Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy

Introduction: Atypical hemolytic uremic syndrome (aHUS) is a complement system (CS)–mediated ultrarare disease that manifests as thrombotic microangiopathy (TMA) with preferential small kidney vessels involvement. Transient CS activation is also observed in secondary TMA or in patients at risk of de...

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Main Authors: Maria Martin, Carmen Llorens-Cebria, Juan León-Román, Janire Perurena-Prieto, Víctor Perez-Beltran, Silvia Saumell, Irina B. Torres, Irene Agraz, Joana Sellarès, Natàlia Ramos, Oriol Bestard, Mercedes López, Francesc Moreso, Gema Ariceta, Maria José Soler, Manuel Hernandez-Gonzalez, Conxita Jacobs-Cachá
Format: Article
Language:English
Published: Elsevier 2024-07-01
Series:Kidney International Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2468024924016450
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author Maria Martin
Carmen Llorens-Cebria
Juan León-Román
Janire Perurena-Prieto
Víctor Perez-Beltran
Silvia Saumell
Irina B. Torres
Irene Agraz
Joana Sellarès
Natàlia Ramos
Oriol Bestard
Mercedes López
Francesc Moreso
Gema Ariceta
Maria José Soler
Manuel Hernandez-Gonzalez
Conxita Jacobs-Cachá
author_facet Maria Martin
Carmen Llorens-Cebria
Juan León-Román
Janire Perurena-Prieto
Víctor Perez-Beltran
Silvia Saumell
Irina B. Torres
Irene Agraz
Joana Sellarès
Natàlia Ramos
Oriol Bestard
Mercedes López
Francesc Moreso
Gema Ariceta
Maria José Soler
Manuel Hernandez-Gonzalez
Conxita Jacobs-Cachá
author_sort Maria Martin
collection DOAJ
description Introduction: Atypical hemolytic uremic syndrome (aHUS) is a complement system (CS)–mediated ultrarare disease that manifests as thrombotic microangiopathy (TMA) with preferential small kidney vessels involvement. Transient CS activation is also observed in secondary TMA or in patients at risk of developing aHUS. There is no gold standard test to monitor disease activity; however, the ex vivo C5b-9 deposition test seems to be a good approach. Methods: We assessed the C5b-9 deposition induced by serum samples of patients with aHUS (n = 8) and with TMA associated with kidney (n = 2), lung (n = 1) or hematopoietic stem cell (HSC) transplantation (HSCT, n = 2) during the acute phase of the disease or in remission. As control for transplant-associated TMA (TA-TMA), we analyzed samples of clinically stable kidney and HSC-transplanted patients without signs of TMA. In addition, we studied 1 child with genetic risk of aHUS during an acute infection. Results: In the acute disease phase or in patients with disease activity despite C5 blockade, a significant increase of C5b-9 deposition was detected. In all patients with clinical response to C5 blockade but one, levels of C5b-9 deposition were within the normal range. Finally, we detected increased C5b-9 deposition levels in an asymptomatic child with genetic risk of aHUS when a concomitant otitis episode was ongoing. Conclusion: The ex vivo C5b-9 deposition test is an auspicious tool to monitor CS activity in aHUS and TA-TMA. In addition, we demonstrate that the test may be useful to detect subclinical increase of CS activity, which expands the spectrum of patients that would benefit from a better CS activity assessment.
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spelling doaj-art-f6592b7cf9064b0c9d64e5c60faf187c2025-08-20T02:05:50ZengElsevierKidney International Reports2468-02492024-07-01972227223910.1016/j.ekir.2024.04.022Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic MicroangiopathyMaria Martin0Carmen Llorens-Cebria1Juan León-Román2Janire Perurena-Prieto3Víctor Perez-Beltran4Silvia Saumell5Irina B. Torres6Irene Agraz7Joana Sellarès8Natàlia Ramos9Oriol Bestard10Mercedes López11Francesc Moreso12Gema Ariceta13Maria José Soler14Manuel Hernandez-Gonzalez15Conxita Jacobs-Cachá16Translational Immunology Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainTranslational Immunology Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainPediatric Nephrology, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainHematology Department, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainPediatric Nephrology, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainPediatric Nephrology, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Universitat Autonoma Barcelona, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Redes de Investigación Cooperativa Orientadas a Resultados en Salud (RICORS2040, RD21/0005/0031), Instituto de Salud Carlos III, Madrid, SpainTranslational Immunology Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, SpainNephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Clinical Biochemistry Department, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Barcelona, Spain; Correspondence: Conxita Jacobs Cachá, Nephrology and Transplantation Research Group, Vall d’Hebron Institut de Recerca, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, Passeig Vall d'Hebron 119-129, 08035 Barcelona, Spain.Introduction: Atypical hemolytic uremic syndrome (aHUS) is a complement system (CS)–mediated ultrarare disease that manifests as thrombotic microangiopathy (TMA) with preferential small kidney vessels involvement. Transient CS activation is also observed in secondary TMA or in patients at risk of developing aHUS. There is no gold standard test to monitor disease activity; however, the ex vivo C5b-9 deposition test seems to be a good approach. Methods: We assessed the C5b-9 deposition induced by serum samples of patients with aHUS (n = 8) and with TMA associated with kidney (n = 2), lung (n = 1) or hematopoietic stem cell (HSC) transplantation (HSCT, n = 2) during the acute phase of the disease or in remission. As control for transplant-associated TMA (TA-TMA), we analyzed samples of clinically stable kidney and HSC-transplanted patients without signs of TMA. In addition, we studied 1 child with genetic risk of aHUS during an acute infection. Results: In the acute disease phase or in patients with disease activity despite C5 blockade, a significant increase of C5b-9 deposition was detected. In all patients with clinical response to C5 blockade but one, levels of C5b-9 deposition were within the normal range. Finally, we detected increased C5b-9 deposition levels in an asymptomatic child with genetic risk of aHUS when a concomitant otitis episode was ongoing. Conclusion: The ex vivo C5b-9 deposition test is an auspicious tool to monitor CS activity in aHUS and TA-TMA. In addition, we demonstrate that the test may be useful to detect subclinical increase of CS activity, which expands the spectrum of patients that would benefit from a better CS activity assessment.http://www.sciencedirect.com/science/article/pii/S2468024924016450aHUSbiomarkersC5 blockadeC5b-9 deposition testcomplement systemtransplant-associated TMA
spellingShingle Maria Martin
Carmen Llorens-Cebria
Juan León-Román
Janire Perurena-Prieto
Víctor Perez-Beltran
Silvia Saumell
Irina B. Torres
Irene Agraz
Joana Sellarès
Natàlia Ramos
Oriol Bestard
Mercedes López
Francesc Moreso
Gema Ariceta
Maria José Soler
Manuel Hernandez-Gonzalez
Conxita Jacobs-Cachá
Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
Kidney International Reports
aHUS
biomarkers
C5 blockade
C5b-9 deposition test
complement system
transplant-associated TMA
title Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
title_full Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
title_fullStr Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
title_full_unstemmed Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
title_short Ex vivo C5b-9 Deposition Test to Monitor Complement Activity in Clinical and Subclinical Atypical Hemolytic Uremic Syndrome and in Transplantation-Associated Thrombotic Microangiopathy
title_sort ex vivo c5b 9 deposition test to monitor complement activity in clinical and subclinical atypical hemolytic uremic syndrome and in transplantation associated thrombotic microangiopathy
topic aHUS
biomarkers
C5 blockade
C5b-9 deposition test
complement system
transplant-associated TMA
url http://www.sciencedirect.com/science/article/pii/S2468024924016450
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