Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN)
Abstract Atypical hemolytic uremic syndrome (aHUS) is a rare cause of thrombotic microangiopathy (TMA) caused by the dysregulation of the alternative complement pathway. The diagnosis of TMA is made clinically by the triad: microangiopathic hemolytic anemia, thrombocytopenia, and organ damage (mainl...
| 出版年: | Brazilian Journal of Nephrology |
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| 主要な著者: | , , , , , , , , , , , , , , , |
| フォーマット: | 論文 |
| 言語: | 英語 |
| 出版事項: |
Sociedade Brasileira de Nefrologia
2025-02-01
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| 主題: | |
| オンライン･アクセス: | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0101-28002025000200801&lng=en&tlng=en |
| _version_ | 1849809658234011648 |
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| author | Maria Helena Vaisbich Luis Gustavo Modelli de Andrade Maria Izabel Neves de Holanda Barbosa Maria Cristina Ribeiro de Castro Silvana Maria Carvalho Miranda Carlos Eduardo Poli-de-Figueiredo Stanley de Almeida Araujo Miguel Ernandes Neto Maria Goretti Moreira Guimarães Penido Roberta Mendes Lima Sobral Oreste Ferra Neto Precil Diego Miranda de Menezes Neves Cassiano Augusto Braga da Silva Fellype Carvalho Barreto Igor Gouveia Pietrobom Lilian Monteiro Pereira Palma |
| author_facet | Maria Helena Vaisbich Luis Gustavo Modelli de Andrade Maria Izabel Neves de Holanda Barbosa Maria Cristina Ribeiro de Castro Silvana Maria Carvalho Miranda Carlos Eduardo Poli-de-Figueiredo Stanley de Almeida Araujo Miguel Ernandes Neto Maria Goretti Moreira Guimarães Penido Roberta Mendes Lima Sobral Oreste Ferra Neto Precil Diego Miranda de Menezes Neves Cassiano Augusto Braga da Silva Fellype Carvalho Barreto Igor Gouveia Pietrobom Lilian Monteiro Pereira Palma |
| author_sort | Maria Helena Vaisbich |
| collection | DOAJ |
| container_title | Brazilian Journal of Nephrology |
| description | Abstract Atypical hemolytic uremic syndrome (aHUS) is a rare cause of thrombotic microangiopathy (TMA) caused by the dysregulation of the alternative complement pathway. The diagnosis of TMA is made clinically by the triad: microangiopathic hemolytic anemia, thrombocytopenia, and organ damage (mainly acute kidney injury). The heterogeneity of clinical manifestation and the lack of a gold standard diagnostic test makes the precise diagnosis of aHUS a challenging process that may impact patient management. Until one decade ago, there was no specific treatment for aHUS and patients were submitted to plasma therapy (plasma exchange and/or plasma infusion) and/or liver transplantation, procedures that are not free of serious complications and that do not address the underlying pathophysiology of the disease. Since 2011, an anti-C5 complement monoclonal antibody has been approved by the Food and Drug Administration (FDA) for aHUS patients beginning a new era in treatment. Clinical trials on new complement inhibitors may also add to the treatment portfolio in the future. The Brazilian population is a mixed race with a unique genetic and clinical profile. This consensus aims to offer recommendations for the diagnosis and treatment of patients with aHUS in this population based on expert experience, data from the aHUS Brazilian Registry and literature review. The GRADE system was used to classify the quality of the evidence. |
| format | Article |
| id | doaj-art-04eab33a5dea4e5194bd714fcc7f8a8b |
| institution | Directory of Open Access Journals |
| issn | 2175-8239 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Sociedade Brasileira de Nefrologia |
| record_format | Article |
| spelling | doaj-art-04eab33a5dea4e5194bd714fcc7f8a8b2025-08-20T01:35:38ZengSociedade Brasileira de NefrologiaBrazilian Journal of Nephrology2175-82392025-02-0147210.1590/2175-8239-jbn-2024-0087enRecommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN)Maria Helena Vaisbichhttps://orcid.org/0000-0002-6084-7831Luis Gustavo Modelli de Andradehttps://orcid.org/0000-0002-0230-0766Maria Izabel Neves de Holanda Barbosahttps://orcid.org/0000-0003-3758-4946Maria Cristina Ribeiro de Castrohttps://orcid.org/0000-0002-6731-8369Silvana Maria Carvalho Mirandahttps://orcid.org/0000-0002-3684-6265Carlos Eduardo Poli-de-Figueiredohttps://orcid.org/0000-0002-7333-8884Stanley de Almeida Araujohttps://orcid.org/0000-0001-9996-4405Miguel Ernandes Netohttps://orcid.org/0000-0001-7442-3354Maria Goretti Moreira Guimarães Penidohttps://orcid.org/0000-0002-1534-3861Roberta Mendes Lima Sobralhttps://orcid.org/0000-0002-5963-0168Oreste Ferra Netohttps://orcid.org/0009-0004-2037-6512Precil Diego Miranda de Menezes Neveshttps://orcid.org/0000-0003-1089-6763Cassiano Augusto Braga da Silvahttps://orcid.org/0000-0002-7126-5028Fellype Carvalho Barretohttps://orcid.org/0000-0002-6394-9227Igor Gouveia Pietrobomhttps://orcid.org/0009-0000-6755-1673Lilian Monteiro Pereira Palmahttps://orcid.org/0000-0002-0334-8470Abstract Atypical hemolytic uremic syndrome (aHUS) is a rare cause of thrombotic microangiopathy (TMA) caused by the dysregulation of the alternative complement pathway. The diagnosis of TMA is made clinically by the triad: microangiopathic hemolytic anemia, thrombocytopenia, and organ damage (mainly acute kidney injury). The heterogeneity of clinical manifestation and the lack of a gold standard diagnostic test makes the precise diagnosis of aHUS a challenging process that may impact patient management. Until one decade ago, there was no specific treatment for aHUS and patients were submitted to plasma therapy (plasma exchange and/or plasma infusion) and/or liver transplantation, procedures that are not free of serious complications and that do not address the underlying pathophysiology of the disease. Since 2011, an anti-C5 complement monoclonal antibody has been approved by the Food and Drug Administration (FDA) for aHUS patients beginning a new era in treatment. Clinical trials on new complement inhibitors may also add to the treatment portfolio in the future. The Brazilian population is a mixed race with a unique genetic and clinical profile. This consensus aims to offer recommendations for the diagnosis and treatment of patients with aHUS in this population based on expert experience, data from the aHUS Brazilian Registry and literature review. The GRADE system was used to classify the quality of the evidence.http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0101-28002025000200801&lng=en&tlng=enGuidelinesDiagnosisTreatmentAtypical Hemolytic Uremic SyndromeThrombotic Microangiopathy |
| spellingShingle | Maria Helena Vaisbich Luis Gustavo Modelli de Andrade Maria Izabel Neves de Holanda Barbosa Maria Cristina Ribeiro de Castro Silvana Maria Carvalho Miranda Carlos Eduardo Poli-de-Figueiredo Stanley de Almeida Araujo Miguel Ernandes Neto Maria Goretti Moreira Guimarães Penido Roberta Mendes Lima Sobral Oreste Ferra Neto Precil Diego Miranda de Menezes Neves Cassiano Augusto Braga da Silva Fellype Carvalho Barreto Igor Gouveia Pietrobom Lilian Monteiro Pereira Palma Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) Guidelines Diagnosis Treatment Atypical Hemolytic Uremic Syndrome Thrombotic Microangiopathy |
| title | Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) |
| title_full | Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) |
| title_fullStr | Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) |
| title_full_unstemmed | Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) |
| title_short | Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN) |
| title_sort | recommendations for diagnosis and treatment of atypical hemolytic uremic syndrome ahus an expert consensus statement from the rare diseases committee of the brazilian society of nephrology comdora sbn |
| topic | Guidelines Diagnosis Treatment Atypical Hemolytic Uremic Syndrome Thrombotic Microangiopathy |
| url | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0101-28002025000200801&lng=en&tlng=en |
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