TY  - JOUR
IS  - 5
N1  - Copyright © 2017, International Society of Nephrology. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
SP  - 1261
VL  - 92
JF  - Kidney International
A1  - Brocklebank, V
A1  - Johnson, S
A1  - Sheerin, TP
A1  - Marks, SD
A1  - Gilbert, RD
A1  - Tyerman, K
A1  - Kinoshita, M
A1  - Awan, A
A1  - Kaur, A
A1  - Webb, N
A1  - Hegde, S
A1  - Finlay, E
A1  - Fitzpatrick, M
A1  - Walsh, PR
A1  - Wong, EKS
A1  - Booth, C
A1  - Kerecuk, L
A1  - Salama, AD
A1  - Almond, M
A1  - Inward, C
A1  - Goodship, TH
A1  - Sheerin, NS
A1  - Marchbank, KJ
A1  - Kavanagh, D
UR  - http://doi.org/10.1016/j.kint.2017.04.028
SN  - 1523-1755
TI  - Factor H autoantibody is associated with atypical hemolytic uremic syndrome in children in the United Kingdom and Ireland
AV  - public
Y1  - 2017/11/01/
EP  - 1271
KW  - acute kidney injury
KW  -  complement
KW  -  hemolytic uremic syndrome
N2  - Factor H autoantibodies can impair complement
regulation, resulting in atypical hemolytic uremic
syndrome, predominantly in childhood. There are no trials
investigating treatment, and clinical practice is only
informed by retrospective cohort analysis. Here we
examined 175 children presenting with atypical hemolytic
uremic syndrome in the United Kingdom and Ireland for
factor H autoantibodies that included 17 children with
titers above the international standard. Of the 17, seven
had a concomitant rare genetic variant in a gene encoding
a complement pathway component or regulator. Two
children received supportive treatment; both developed
established renal failure. Plasma exchange was associated
with a poor rate of renal recovery in seven of 11 treated. Six
patients treated with eculizumab recovered renal function.
Contrary to global practice, immunosuppressive therapy to
prevent relapse in plasma exchange?treated patients was
not adopted due to concerns over treatment-associated
complications. Without immunosuppression, the relapse
rate was high (five of seven). However, reintroduction of
treatment resulted in recovery of renal function. All
patients treated with eculizumab achieved sustained
remission. Five patients received renal transplants without
specific factor H autoantibody?targeted treatment with
recurrence in one who also had a functionally significant CFI mutation. Thus, our current practice is to initiate
eculizumab therapy for treatment of factor H
autoantibody?mediated atypical hemolytic uremic
syndrome rather than plasma exchange with or without
immunosuppression. Based on this retrospective analysis
we see no suggestion of inferior treatment, albeit the
strength of our conclusions is limited by the small sample size
ID  - discovery1572803
PB  - ELSEVIER SCIENCE INC
ER  -