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Updated: May 5, 2026

A Method for Screening and Validation of Resistant Mutations Against Kinase Inhibitors
Published on: December 7, 2014
EFFICACY AND TOLERABILITY OF JANUS KINASE INHIBITOR TOFACITINIB IN JUVENILE LINEAR SCLERODERMA. CASE SERIES OF 5
M Osminina1, N Podchernyaeva1, V Seraya1
11Federal State Autonomous Educational Institution of Higher Education I.M. Sechenov First Moscow State Medical University, Russia.
Aim:
to analyze the efficacy of the Janus kinase inhibitor tofacitinib (TF) in children with linear form of juvenile localized scleroderma (JLSL).
Materials And Methods:
5 patients with JLSL were treated with TF, we evaluated the efficacy of therapy retrospectively.
Results:
The mean disease duration prior to TF therapy was high at 71 months. All patients did not respond well to glucocorticosteroid (Glu) and methotrexate (MTX) therapy, two did not respond to combination therapy of Glu, MTX, mycophenolate mofetil (MFM), which prompted the initiation of TF therapy. All patients received TF in combination with MFM or MTX or GCS. The mean duration of TF therapy was 12.8±1.78 months. From 3 to 6 months of TF therapy, we observed positive changes in scleroderma skin and joint involvement. The positive results continued to increase up to the 12th month of treatment, as evidenced by the reduction of the mLoSSI and LoSDI indices. We decided to continue treatment for up to 18 months. The patients tolerated TF well, there were no serious infections, abnormalities in liver function.
Conclusion:
Our case series showed a significant improvement in skin and joint damage after 12 months of TF therapy in resistant JLSL patients. Further experience with TF is needed to determine its place in the treatment of juvenile scleroderma.
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