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 50 条
  • [41] COMPLEMENT FACTOR H FUNCTIONAL ASSAY MAY HELP TO MONITOR ATYPICAL HAEMOLYTIC URAEMIC SYNDROME: A PILOT STUDY
    Massart, A.
    Golmarvi, S.
    Hachimi-Idrissi, S.
    Broeders, E.
    Tournay, Y.
    Nortier, J.
    Abramowicz, D.
    Tielemans, C.
    Stordeur, P.
    ACTA CLINICA BELGICA, 2013, 68 (01): : 9 - 14
  • [42] Functional analyses indicate a pathogenic role of factor H autoantibodies in atypical haemolytic uraemic syndrome
    Strobel, Stefanie
    Hoyer, Peter F.
    Mache, Christoph J.
    Sulyok, Endre
    Liu, Wei-shih
    Richter, Heiko
    Oppermann, Martin
    Zipfel, Peter F.
    Jozsi, Mihaly
    NEPHROLOGY DIALYSIS TRANSPLANTATION, 2010, 25 (01) : 136 - 144
  • [43] Complement factor H mutation associated with membranoproliferative glomerulonephritis with transformation to atypical haemolytic uraemic syndrome
    van Doorn, Karin Janssen
    Dirinck, Eveline
    Verpooten, Gert A.
    Couttenye, Marie M.
    CLINICAL KIDNEY JOURNAL, 2013, 6 (02): : 216 - 219
  • [44] Eculizumab: A Review of Its Use in Atypical Haemolytic Uraemic Syndrome
    Gillian M. Keating
    Drugs, 2013, 73 : 2053 - 2066
  • [45] Eculizumab: A guide to its use in atypical haemolytic uraemic syndrome
    Keating G.M.
    Drugs & Therapy Perspectives, 2014, 30 (5) : 166 - 172
  • [46] Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan
    Ito, Shuichi
    Hataya, Hiroshi
    Ashida, Akira
    Hamada, Riku
    Ishikawa, Tomoaki
    Ishikawa, Yumiko
    Shimono, Akihiko
    Konomoto, Takao
    Miyazawa, Tomoki
    Ogura, Masao
    Tanaka, Kazuki
    Kagami, Shoji
    NEPHROLOGY DIALYSIS TRANSPLANTATION, 2023, 38 (02) : 414 - 424
  • [47] Clinical Practice Guidelines for the management of atypical Haemolytic Uraemic Syndrome in the United Kingdom
    Taylor, C. Mark
    Machin, Sam
    Wigmore, Stephen J.
    Goodship, Tim H. J.
    BRITISH JOURNAL OF HAEMATOLOGY, 2010, 148 (01) : 37 - 47
  • [48] Eculizumab in an anephric patient with atypical haemolytic uraemic syndrome and advanced vascular lesions
    Bekassy, Zivile D.
    Kristoffersson, Ann-Charlotte
    Cronqvist, Mats
    Roumenina, Lubka T.
    Rybkine, Tania
    Vergoz, Laura
    Hue, Christophe
    Fremeaux-Bacchi, Veronique
    Karpman, Diana
    NEPHROLOGY DIALYSIS TRANSPLANTATION, 2013, 28 (11) : 2899 - 2907
  • [49] Characterization of genetic predisposition and autoantibody profile in atypical haemolytic-uraemic syndrome
    Gurjar, Bahadur Singh
    Manikanta Sriharsha, Tholu
    Bhasym, Angika
    Prabhu, Savit
    Puraswani, Mamta
    Khandelwal, Priyanka
    Saini, Himanshi
    Saini, Savita
    Verma, Anita Kamra
    Chatterjee, Priyadarshini
    Guchhait, Prasenjit
    Bal, Vineeta
    George, Anna
    Rath, Satyajit
    Sahu, Arvind
    Sharma, Amita
    Hari, Pankaj
    Sinha, Aditi
    Bagga, Arvind
    IMMUNOLOGY, 2018, 154 (04) : 663 - 672
  • [50] Haemolytic uraemic syndrome caused by factor H mutation: is single kidney transplantation under intensive plasmatherapy an option?
    Hirt-Minkowski, Patricia
    Schaub, Stefan
    Mayr, Michael
    Schifferli, Juerg A.
    Dickenmann, Michael
    Fremeaux-Bacchi, Veronique
    Steiger, Juerg
    NEPHROLOGY DIALYSIS TRANSPLANTATION, 2009, 24 (11) : 3548 - 3551