Clinical benefits of TNF-α inhibitors in Chinese adult patients with NLRP3-associated autoinflammatory disease.
J Intern Med
; 290(4): 878-885, 2021 10.
Article
em En
| MEDLINE
| ID: mdl-34037998
ABSTRACT
BACKGROUND:
Nucleotide-binding oligomerization domain-like receptor family, pyrin domain containing 3 (NLRP3)-associated autoinflammatory disease (NLRP3-AID) is a rare, heterogeneous disease entity associated with mutations in NLRP3. Biologic therapy for NLRP3-AID yields diverse results.OBJECTIVES:
We aimed to evaluate the clinical features and outcomes of Chinese adult patients with NLRP3-AID who were treated with tumour necrosis factor (TNF)-α inhibitors.METHODS:
Five patients with NLRP3-AID were diagnosed and treated with TNF-α inhibitors at Peking Union Medical College Hospital between 2017 and 2020 and were followed up for 6 to 12 months. All patients were systematically studied for treatment outcomes, including clinical manifestations and inflammatory markers.RESULTS:
All five adult NLRP3-AID patients were Chinese Han, and four patients were males. The mean age at disease onset was 4.2 ± 4.1 years, and the mean time of diagnosis delay was 19.8 ± 6 years. All patients received TNF-α inhibitors with or without methotrexate/prednisone. During follow-up, all patients achieved remarkable clinical remission of skin lesions and polyarthritis and showed improvements in acute-phase reactants, inflammatory cytokines, patient visual analogue scale, physician global assessment and 36-item Short Form (SF-36).CONCLUSIONS:
Early diagnosis and effective therapy for NLRP3-AID are essential for avoiding irreversible organ damage. TNF-α inhibitors might serve as a therapeutic alternative for patients with NLRP3-AID who have unsatisfactory responses or no access to interleukin-1 inhibitors.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Hereditárias Autoinflamatórias
/
Proteína 3 que Contém Domínio de Pirina da Família NLR
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Inibidores do Fator de Necrose Tumoral
Tipo de estudo:
Risk_factors_studies
/
Screening_studies
Limite:
Adult
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Female
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Humans
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Male
País/Região como assunto:
Asia
Idioma:
En
Revista:
J Intern Med
Assunto da revista:
MEDICINA INTERNA
Ano de publicação:
2021
Tipo de documento:
Article