Atypical hemolytic uremic syndrome

Yoshida, Yoko; Kato, Hideki; Nangaku, Masaomi

doi:10.1186/s41100-016-0088-1

Cited by 7 publications

(6 citation statements)

References 78 publications

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“…Atypical haemolytic uraemic syndrome (aHUS) is a form of thrombotic microangiopathy (TMA) that is defined by a triad of non-immune microangiopathic haemolytic anaemia (MAHA), thrombocytopaenia and acute kidney injury (AKI) [ 1 , 2 ]. In the USA, the annual incidence is estimated to be two cases per million and the prevalence is reported to be 3.3 per million children [ 1 , 3 ]. In Japan, ∼100–200 patients are diagnosed with aHUS [ 1 , 3 ].…”

Section: Introductionmentioning

confidence: 99%

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Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan

Ito

Hataya

Ashida

et al. 2022

Nephrology Dialysis Transplantation

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Background Eculizumab was approved for atypical haemolytic-uremic syndrome (aHUS) in Japan in 2013. Post-marketing surveillance (PMS) was mandated by regulatory authorities to assess the safety and effectiveness of eculizumab in patients with aHUS in the real-world setting. Methods Paediatric patients in the PMS cohort who were < 18 years old at first administration of eculizumab and diagnosed with aHUS (excluding Shiga toxin-producing Escherichia coli HUS, thrombotic thrombocytopenic purpura, and secondary thrombotic microangiopathy [TMA]) were included in the effectiveness and safety analysis. Clinical endpoints of effectiveness (complete TMA response, TMA event-free status, platelet count [PLT] and lactate dehydrogenase [LDH] normalization, serum creatinine [sCr] decrease, and estimated glomerular filtration rate [eGFR] improvement) were analysed in patients treated with ≥ 1 dose of eculizumab. Serious adverse events (SAEs) were also evaluated. Results Forty paediatric patients (median age 5 years) were included. Median eculizumab treatment duration was 66 weeks. PLT, LDH and eGFR significantly improved at 10 days post-treatment. Complete TMA response, haematologic normalization, sCr decrease, eGFR improvement, and TMA event-free status were achieved by 73.3%, 73.3%, 70.0%, 78.3%, and 77.5%, respectively. Discontinuation criteria were met by 18 patients: 13 patients maintained treatment discontinuation at the end of observation; and 5 patients, including one patient with aHUS relapse, continued the treatment but extended treatment interval. During eculizumab treatment, 59 SAEs (0.66/person-year) were reported. Although four deaths were reported, none of them were related to eculizumab. Conclusion Eculizumab was well tolerated and effective for paediatric patients with aHUS in the real-world setting in Japan.

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Section: Introductionmentioning

confidence: 99%

“…In the USA, the annual incidence is estimated to be two cases per million and the prevalence is reported to be 3.3 per million children [ 1 , 3 ]. In Japan, ∼100–200 patients are diagnosed with aHUS [ 1 , 3 ].…”

Section: Introductionmentioning

confidence: 99%

Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan

Ito

Hataya

Ashida

et al. 2022

Nephrology Dialysis Transplantation

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“…14,15 Due to the rarity of the disease, global aHUS disease epidemiology is not well known. 16 When reported, incidence and prevalence estimates are often combined with similar disorders such as shiga toxin-producing e.coli associated hemolytic uremic syndrome (STEC-HUS) or thrombotic thrombocytopenic purpura (TTP), leading to inaccurate estimates of the true number of individuals diagnosed with aHUS. [17][18][19] Although population-based estimates exist for some countries, there are gaps in global aHUS incidence and prevalence.…”

Section: Introductionmentioning

confidence: 99%

<p>Epidemiology of Atypical Hemolytic Uremic Syndrome: A Systematic Literature Review</p>

Yan¹,

Desai²,

Gullapalli³

et al. 2020

CLEP

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Atypical hemolytic uremic syndrome (aHUS) is a rare but severe disorder that frequently has a genetic component and results from the overactivation of the alternative complement pathway. As research moves toward improved diagnosis and therapy of aHUS, it will be important to better understand its epidemiology. Our objective was to conduct a systematic literature review to assess the incidence and prevalence estimates of aHUS globally. A comprehensive literature search was conducted in Embase and MEDLINE. Additionally, practice guidelines, databases of national/international organizations, and regulatory agencies were searched. From 2960 publications identified via MEDLINE and Embase, 105 publications were eligible for full-text screening, and a total of eight full-text articles met eligibility criteria for inclusion. Regional epidemiologic estimates were obtained for Europe and Oceania. Country-specific data were available for France, Norway, Australia, and Italy. Four of the identified studies reported on the prevalence of aHUS, prevalence in the age group of 20 years or younger was ranging from 2.2 to 9.4 per million population, while the only study that reported prevalence in all ages showed a prevalence of 4.9 per million population. Six studies reported on the incidence of aHUS, annual incidence in the age group of 20 years or younger was ranging from 0.26 to 0.75 per million population, and for all ages, annual incidence was ranging from 0.23 to 1.9 per million population. To our knowledge, this is the first systematic review conducted to provide a comprehensive overview of global incidence and prevalence estimates of aHUS. In general, incidence estimates were similar across all the studies; however, prevalence data were found to be more variable. Study limitations were related to inconsistencies in the definitions of aHUS between studies and also a dearth of epidemiological studies assessing incidence and prevalence of aHUS outside of Europe.

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“…A primeira é secundária à infecção por alguns sorotipos de Escherichia. coli, produtores da toxina Shiga-like, com quadros de diarreia sanguinolenta característicos, recorrência rara e melhor prognóstico 3 . A forma "atípica" (SHUa) pode ser esporádica ou familiar e tem prognóstico desfavorável, com mortalidade de 10% a 15% durante a fase aguda.…”

Section: Introductionunclassified

“…As formas genéticas da SHUa podem ser de herança familiar ou não, e estão mais comumente associadas com rápida progressão para estágio final de doença renal e alto risco de manifestação pós-transplante 3-4 . Apesar das várias origens possíveis para a forma atípica, estudos mostram que mutações que comprometem a expressão e/ou funcionalidade das proteínas reguladoras da via alternativa do sistema complemento [fator H (CFH), cofator proteína de membrana (MCP ou CD46), fator I (CFI)], da trombomodulina (THBD) e de duas proteínas da C3 convertase, C3 e fator B (CFB) exercem importante papel na patogênese da SHUa. Em até 70% das crianças e adultos acometidos por uma ou várias anomalias do sistema complemento, as mutações genéticas são identificadas, enquanto nos outros 30% não há identificação de mutações genéticas conhecidas 3 .…”

Section: Introductionunclassified

Uso do eculizumab na síndrome hemolítico urêmica atípica associada a comprometimento da função renal: revisão integrativa

Lima

Teixeira

Linhares

et al. 2019

ACS

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Introdução: a Síndrome Hemolítico Urêmica é uma doença rara e grave que se define pela anemia hemolítica microangiopática não imune, trombocitopenia e insuficiência renal aguda. Objetivo: identificar informações disponíveis na literatura acerca do uso do eculizumab no tratamento da síndrome hemolítico urêmica atípica com comprometimento da função renal associado. Material e métodos: trata-se de uma revisão integrativa realizada nas seguintes bases de dados: Literatura LatinoAmericana e do Caribe em Ciências da Saúde e Medical Literature Analysis and Retrieval System Online. O estudo foi norteado pela seguinte questão: O que há na literatura acerca do uso do eculizumab no tratamento da SHUa com comprometimento da função renal? Para busca de artigos foram utilizados três descritores em português, inglês e espanhol. O recorte temporal foi de 2007 à 2017. Resultados: Inicialmente foram localizados dez artigos e após a aplicação dos critérios de inclusão e exclusão foram selecionados sete deles. Todos os artigos relatam melhora da função renal logo no início da terapia e não há consenso quanto a indicação de descontinuação do uso do eculizumab nesses pacientes, mesmo nos casos onde houve completa remissão da patologia. Conclusão: Ainda são poucos os estudos sobre o uso do eculizumab. Estudos dessa natureza tem relevante contribuição para o avanço da terapia em casos raros como a SHUa, tendo em vista que o uso do anticorpo pode aumentar a sobrevida e melhorar a qualidade de vida de pessoas acometidas com a doença.

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Atypical hemolytic uremic syndrome

Cited by 7 publications

References 78 publications

Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan

Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan

<p>Epidemiology of Atypical Hemolytic Uremic Syndrome: A Systematic Literature Review</p>

Uso do eculizumab na síndrome hemolítico urêmica atípica associada a comprometimento da função renal: revisão integrativa

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