Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome

Autores
Coccia, Paula Alejandra; Alconcher, Laura; Ferraris, Veronica; Lucarelli, Lucas Ivan; Grillo, Maria Agostina; Arias, Andrea; Saurit, Mariana; Ratto, Viviana Marcela; Raddavero, Claudia Andrea; Dos Santos, Célia; Sánchez Luceros, Analía Gabriela
Año de publicación
2024
Idioma
inglés
Tipo de recurso
artículo
Estado
versión publicada
Descripción
Background:Complement Factor H (CFH) antibodies mediated Hemolytic Uremic Syndrome (HUS) has varying prevalence globally. Plasmapheresis and Immunosuppressive drugs are the standard treatment. Recently, Eculizumab has been reported as an effective alternative. The aim of this study is to report four children with CFH antibodies mediated HUS managed with Eculizumab plus immunosuppression as first line therapy.Methods:A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with CFH antibodies mediated HUS treated with Eculizumab as first-line therapy were included.Results:Four children (aged 6–11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5–23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive CFH antibody results. Hematological signs and kidney function improved after the second Eculizumab dose. Eculizumab was discontinued in three patients after six months. One patient required rituximab due to persistent high CFH antibody titers, discontinuation of Eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6–24 months), no relapses were recorded and all patients ended with normal GFR.ConclusionOur data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse TMA symptoms and improve kidney function in severe patients with CFH antibody mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time.
Fil: Coccia, Paula Alejandra. Hospital Italiano; Argentina
Fil: Alconcher, Laura. Hospital Municipal General de Agudos Doctor José Penna; Argentina
Fil: Ferraris, Veronica. Hospital Italiano; Argentina
Fil: Lucarelli, Lucas Ivan. Hospital Municipal General de Agudos Doctor José Penna; Argentina
Fil: Grillo, Maria Agostina. Hospital Italiano; Argentina
Fil: Arias, Andrea. Hospital Materno Infantil Dr Hector Quintana Jujuy; Argentina
Fil: Saurit, Mariana. Gobierno de la Provincia de Salta. Hospital Publico Materno Infantil.; Argentina
Fil: Ratto, Viviana Marcela. Fundacion Hospitalaria;
Fil: Raddavero, Claudia Andrea. Hospital Italiano; Argentina
Fil: Dos Santos, Célia. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; Argentina
Fil: Sánchez Luceros, Analía Gabriela. Academia Nacional de Medicina de Buenos Aires. Instituto de Investigaciones Hematológicas "Mariano R. Castex"; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; Argentina
Materia
anti factor H antibodiesat
atypical HUS
eculizumab
Thrombotic microangiopathy
Nivel de accesibilidad
acceso abierto
Condiciones de uso
https://creativecommons.org/licenses/by-nc-sa/2.5/ar/
Repositorio
CONICET Digital (CONICET)
Institución
Consejo Nacional de Investigaciones Científicas y Técnicas
OAI Identificador
oai:ri.conicet.gov.ar:11336/267023
Acceder
id CONICETDig_0778930a5a1c03a5f7ce5063d9d5d60b
oai_identifier_str oai:ri.conicet.gov.ar:11336/267023
network_acronym_str CONICETDig
repository_id_str 3498
network_name_str CONICET Digital (CONICET)
spelling Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic SyndromeCoccia, Paula AlejandraAlconcher, LauraFerraris, VeronicaLucarelli, Lucas IvanGrillo, Maria AgostinaArias, AndreaSaurit, MarianaRatto, Viviana MarcelaRaddavero, Claudia AndreaDos Santos, CéliaSánchez Luceros, Analía Gabrielaanti factor H antibodiesatatypical HUSeculizumabThrombotic microangiopathyhttps://purl.org/becyt/ford/3.5https://purl.org/becyt/ford/3Background:Complement Factor H (CFH) antibodies mediated Hemolytic Uremic Syndrome (HUS) has varying prevalence globally. Plasmapheresis and Immunosuppressive drugs are the standard treatment. Recently, Eculizumab has been reported as an effective alternative. The aim of this study is to report four children with CFH antibodies mediated HUS managed with Eculizumab plus immunosuppression as first line therapy.Methods:A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with CFH antibodies mediated HUS treated with Eculizumab as first-line therapy were included.Results:Four children (aged 6–11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5–23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive CFH antibody results. Hematological signs and kidney function improved after the second Eculizumab dose. Eculizumab was discontinued in three patients after six months. One patient required rituximab due to persistent high CFH antibody titers, discontinuation of Eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6–24 months), no relapses were recorded and all patients ended with normal GFR.ConclusionOur data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse TMA symptoms and improve kidney function in severe patients with CFH antibody mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time.Fil: Coccia, Paula Alejandra. Hospital Italiano; ArgentinaFil: Alconcher, Laura. Hospital Municipal General de Agudos Doctor José Penna; ArgentinaFil: Ferraris, Veronica. Hospital Italiano; ArgentinaFil: Lucarelli, Lucas Ivan. Hospital Municipal General de Agudos Doctor José Penna; ArgentinaFil: Grillo, Maria Agostina. Hospital Italiano; ArgentinaFil: Arias, Andrea. Hospital Materno Infantil Dr Hector Quintana Jujuy; ArgentinaFil: Saurit, Mariana. Gobierno de la Provincia de Salta. Hospital Publico Materno Infantil.; ArgentinaFil: Ratto, Viviana Marcela. Fundacion Hospitalaria;Fil: Raddavero, Claudia Andrea. Hospital Italiano; ArgentinaFil: Dos Santos, Célia. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; ArgentinaFil: Sánchez Luceros, Analía Gabriela. Academia Nacional de Medicina de Buenos Aires. Instituto de Investigaciones Hematológicas "Mariano R. Castex"; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; ArgentinaSpringer2024-06info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttp://purl.org/coar/resource_type/c_6501info:ar-repo/semantics/articuloapplication/pdfapplication/pdfhttp://hdl.handle.net/11336/267023Coccia, Paula Alejandra; Alconcher, Laura; Ferraris, Veronica; Lucarelli, Lucas Ivan; Grillo, Maria Agostina; et al.; Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome; Springer; Pediatric Nephrology; 6-2024; 1-140931-041XCONICET DigitalCONICETenginfo:eu-repo/semantics/altIdentifier/doi/10.21203/rs.3.rs-4492192/v1info:eu-repo/semantics/altIdentifier/url/https://www.researchsquare.com/article/rs-4492192/v1info:eu-repo/semantics/openAccesshttps://creativecommons.org/licenses/by-nc-sa/2.5/ar/reponame:CONICET Digital (CONICET)instname:Consejo Nacional de Investigaciones Científicas y Técnicas2025-09-29T09:42:23Zoai:ri.conicet.gov.ar:11336/267023instacron:CONICETInstitucionalhttp://ri.conicet.gov.ar/Organismo científico-tecnológicoNo correspondehttp://ri.conicet.gov.ar/oai/requestdasensio@conicet.gov.ar; lcarlino@conicet.gov.arArgentinaNo correspondeNo correspondeNo correspondeopendoar:34982025-09-29 09:42:23.416CONICET Digital (CONICET) - Consejo Nacional de Investigaciones Científicas y Técnicasfalse
dc.title.none.fl_str_mv Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
title Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
spellingShingle Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
Coccia, Paula Alejandra
anti factor H antibodiesat
atypical HUS
eculizumab
Thrombotic microangiopathy
title_short Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
title_full Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
title_fullStr Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
title_full_unstemmed Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
title_sort Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome
dc.creator.none.fl_str_mv Coccia, Paula Alejandra
Alconcher, Laura
Ferraris, Veronica
Lucarelli, Lucas Ivan
Grillo, Maria Agostina
Arias, Andrea
Saurit, Mariana
Ratto, Viviana Marcela
Raddavero, Claudia Andrea
Dos Santos, Célia
Sánchez Luceros, Analía Gabriela
author Coccia, Paula Alejandra
author_facet Coccia, Paula Alejandra
Alconcher, Laura
Ferraris, Veronica
Lucarelli, Lucas Ivan
Grillo, Maria Agostina
Arias, Andrea
Saurit, Mariana
Ratto, Viviana Marcela
Raddavero, Claudia Andrea
Dos Santos, Célia
Sánchez Luceros, Analía Gabriela
author_role author
author2 Alconcher, Laura
Ferraris, Veronica
Lucarelli, Lucas Ivan
Grillo, Maria Agostina
Arias, Andrea
Saurit, Mariana
Ratto, Viviana Marcela
Raddavero, Claudia Andrea
Dos Santos, Célia
Sánchez Luceros, Analía Gabriela
author2_role author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv anti factor H antibodiesat
atypical HUS
eculizumab
Thrombotic microangiopathy
topic anti factor H antibodiesat
atypical HUS
eculizumab
Thrombotic microangiopathy
purl_subject.fl_str_mv https://purl.org/becyt/ford/3.5
https://purl.org/becyt/ford/3
dc.description.none.fl_txt_mv Background:Complement Factor H (CFH) antibodies mediated Hemolytic Uremic Syndrome (HUS) has varying prevalence globally. Plasmapheresis and Immunosuppressive drugs are the standard treatment. Recently, Eculizumab has been reported as an effective alternative. The aim of this study is to report four children with CFH antibodies mediated HUS managed with Eculizumab plus immunosuppression as first line therapy.Methods:A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with CFH antibodies mediated HUS treated with Eculizumab as first-line therapy were included.Results:Four children (aged 6–11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5–23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive CFH antibody results. Hematological signs and kidney function improved after the second Eculizumab dose. Eculizumab was discontinued in three patients after six months. One patient required rituximab due to persistent high CFH antibody titers, discontinuation of Eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6–24 months), no relapses were recorded and all patients ended with normal GFR.ConclusionOur data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse TMA symptoms and improve kidney function in severe patients with CFH antibody mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time.
Fil: Coccia, Paula Alejandra. Hospital Italiano; Argentina
Fil: Alconcher, Laura. Hospital Municipal General de Agudos Doctor José Penna; Argentina
Fil: Ferraris, Veronica. Hospital Italiano; Argentina
Fil: Lucarelli, Lucas Ivan. Hospital Municipal General de Agudos Doctor José Penna; Argentina
Fil: Grillo, Maria Agostina. Hospital Italiano; Argentina
Fil: Arias, Andrea. Hospital Materno Infantil Dr Hector Quintana Jujuy; Argentina
Fil: Saurit, Mariana. Gobierno de la Provincia de Salta. Hospital Publico Materno Infantil.; Argentina
Fil: Ratto, Viviana Marcela. Fundacion Hospitalaria;
Fil: Raddavero, Claudia Andrea. Hospital Italiano; Argentina
Fil: Dos Santos, Célia. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; Argentina
Fil: Sánchez Luceros, Analía Gabriela. Academia Nacional de Medicina de Buenos Aires. Instituto de Investigaciones Hematológicas "Mariano R. Castex"; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas. Instituto de Medicina Experimental. Academia Nacional de Medicina de Buenos Aires. Instituto de Medicina Experimental; Argentina
description Background:Complement Factor H (CFH) antibodies mediated Hemolytic Uremic Syndrome (HUS) has varying prevalence globally. Plasmapheresis and Immunosuppressive drugs are the standard treatment. Recently, Eculizumab has been reported as an effective alternative. The aim of this study is to report four children with CFH antibodies mediated HUS managed with Eculizumab plus immunosuppression as first line therapy.Methods:A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with CFH antibodies mediated HUS treated with Eculizumab as first-line therapy were included.Results:Four children (aged 6–11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5–23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive CFH antibody results. Hematological signs and kidney function improved after the second Eculizumab dose. Eculizumab was discontinued in three patients after six months. One patient required rituximab due to persistent high CFH antibody titers, discontinuation of Eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6–24 months), no relapses were recorded and all patients ended with normal GFR.ConclusionOur data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse TMA symptoms and improve kidney function in severe patients with CFH antibody mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time.
publishDate 2024
dc.date.none.fl_str_mv 2024-06
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
http://purl.org/coar/resource_type/c_6501
info:ar-repo/semantics/articulo
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/11336/267023
Coccia, Paula Alejandra; Alconcher, Laura; Ferraris, Veronica; Lucarelli, Lucas Ivan; Grillo, Maria Agostina; et al.; Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome; Springer; Pediatric Nephrology; 6-2024; 1-14
0931-041X
CONICET Digital
CONICET
url http://hdl.handle.net/11336/267023
identifier_str_mv Coccia, Paula Alejandra; Alconcher, Laura; Ferraris, Veronica; Lucarelli, Lucas Ivan; Grillo, Maria Agostina; et al.; Eculizumab as first line treatment for patients with severe presentation of Complement Factor H antibodies mediated Hemolytic Uremic Syndrome; Springer; Pediatric Nephrology; 6-2024; 1-14
0931-041X
CONICET Digital
CONICET
dc.language.none.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv info:eu-repo/semantics/altIdentifier/doi/10.21203/rs.3.rs-4492192/v1
info:eu-repo/semantics/altIdentifier/url/https://www.researchsquare.com/article/rs-4492192/v1
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
https://creativecommons.org/licenses/by-nc-sa/2.5/ar/
eu_rights_str_mv openAccess
rights_invalid_str_mv https://creativecommons.org/licenses/by-nc-sa/2.5/ar/
dc.format.none.fl_str_mv application/pdf
application/pdf
dc.publisher.none.fl_str_mv Springer
publisher.none.fl_str_mv Springer
dc.source.none.fl_str_mv reponame:CONICET Digital (CONICET)
instname:Consejo Nacional de Investigaciones Científicas y Técnicas
reponame_str CONICET Digital (CONICET)
collection CONICET Digital (CONICET)
instname_str Consejo Nacional de Investigaciones Científicas y Técnicas
repository.name.fl_str_mv CONICET Digital (CONICET) - Consejo Nacional de Investigaciones Científicas y Técnicas
repository.mail.fl_str_mv dasensio@conicet.gov.ar; lcarlino@conicet.gov.ar
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score 13.070432

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