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Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome
Sponsor: Peking Union Medical College Hospital
Summary
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.
Official title: Efficacy and Safety of Tofacitinib in Patients with Refractory Blau Syndrome: a Prospective Cohort Study
Key Details
Gender
All
Age Range
0 Years - 18 Years
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2024-10-01
Completion Date
2028-10-01
Last Updated
2024-10-28
Healthy Volunteers
No
Conditions
Interventions
Janus Kinase Inhibitor
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.
Locations (1)
Peking Union Medical College Hospital
Beijing, China