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.
引用
收藏
页码:E93 / E99
页数:7
相关论文
共 29 条
[1]   Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation [J].
Al-Akash, Samhar I. ;
Almond, P. Stephen ;
Savell, Van H., Jr. ;
Gharaybeh, Salam I. ;
Hogue, Cris .
PEDIATRIC NEPHROLOGY, 2011, 26 (04) :613-619
[2]  
Alonso A, 2011, PEDIATR NEPHROL, V26, P1598
[3]   Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: Prognostic significance-of genetic background [J].
Bresin, Elena ;
Daina, Erica ;
Noris, Marina ;
Castelletti, Federica ;
Stefanov, Rumen ;
Hill, Prudence ;
Goodship, Timothy H. J. ;
Remuzzi, Giuseppe .
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2006, 1 (01) :88-99
[4]   Attempted treatment of factor H deficiency by liver transplantation [J].
Cheong, HI ;
Lee, BS ;
Kang, HG ;
Hahn, H ;
Suh, KS ;
Ha, IS ;
Choi, Y .
PEDIATRIC NEPHROLOGY, 2004, 19 (04) :454-458
[5]   Complement factor H polymorphism, complement activators, and risk of age-related macular degeneration [J].
Despriet, Dominiek D. G. ;
Klaver, Caroline C. W. ;
Witteman, Jacqueline C. M. ;
Bergen, Arthur A. B. ;
Kardys, Isabella ;
de Maat, Moniek P. M. ;
Boekhoorn, Sharmila S. ;
Vingerling, Johannes R. ;
Hofman, Albert ;
Oostra, Ben A. ;
Uitterlinden, Andre G. ;
Stijnen, Theo ;
van Duijn, Cornelia M. ;
de Jong, Paulus T. V. M. .
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2006, 296 (03) :301-309
[6]   Genetic and functional analyses of membrane cofactor protein (CD46) mutations in atypical hemolytic uremic syndrome [J].
Fremeaux-Bacchi, Veronique ;
Moulton, Elizabeth A. ;
Kavanagh, David ;
Dragon-Durey, Marie-Agnes ;
Blouin, Jacques ;
Caudy, Amy ;
Arzouk, Nadia ;
Cleper, Roxanna ;
Francois, Maud ;
Guest, Genevieve ;
Pourrat, Jacques ;
Seligman, Roland ;
Fridman, Wolf Herman ;
Loirat, Chantal ;
Atkinson, John P. .
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2006, 17 (07) :2017-2025
[7]   Successful Isolated Liver Transplantation in a Child with Atypical Hemolytic Uremic Syndrome and a Mutation in Complement Factor H [J].
Haller, W. ;
Milford, D. V. ;
Goodship, T. H. J. ;
Sharif, K. ;
Mirza, D. F. ;
McKiernan, P. J. .
AMERICAN JOURNAL OF TRANSPLANTATION, 2010, 10 (09) :2142-2147
[8]   Successful liver-kidney transplantation in two children with aHUS caused by a mutation in complement factor H [J].
Jalanko, H. ;
Peltonen, S. ;
Koskinen, A. ;
Puntila, J. ;
Isoniemi, H. ;
Holmberg, C. ;
Pinomaki, A. ;
Armstrong, E. ;
Koivusalo, A. ;
Tukiainen, E. ;
Makisalo, H. ;
Saland, J. ;
Remuzzi, G. ;
de Cordoba, S. ;
Lassila, R. ;
Meri, S. ;
Jokiranta, T. S. .
AMERICAN JOURNAL OF TRANSPLANTATION, 2008, 8 (01) :216-221
[9]   Where next with atypical hemolytic uremic syndrome? [J].
Jokiranta, T. Sakari ;
Zipfel, Peter F. ;
Fremeaux-Bacchi, Veronique ;
Taylor, C. Mark ;
Goodship, Timothy J. H. ;
Noris, Marina .
MOLECULAR IMMUNOLOGY, 2007, 44 (16) :3889-3900
[10]   Genetics and complement in atypical HUS [J].
Kavanagh, David ;
Goodship, Tim .
PEDIATRIC NEPHROLOGY, 2010, 25 (12) :2431-2442