TY  - JOUR
IS  - 1
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher?s terms and conditions.
SP  - 113
VL  - 186
A1  - Neave, L
A1  - Gale, DP
A1  - Cheesman, S
A1  - Shah, R
A1  - Scully, M
JF  - British Journal of Haematology
UR  - https://doi.org/10.1111/bjh.15899
SN  - 0007-1048
AV  - public
Y1  - 2019/07//
EP  - 124
TI  - Atypical haemolytic uraemic syndrome in the eculizumab era: presentation, response to treatment and evaluation of an eculizumab withdrawal strategy
KW  - aHUS
KW  -  TMA
KW  -  atypical haemolytic uraemic syndrome
KW  -  eculizumab
KW  -  withdrawal
N2  - The complement inhibitor, eculizumab, has revolutionised the management of atypical haemolytic uraemic syndrome (aHUS), although the optimum treatment duration is debated. Twenty-two cases of acute aHUS managed with eculizumab were retrospectively reviewed, including outcomes after eculizumab withdrawal. Although 41% had an associated complement genetic abnormality, mutation status did not affect severity of clinical presentation. Sixty-four percent required renal replacement acutely, with a high incidence of nephrotic range proteinuria (47%). Eculizumab followed a median of 6 days of plasma exchange. After a median duration of therapy of 11 weeks (range 1-227), haematological recovery was seen in 100%, while 81% achieved at least partial renal recovery (median increase in estimated glomerular filtration rate (eGFR) 49 ml/min/1·73 m2 ). At median duration of follow-up of 85 weeks (range 4-255), 54·5% had eGFR ? 60 ml/min/1·73 m2 , 27% had CKD, 14% were on dialysis, and 4·5% had died. Eculizumab was withdrawn in 59% (13/22) cases following complete haematological and renal recovery. Three of these 13 patients (23%) subsequently relapsed, with defined triggers in 2/3, but all made a full recovery with rapid resumption of eculizumab. There was a significant association between higher presenting creatinine and poorer renal outcomes. A strategy of eculizumab withdrawal in selected cases is both safe and cost effective.
ID  - discovery10073189
ER  -