Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients

Abstract Objective Blau syndrome (BS), a rare, autosomal-dominant autoinflammatory syndrome, is characterized by a clinical triad of granulomatous recurrent uveitis, dermatitis, and symmetric arthritis and associated with mutations of the nucleotide-binding oligomerization domain containing 2 (NOD2)...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Song Zhang, Zhe Cai, Xiaolan Mo, Huasong Zeng
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	JAK inhibitors Tofacitinib Blau syndrome NOD2 Arthritis Pediatrics RJ1-570 Diseases of the musculoskeletal system RC925-935
Acceso en línea:	https://doaj.org/article/947aeaf2900a4e16aa45ea2660aa77f5
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:947aeaf2900a4e16aa45ea2660aa77f5
record_format	dspace
spelling	oai:doaj.org-article:947aeaf2900a4e16aa45ea2660aa77f52021-11-21T12:03:37ZTofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients10.1186/s12969-021-00634-x1546-0096https://doaj.org/article/947aeaf2900a4e16aa45ea2660aa77f52021-11-01T00:00:00Zhttps://doi.org/10.1186/s12969-021-00634-xhttps://doaj.org/toc/1546-0096Abstract Objective Blau syndrome (BS), a rare, autosomal-dominant autoinflammatory syndrome, is characterized by a clinical triad of granulomatous recurrent uveitis, dermatitis, and symmetric arthritis and associated with mutations of the nucleotide-binding oligomerization domain containing 2 (NOD2) gene. Aim of this study was to assess the efficacy of tofacitinib in Chinese paediatric patients with BS. Methods Tofacitinib was regularly administered to three BS patients (Patient 1, Patient 2, and Patient 3) at different dosages: 1.7 mg/day (0.11 mg/kg), 2.5 mg/day (0.12 mg/kg), and 2.5 mg/day (0.33 mg/kg). The clinical manifestations of the patients, magnetic resonance imaging results, serological diagnoses, therapeutic measures and outcomes of treatments are described in this report. Results The clinical characteristics and serological diagnoses of all BS patients were greatly improved after the administration of tofacitinib treatment. All patients reached clinical remission of polyarthritis and improvements in the erythrocyte sedimentation rate (ESR) and levels of C-reactive protein (CRP) and inflammatory cytokines. Conclusion Tofacitinib, a Janus kinase (JAK) inhibitor, is a promising agent for BS patients who have unsatisfactory responses to corticosteroids, traditional disease-modifying antirheumatic drugs, and biological agents.Song ZhangZhe CaiXiaolan MoHuasong ZengBMCarticleJAK inhibitorsTofacitinibBlau syndromeNOD2ArthritisPediatricsRJ1-570Diseases of the musculoskeletal systemRC925-935ENPediatric Rheumatology Online Journal, Vol 19, Iss 1, Pp 1-7 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	JAK inhibitors Tofacitinib Blau syndrome NOD2 Arthritis Pediatrics RJ1-570 Diseases of the musculoskeletal system RC925-935
spellingShingle	JAK inhibitors Tofacitinib Blau syndrome NOD2 Arthritis Pediatrics RJ1-570 Diseases of the musculoskeletal system RC925-935 Song Zhang Zhe Cai Xiaolan Mo Huasong Zeng Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
description	Abstract Objective Blau syndrome (BS), a rare, autosomal-dominant autoinflammatory syndrome, is characterized by a clinical triad of granulomatous recurrent uveitis, dermatitis, and symmetric arthritis and associated with mutations of the nucleotide-binding oligomerization domain containing 2 (NOD2) gene. Aim of this study was to assess the efficacy of tofacitinib in Chinese paediatric patients with BS. Methods Tofacitinib was regularly administered to three BS patients (Patient 1, Patient 2, and Patient 3) at different dosages: 1.7 mg/day (0.11 mg/kg), 2.5 mg/day (0.12 mg/kg), and 2.5 mg/day (0.33 mg/kg). The clinical manifestations of the patients, magnetic resonance imaging results, serological diagnoses, therapeutic measures and outcomes of treatments are described in this report. Results The clinical characteristics and serological diagnoses of all BS patients were greatly improved after the administration of tofacitinib treatment. All patients reached clinical remission of polyarthritis and improvements in the erythrocyte sedimentation rate (ESR) and levels of C-reactive protein (CRP) and inflammatory cytokines. Conclusion Tofacitinib, a Janus kinase (JAK) inhibitor, is a promising agent for BS patients who have unsatisfactory responses to corticosteroids, traditional disease-modifying antirheumatic drugs, and biological agents.
format	article
author	Song Zhang Zhe Cai Xiaolan Mo Huasong Zeng
author_facet	Song Zhang Zhe Cai Xiaolan Mo Huasong Zeng
author_sort	Song Zhang
title	Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
title_short	Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
title_full	Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
title_fullStr	Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
title_full_unstemmed	Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients
title_sort	tofacitinib effectiveness in blau syndrome: a case series of chinese paediatric patients
publisher	BMC
publishDate	2021
url	https://doaj.org/article/947aeaf2900a4e16aa45ea2660aa77f5
work_keys_str_mv	AT songzhang tofacitinibeffectivenessinblausyndromeacaseseriesofchinesepaediatricpatients AT zhecai tofacitinibeffectivenessinblausyndromeacaseseriesofchinesepaediatricpatients AT xiaolanmo tofacitinibeffectivenessinblausyndromeacaseseriesofchinesepaediatricpatients AT huasongzeng tofacitinibeffectivenessinblausyndromeacaseseriesofchinesepaediatricpatients
_version_	1718419319312875520

Tofacitinib effectiveness in Blau syndrome: a case series of Chinese paediatric patients

Ejemplares similares