FRI0459 TOFACITINIB TREATMENT IN RECALCITRANT JDM PATIENTS

Abstract

Background:Juvenile dermatomyositis (JDM) is a systemic, autoimmune inflammatory muscle disorder and vasculopathy that affects children younger than 18 years. Although the cause of JDM remains unknown it is clear that genetic and environmental influences play a role in the aetiology. New treatments are becoming available and being tested through international multicentre trials. Increasing evidence suggests a role for types I and II IFN in juvenile and adult dermatomyositis, including elevated IFN-response gene signatures in the muscle, skin and blood. It has recently been reported that patients with refractory JDM responded well to treatment with tofacitinib, a JAK inhibitor, with corresponding downregulation in selected IFN-response genes.Objectives:In this study, we evaluated our cases with resistant JDM who received tofacitinib treatment.Methods:Six patients who received tofacitinib because of severe skin involvement of JDM were included in the study. The data were obtained retrospectively from the hospital records.Results:The age ranges of the cases were between 7-17 years and the ratio of girls and boys was 1 (3/3). The age of diagnosis was between 2-13 years, and the follow-up period was between 3-9 years. Calcinosis cutis in 5 cases, decreased muscle strength in 3 cases, joint involvement in 4 cases were detected.. Systemic steroids, methotrexate, and non-steroid anti-inflammatory drugs were given in all cases before tofacitinib treatment. Pamidronate was used in 4 cases because of severe skin calcinosis, high dose intravenous immunoglobulin in 4 cases, mycophenolate mofetil in 3 cases, rituximab in 3 cases and cyclophosphamide in 1 case previously. Tofacitinib treatment (10mg/gün) was started in six cases with treatment-resistant JDM. Five cases had been treated with tofacitinib for 6-24 month intervals. The treatment was discontinued in one case because of severe allergic reaction. Variable level of improvement were detected in the skin findings of all cases during the therapy period. The treatment was interrupted for 1 month in only one case due to neutropenia.Conclusion:Tofacitinib seems to be an effective and safe treatment option in patients with JDM who are resistant to conventional treatments. More studies are needed on this subject.Disclosure of Interests:None declared