Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome: an update.
Pediatr Nephrol
; 33(3): 457-461, 2018 03.
Article
em En
| MEDLINE
| ID: mdl-29046944
ABSTRACT
BACKGROUND:
Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear.METHODS:
Here, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission.RESULTS:
We report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5-60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4-24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8-80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed.CONCLUSION:
Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Proteínas do Sistema Complemento
/
Monitoramento de Medicamentos
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Inativadores do Complemento
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Anticorpos Monoclonais Humanizados
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Síndrome Hemolítico-Urêmica Atípica
Tipo de estudo:
Observational_studies
/
Risk_factors_studies
Limite:
Adolescent
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Adult
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Child
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Child, preschool
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Female
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Humans
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Infant
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Male
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Middle aged
Idioma:
En
Revista:
Pediatr Nephrol
Assunto da revista:
NEFROLOGIA
/
PEDIATRIA
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
Itália