Recovery of renal function after long-term dialysis and resolution of cardiomyopathy in a patient with aHUS receiving eculizumab

Abstract

We present the case of a 18-month-old girl with renal and cardiac manifestations of atypical haemolytic uraemic syndrome (aHUS), and a novel complement factor H mutation. Transient haematological remission was achieved with intensive plasmapheresis, but cardiac function deteriorated and renal function was not restored. Initiation of eculizumab after 6 months of dialysis significantly improved organ function. At 43 months after presentation, haematological values had normalised and cardiac function had improved. Dialysis was discontinued after 10 months (the longest reported time in a patient with aHUS) and the estimated glomerular filtration rate had recovered to 70 mL/min/1.73 m(2). In conclusion, treatment of aHUS with eculizumab, even after long-term dialysis, can significantly improve renal function. Discontinuation of dialysis and resolution of cardiac function has implications on the potential recovery and treatment choice of such patients. Earlier initiation of eculizumab, however, might have prevented the irreversible renal sclerosis and cardiac dysfunction.

Discontinuation of Peritoneal Dialysis after Late Initiation of Eculizumab in a Case of Familial Atypical Hemolytic-Uremic Syndrome: A Case Report

“…Initiation of eculizumab treatment after long-term dialysis is a rare occurrence but it has been previously reported to improve renal function [7, 8]. In the present study, the patient initiated eculizumab treatment after an interval of 62 months (since the drug was not available at diagnosis).…”

“…The median interval between clinical presentation of aHUS and initiation of eculizumab treatment ranged between 0.8 and 8.6 months among patients with progressive thrombotic microangiopathy and renal damage (regardless of decreases in platelet counts). Initiation of eculizumab treatment after long-term dialysis is a rare occurrence but it has been previously reported to improve renal function [ 7 , 8 ]. In the present study, the patient initiated eculizumab treatment after an interval of 62 months (since the drug was not available at diagnosis).…”

“…However, the duration of eculizumab treatment remains a controversial issue [ 6 ]. Moreover, data regarding clinical outcomes among patients starting eculizumab treatment long after the acute clinical manifestations of aHUS are scarce [ 7 , 8 ].…”

Renal Recovery Years After Initial Diagnosis of Hemolytic Uremic Syndrome

“…11 Even in “Atypical” HUS, renal recovery after prolonged renal replacement is rare, occurring at a maximum of 6 and 8 months respectively. 11,12…”

The experience of using the Russian biosimilar of the original drug eculizumab for the treatment of patients with atypical hemolytic-uremic syndrome

“…Первое применение экулизумаба в Россиискои Федерации у пациентки 2 лет датировано июнем 2012 г. [35]. В 2019 г. на территории РФ зарегистрирован биоаналог экулизумабапрепарат Элизария ® (АО «ГЕНЕРИУМ»), прошедшии необходимыи комплекс доклинических и клинических исследовании для подтверждения его биоаналогичности оригинальному препарату [26,27].…”