ESPN 50th Annual Meeting

ESPN 2017


 
Eculizumab treatment on renal function in paediatric aHUS patients with kidney transplant: Data from the aHUS Global Registry
CHONI RINAT 1 OLIVIA BOYER 2 LARRY GREENBAUM 3 GURINDER KUMAR 4 MARTIN KONRAD 5 LARS PAPE 6 LESLEY REES 7 GIANLUIGI ARDISSINO 8

1- PEDIATRIC NEPHROLOGY, SHAARE ZEDEK MEDICAL CENTER, JERUSALEM, ISRAEL
2- HÔPITAL NECKER-ENFANTS MALADES, INSTITUT IMAGINE, UNIVERSITÉ PARIS-DESCARTES, PARIS, FRANCE
3- EMORY UNIVERSITY, ATLANTA, GA, USA
4- MEDICAL INSTITUTE, SHEIKH KHALIFA MEDICAL CITY, ABU DHABI, UAE
5- UNIVERSITAETSKLINIKUM MUENSTER, GERMANY
6- MEDIZINISCHE HOCHSCHULE, HANNOVER, GERMANY
7- GREAT ORMOND STREET HOSPITAL, LONDON, UK
8- FONDAZZIONE IRCCS CA’ GRANDA OSPEDALE MAGGIORE POLICLINICO, MILAN, ITALY
 
Introduction:

To analyse the need for dialysis in paediatric patients with atypical haemolytic uraemic syndrome (aHUS) and kidney transplant (KTx) who received eculizumab either pre- or post-transplantation.

Material and methods:

Data from the Global aHUS Registry (NCT01522183) on eculizumab-treated paediatric patients (<18 years at most recent KTx) with at least 1 KTx and ≥1 year of observation post-KTx were analysed for the incidence of dialysis events post-transplant. Patients were grouped by timing of eculizumab treatment: ongoing treatment (≥1 dose) up to time of KTx (pre-KTx; n=18) vs treatment initiation post-KTx (n=19).

Results:

As of August 2016, 1286 patients with aHUS were enrolled in the registry; data from 37 paediatric KTx patients are reported here. Patients had a median age at diagnosis of 5.2 years, 78% were male, 30% had a family history of aHUS and 16% had ≥2 KTx. Demographics and clinical characteristics were similar between treatment groups (Table). aHUS diagnosis was made prior to KTx in 31/37 patients and after KTx in six patients (all in the eculizumab treatment post-KTx group). One of 18 patients treated with eculizumab pre-KTx required short-term (lasting <3 months) dialysis (incidence rate: 1.3/100 patient-years). Six of 19 patients receiving eculizumab post-KTx required 16 periods of dialysis following KTx (incidence rate: 4.4/100 patient-years). Two of these six patients received dialysis for more than 3 months.

Conclusions:

We provide the first real-world data on the association of the timing of eculizumab treatment on the need for post-KTx dialysis in paediatric patients with aHUS. This descriptive, retrospective analysis suggests that eculizumab pre-KTx may decrease the incidence of dialysis post-KTx when compared to eculizumab post-KTx in paediatric patients with aHUS. Further studies are required to confirm whether initiating eculizumab prior to transplant reduces dialysis events in children with aHUS following KTx.

Acknowledgments: We wish to thank the patients and registry investigators