This is a prospective cohort study to observe the efficacy and safety of Tofacitinib in children with Blau syndrome (BS). The investigators would analyze the rate of remission or low disease activity after treatment as well as changes in inflammatory markers, patients' and physician's global assessment of disease activity to determine the efficacy and safety of Tofacitinib.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Tofacitinib is used according to weight: 5\~\<7kg，2mg；7\~\<10kg，2.5mg；10\~\<15kg，3mg；15\~\<25kg，3.5mg；25\~\<40kg，4mg；≥40kg，5mg. All is twice a day.
Peking Union Medical College Hospital
Beijing, China
rate of remission or low disease activity
Time frame: From enrollment to the end of treatment at 6 months
RCB(Response in Chinese children with Blau syndrome) 30, 50, 70 response rates
Time frame: From enrollment to the end of treatment at 3, 6, 9, 12 months
Changes in inflammatory markers (including erythrocyte sedimentation rate, C reactive protein), cytokines (including IL-1β, IL-6, IL-17, IL-18, TNFα, IFN γ) and expression of type I interferon-stimulated genes over baseline
Changes are standardized as the ratio of the numerical difference before and after treatment to the baseline value
Time frame: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Proportion of recurrent uveitis
Time frame: From enrollment to the end of treatment at 12 months
Incidence of new organ involvement
Time frame: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Number of participants with adverse effect
Time frame: From enrollment to the end of treatment at 12 months
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