INTRODUCTION: aHUS is marked by uncontrolled complement activation and TMA. Eculizumab (ecu), approved for aHUS, selectively inhibits terminal complement activation. Ongoing aHUS following renal transplant leads to graft failure within 1 yr in 60% of patients (pts).1 Case reports suggest ecu may prevent ongoing complement-mediated TMA and resulting graft loss in aHUS pts.2
OBJECTIVE: Examine efficacy and safety of ecu in prior transplant (TP) and non-transplant (NTP) pts with long duration of aHUS.
METHODS: An open-label, single-arm, 26-wk trial with long-term extension evaluated ecu efficacy and safety in aHUS pts age ≥12 yrs with long disease duration and prolonged PE/PI.
RESULTS: Table 1 lists baseline characteristics. Median treatment duration was 114 wks. eGFR improved in both groupsmean±SD increase was 7.3±7.5 and 3.9±23.8 mL/min/1.73m2 for NTP and TP, respectively. Significantly greater eGFR improvement occurred with earlier ecu treatment in all pts (P=0.03). No pt required initiation of dialysis over 2 yrs.
CONCLUSIONS: Ecu improved renal function in NTP and TP. Grafts were preserved in all 5 aHUS pts with prior graft loss. Impact of duration of clinical aHUS complication, immunosuppressive therapy, prior eGFR, and dialysis duration require further study. Ecu is effective in preserving renal function, and preventing graft loss in TP due to aHUS. These data support early ecu treatment to prevent ESRD and prophylactic treatment in pts undergoing transplant.
Bedrosian, C.: Employee, Soliris (Alexion Pharmaceuticals).
To cite this abstract in AMA style:Legendre C, Licht C, Hourmant M, Delmas Y, Herthelius M, Trivelli A, Goodship T, Haller H, Moulin B, Bedrosian C, Loirat C. Eculizumab in aHUS Pts with Long Disease Duration, with or without Renal Transplant [abstract]. Am J Transplant. 2013; 13 (suppl 5). https://atcmeetingabstracts.com/abstract/eculizumab-in-ahus-pts-with-long-disease-duration-with-or-without-renal-transplant/. Accessed October 27, 2020.
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