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Atypical haemolytic uraemic syndrome in the eculizumab era: presentation, response to treatment and evaluation of an eculizumab withdrawal strategy
Author(s) -
Neave Lucy,
Gale Daniel P.,
Cheesman Simon,
Shah Raakhee,
Scully Marie
Publication year - 2019
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.15899
Subject(s) - eculizumab , atypical hemolytic uremic syndrome , medicine , renal function , dialysis , creatinine , gastroenterology , proteinuria , nephrotic syndrome , surgery , complement system , kidney , immunology , antibody
Summary 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 m 2 ). At median duration of follow‐up of 85 weeks (range 4–255), 54·5% had eGFR ≥ 60 ml/min/1·73 m 2 , 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 Eculizumab was withdrawn in 59% (13/22) cases following complete haematological and renal recovery.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.