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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Case+Rep+Nephrol+Dial
2017 ; 7
(1
): 18-25
Nephropedia Template TP
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Discontinuation of Peritoneal Dialysis after Late Initiation of Eculizumab in a
Case of Familial Atypical Hemolytic-Uremic Syndrome: A Case Report
#MMPMID28612003
Alonso Valente R
; García Rodríguez GE
; García Marcote Y
; Fidalgo Díaz M
; Becerra Mosquera V
; Novoa García D
; Cordal Martínez T
; Díaz Rodríguez C
Case Rep Nephrol Dial
2017[Jan]; 7
(1
): 18-25
PMID28612003
show ga
BACKGROUND: Atypical hemolytic-uremic syndrome is caused by a thrombotic
microangiopathy and manifests itself with hemolytic anemia, thrombocytopenia, and
organ ischemia. Its etiology is a mutation affecting the genes encoding for
proteins of the complement system. Early treatment with eculizumab (8.6 months
from the moment of presentation), a humanized monoclonal antibody against
complement, is shown to be effective in controlling symptoms and reversing organ
damage. We present a patient with a mutation not previously described in the
literature. Late treatment with eculizumab resulted in a good therapeutic
response, eliminating the need for peritoneal dialysis. CASE PRESENTATION: A
34-year-old woman showed symptoms and laboratory findings consistent with
atypical hemolytic-uremic syndrome. Genetic analysis revealed an unusual mutation
of the complement regulatory gene not seen previously. Due to unavailability of
eculizumab at the time of presentation, conventional treatment was started with
poor response. Late initiation of eculizumab resulted in discontinuation of
peritoneal dialysis and yielded a good and sustained clinical response.
CONCLUSIONS: This case shows that eculizumab treatment for patients with atypical
hemolytic-uremic syndrome, even when initiated many months after beginning on
dialysis, might offer substantial benefits and improve the patients' quality of
life.