INTRODUCTION: Ongoing aHUS post renal transplant leads to graft failure within 1 yr in 60% of patients (pts).1 Case reports suggest eculizumab (ecu) prevents ongoing complement-mediated TMA and resulting graft loss in pts with transplant and aHUS.2
OBJECTIVE: Analyze outcomes in non-transplant (NTP) and prior transplant (TP) aHUS pts with progressing TMA and 2 yrs of ongoing ecu treatment.
METHODS: Ecu efficacy and safety were evaluated in an open-label, single-arm, 26-wk, phase 2 trial with long-term extension in pts age ≥12 yrs with progressing TMA.
RESULTS: Median duration of ecu treatment: 100 wks. Mean±SD change in platelet count: 131.9±70.1 for NTP and 72.4±48.3 x109/L for TP. Mean±SD eGFR increase: 48.3±38.4 (179% change) for NTP and 14.8±18.7 mL/min/1.73 m2 (87% change) for TP. Neither transplant-to-screening time nor baseline eGFR had predictive value for eGFR change. Early ecu treatment led to greater eGFR improvement in both groups (P=0.008).
CONCLUSIONS: Ecu improved platelet counts and renal function. Earlier treatment improved efficacy. More study is needed to assess the impact of prior eGFR, duration of clinical aHUS complication, immunosuppressive therapy, and dialysis duration. Ecu is an effective therapy in aHUS pts to improve renal function and to prevent 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, Greenbaum L, Sheerin N, Cohen D, Gaber A, Eitner F, Delmas Y, Furman R, Feldkamp T, Fouque D, Bedrosian C, Loirat C. Eculizumab Efficacy in aHUS Pts with Progressing TMA, with or without Prior Renal Transplant [abstract]. Am J Transplant. 2013; 13 (suppl 5). https://atcmeetingabstracts.com/abstract/eculizumab-efficacy-in-ahus-pts-with-progressing-tma-with-or-without-prior-renal-transplant/. Accessed October 20, 2020.
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