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Changing strategies for organ transplantation in atypical haemolytic uraemic syndrome: A tertiary case series
被引:9
作者:
Forbes, Thomas A.
[1
]
Bradbury, Mark G.
[2
]
Goodship, Tim H. J.
[3
]
McKiernan, Patrick J.
[4
]
Milford, David V.
[1
]
机构:
[1] Birmingham Childrens Hosp NHS Fdn Trust, Birmingham, W Midlands, England
[2] Royal Manchester Childrens Hosp, Dept Paediat Nephrol, Manchester M27 1HA, Lancs, England
[3] Newcastle Univ, Inst Med Genet, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
[4] Birmingham Childrens Hosp NHS Fdn Trust, Liver Unit, Birmingham, W Midlands, England
关键词:
atypical haemolytic uraemic syndrome;
chronic;
complement factor H;
eculizumab;
kidney transplantation;
liver transplantation;
paediatric;
renal insufficiency;
COMPLEMENT-FACTOR-H;
LIVER-KIDNEY TRANSPLANTATION;
COFACTOR PROTEIN CD46;
RENAL-TRANSPLANTATION;
MUTATION;
RECURRENCE;
DEFICIENCY;
CHILDREN;
RISK;
AHUS;
D O I:
10.1111/petr.12066
中图分类号:
R72 [儿科学];
学科分类号:
100202 ;
摘要:
We present three cases of organ transplantation for atypical haemolytic uraemic syndrome secondary to complement factor H mutation: one isolated renal transplant; one previously reported isolated liver transplant; and one combined liver and kidney transplant. All three patients were treated prior to the licensing of eculizumab for this condition, and all have had favourable outcomes with maintenance of graft function for years following transplantation. We discuss the evolution of transplantation therapy for aHUS over the last two decades. Transplantation decision-making in aHUS has evolved over this time with expanding knowledge of pathophysiology and genetics, alongside refined plasma exchange and anticoagulation protocols and improved centre experience. Our cases demonstrate how individual patient factors within this heterogeneous condition also underlie transplantation decisions and outcomes. Whilst our cases demonstrate that transplantation in aHUS can be a successful long-term treatment providing good quality of life, worldwide experience has proven that most curative treatment for aHUS strategies represents significant risks. Whether new pharmacotherapies such as eculizumab will alter this risk is yet to be determined.
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页码:E93 / E99
页数:7
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