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)

被引：0

作者：

Vaisbich, Maria Helena ^{[1
]}

de Andrade, Luis Gustavo Modelli ^{[2
]}

Barbosa, Maria Izabel Neves de Holanda ^{[3
,9
]}

de Castro, Maria Cristina Ribeiro ^{[4
]}

Miranda, Silvana Maria Carvalho ^{[5
]}

Poli-de-Figueiredo, Carlos Eduardo ^{[6
]}

Araujo, Stanley de Almeida ^{[7
]}

Neto, Miguel Ernandes ^{[8
]}

Sobral, Roberta Mendes Lima ^{[9
,10
]}

Neto, Oreste Ferra ^{[10
,11
,12
,13
]}

Neves, Precil Diego Miranda de Menezes ^{[12
,13
,14
]}

da Silva, Cassiano Augusto Braga ^{[14
,15
]}

Pietrobom, Igor Gouveia ^{[15
,16
]}

Palma, Lilian Monteiro Pereira ^{[16
,17
]}

机构：

[1] Univ Fed Sao Paulo, Hosp Clin, Inst Crianca, HCFMUSP, Sao Paulo, SP, Brazil

[2] Univ Estadual Paulista, Dept Med Interna, Sao Paulo, SP, Brazil

[3] Hosp Fed Bonsucesso, Serv Nefrol & Transplante Renal, Rio De Janeiro, RJ, Brazil

[4] Univ Sao Paulo, Unidade Transplante Renal, Sao Paulo, SP, Brazil

[5] Hosp Santa Casa Belo Horizonte, Belo Horizonte, MG, Brazil

[6] Pontificia Univ Catolica Rio Grande do Sul, Porto Alegre, RS, Brazil

[7] Univ Fed Minas Gerais, Ctr Microscopia Eletron, Ctr Microscopia Eletron, Belo Horizonte, MG, Brazil

[8] Hosp Beneficiencia Portuguesa Sao Paulo, Sao Paulo, SP, Brazil

[9] Univ Fed Minas Gerais, Ctr Nefrol Santa Casa Belo Horizonte, Nefrol Pediat, Belo Horizonte, MG, Brazil

[10] Univ Fed Bahia, Hosp Univ Prof Edgard Santos, Unidade Aparelho Urinario, Salvador, BA, Brazil

[11] Univ Fed Mato Grosso do Sul, Hosp Univ Maria Aparecida Pedrossian, Campo Grande, MS, Brazil

[12] Univ Sao Paulo, Dept Nefrol, Sao Paulo, SP, Brazil

[13] Hosp Alemao Oswaldo Cruz, Ctr Dialise & Nefrol, Sao Paulo, SP, Brazil

[14] Clin Senhor Bonfim, Dept Nefrol, Feira De Santana, BA, Brazil

[15] Univ Fed Parana, Curitiba, PR, Brazil

[16] Univ Fed Sao Paulo, Sao Paulo, SP, Brazil

[17] Univ Estadual Campinas, Nefrol Pediat, Campinas, SP, Brazil

来源：

JORNAL BRASILEIRO DE NEFROLOGIA | 2025年 / 47卷 / 02期

关键词：

Guidelines; Diagnosis; Treatment; Atypical Hemolytic Uremic Syndrome; Thrombotic Microangiopathy; COMPLEMENT INHIBITOR ECULIZUMAB; THROMBOTIC MICROANGIOPATHIES; C5; INHIBITOR; MANAGEMENT; DISCONTINUATION; RECURRENCE; PREGNANCY; HUS; OUTCOMES; SAFE;

D O I：

10.1590/2175-8239-JBN-2024-0087en

中图分类号：

R5 [内科学]; R69 [泌尿科学（泌尿生殖系疾病）];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页数：22