de Alvaro Moreno, Fernando

Dutt, Tina

Fehrman, Ingela

Hadaya, Karine

Heyne, Nils

Macia, Manuel

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CHRONIC KIDNEY DISEASE.PATHOPHYSIOLOGY, PROGRESSION &RISK FACTORS - 2SP283 RISKOF THROMBOTICMICROANGIOPATHY IN PATIENTSWITH ATYPICAL HAEMOLYTIC URAEMIC SYNDROMEDISCONTINUING FROMCHRONIC ECULIZUMAB THERAPYNils Heyne1, Fernando de Alvaro Moreno2, Tina Dutt3, Ingela Fehrman4,Karine Hadaya5 and Manuel Macia61University of Tübingen, University of Tübingen, Tübingen, Germany, 2UniversityHospital Infanta Sofia, University Hospital Infanta Sofia, Madrid, Spain, 3Alder HeyChildren's NHS Foundation Trust, Alder Hey Children's NHS Foundation Trust,Liverpool, United Kingdom, 4Karolinska University Hospital, Karolinska UniversityHospital, Stockholm, Sweden, 5Geneva University Hospitals, Geneva UniversityHospitals, Geneva, Switzerland, 6University Hospital NS de Candelaria, UniversityHospital NS de Candelaria, Santa Cruz de Tenerife, SpainIntroduction and Aims: Atypical haemolytic uraemic syndrome (aHUS) is a rare,genetic life-threatening disorder characterised by thrombotic microangiopathy(TMA) and end organ damage. Clinical manifestations of TMA are seenthroughout disease progress and, historically, outcomes have been poor. Prospectiveclinical trials (100 patients [pts]) have shown eculizumab (Ecu), a monoclonalantibody blocking terminal complement, to inhibit complement-mediated TMAand be well tolerated in pts with aHUS. Life-long treatment with Ecu is indicatedfor pts with aHUS due to risk of TMA, but discontinuation of treatment has beenattempted.Methods: A retrospective analysis of a series of selected cases of 6 pts withaHUS where Ecu therapy was discontinued or altered from the indicated dosingregimen.Results: Pt details including genetic mutation, duration of Ecu treatment andoutcomes post Ecu discontinuation are described in the table. During Ecutreatment, all pts demonstrated improvement or normalisation of haematologicalparameters and no new TMA manifestations. Four pts had improved renal function,while 2 remained on dialysis. The median time on indicated Ecu dose was 5.2 (range1–14) months and the median time to new TMA post-discontinuation was 3 (range2–12) months. Two pts have not had any new TMA manifestations followingalteration or discontinuation of the approved dosing regimen. Pt 4 has continued toreceive Ecu at a reduced dose (900 mg/month) for 12 months and pt 6 discontinuedEcu and has been followed-up for 19 months.Conclusions: Discontinuation of chronic Ecu treatment in this case series of ptswith aHUS led to new TMA manifestations in 4/6 pts (2 without identifiedmutations, 1 with an MCP mutation and 1 with a C3 mutation) requiringre-initiation of Ecu. These findings highlight the importance of sustainedcomplement blockade in pts with aHUS.© The Author 2015. Published by Oxford University Press on behalf of ERA-EDTA. All rights reserved.Nephrology Dialysis Transplantation 30 (Supplement 3): iii472–iii489, 2015doi:10.1093/ndt/gfv191.3

SP283RISK OF THROMBOTIC MICROANGIOPATHY IN PATIENTS WITH ATYPICAL HAEMOLYTIC URAEMIC SYNDROME DISCONTINUING FROM CHRONIC ECULIZUMAB THERAPY

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SP283RISK OF THROMBOTIC MICROANGIOPATHY IN PATIENTS WITH ATYPICAL HAEMOLYTIC URAEMIC SYNDROME DISCONTINUING FROM CHRONIC ECULIZUMAB THERAPY

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