complementary therapy.
药物性血栓性微血管病(DITMA)占所有血栓性微血管病(TMA)病例的10%-13%,占继发性TMA的20%-30%,仅次于与妊娠相关和与感染相关的类型。尽管可能导致TMA的药物清单正在迅速增加,但关于DITMA的科学文献相当匮乏(大多为个案报告或少量病例系列),导致对其病理生理机制和临床管理的了解不足。在本综述中,我们聚焦于DITMA的这些关键方面。我们提供了一份从科学文献综述中筛选出的与TMA相关药物的更新清单,仅包括那些与TMA有明确或可能因果关联的药物。该药物清单种类繁多,有助于不同领域的医生熟悉DITMA。我们描述了DITMA的临床特征,呈现了从全身性到肾脏局限性形式的全谱临床表现。我们还分析了体征/症状(即恶性高血压、血小板减少)与特定DITMA致病药物(即干扰素、噻氯匹定)之间的关联。我们强调了它们多种不同的病理生理机制,常被分类为免疫介导(特异质性)和剂量相关/毒性机制。特别是,为了阐明补体系统的作用以及相关基因的遗传失调,我们对科学文献进行了修订,寻找接受肾活检和/或补体基因遗传分析的DITMA病例。我们在一半的患者(37/66;57%)的肾活检中发现了补体沉积,一些药物与大量沉积相关(即吉西他滨和雷莫西尤单抗),特别是在毛细血管中(24/27;88%),而其他药物则无沉积(酪氨酸激酶抑制剂和眼内抗VEGF药物)。我们还发现,与其他继发性TMA(如妊娠相关TMA和恶性高血压TMA)不同,补体基因病理突变在DITMA中很少见(2/122,1.6%)。这些数据表明DITMA中存在可变的非遗传性补体过度激活,这可能取决于所涉及的致病药物。最后基于近期文献数据,我们提出了一种DITMA的治疗方法,强调了停药的重要性以及治疗性血浆置换(TPE)、利妥昔单抗和抗补体治疗的作用。