Portoles José, Huerta Ana, Arjona Emilia, Gavela Eva, Agüera Marisa, Jiménez Carlos, Cavero Teresa, Marrero Domingo, Rodríguez de Córdoba Santiago, Diekmann Fritz
Nephrology Department, University Hospital Puerta de Hierro, Madrid, Spain.
RedInRen 16/009, RTYC ISCIII, Madrid, Spain.
Clin Kidney J. 2020 Aug 13;14(4):1173-1180. doi: 10.1093/ckj/sfaa096. eCollection 2021 Apr.
Kidney transplantation (KTx) is a strong trigger for the development of either recurrent or atypical haemolytic uraemic syndrome (aHUS). According to previous studies, eculizumab (ECU) is effective for prophylaxis and for treatment of recurrence.
We evaluated the experiences of Spanish patients with recurrent and aHUS associated with KTx, treated or not treated with ECU. In the group, we classified patients as having early (during the first month) or late aHUS (subsequent onset).
We analysed 36 cases of aHUS associated with KTx. All of the 14 patients with pre-KTx diagnosis of aHUS were considered to have high or moderate risk of recurrence. Despite receiving grafts from suboptimal donors, prophylactic ECU was effective for avoiding recurrence. The drug was stopped only in two cases with low-moderate risk of recurrence and was maintained in high-risk patients with no single relapse. There were 22 aHUS cases and 16 belonged to the early group. The median time of onset in the late group was 3.4 years. The early group had a better response to ECU than the late group, probably due to earlier diagnosis and use of the drug. No genetic pathogenic variant was detected in aHUS cases, suggesting a secondary profile of the disease. ECU was stopped in all patients with no relapses. ECU was well tolerated in all cases.
Both groups (pre-aHUS and ) presented different clinical profiles, management approaches and outcomes. One should consider aHUS regardless of time after KTx. Genetic studies are crucial to stratify risks of relapse and to determine necessary lengths of treatment. We suggest short ECU treatment for cases without pathogenic mutation and that ECU treatment be considered pre-emptively for patients with moderate or high risk of recurrence.
肾移植（KTx）是复发性或非典型溶血尿毒综合征（aHUS）发生的强烈诱因。根据以往研究，依库珠单抗（ECU）对预防和治疗复发有效。
我们评估了西班牙患有与KTx相关的复发性和aHUS的患者接受或未接受ECU治疗的经验。在该组中，我们将患者分为早期（第一个月内）或晚期aHUS（随后发病）。
我们分析了36例与KTx相关的aHUS病例。所有14例肾移植前诊断为aHUS的患者均被认为有高或中度复发风险。尽管接受了次优供体的移植物，但预防性使用ECU可有效避免复发。仅在2例复发风险低-中度的病例中停用了该药物，而在无单次复发的高风险患者中维持使用。有22例aHUS病例，其中16例属于早期组。晚期组的中位发病时间为3.4年。早期组对ECU的反应比晚期组更好，可能是由于诊断和使用药物更早。在aHUS病例中未检测到基因致病变异，提示该疾病为继发性。所有无复发的患者均停用了ECU。所有病例中ECU耐受性良好。
两组（移植前aHUS和移植后aHUS）呈现出不同的临床特征、管理方法和结局。无论肾移植后时间如何，都应考虑aHUS。基因研究对于分层复发风险和确定必要的治疗时长至关重要。我们建议对无致病突变的病例进行短期ECU治疗，并对复发风险为中度或高度的患者预先考虑使用ECU治疗。
