The Efficacy and Safety of JAK Inhibitor in the Treatment of Anti-MDA5 Antibody-positive Dermatomyositis Patients
Status:
Recruiting
Trial end date:
2021-12-30
Target enrollment:
Participant gender:
Summary
Anti-melanoma differentiation-associated gene5 (Anti-MDA5) antibody positive Dermatomyositis
(DM) is a subtype of DM that is more frequent in East Asia, which is often exhibit skin
lesion, clinically amyopathic and interstitial lung disease. About 42%-100% of patients with
Anti-MDA5+ DM develop rapidly progressive interstitial lung disease (RPILD) and result in
respiratory failure. The mortality is as high as 40% within 6 months. In addition, not every
patient with Anti-MDA5+ DM respond to traditional treatment strategy and most of the patients
are resistant to immunosuppressive therapy including a combination of high dose
glucocorticoids (GCs) and immunosuppressants such as cyclosporine, tacrolimus, or
cyclophosphamide. However, RP-ILD is still the main cause of death due to fatal respiratory
failure. Therefore, treatment of Anti-MDA5+ DM patients is challenging.Blocking multiple
cytokines may become a new target for the treatment of this disease.Jakinibs is a Janus
kinase (JAK) inhibitor that blocks a variety of cytokines, such as type I and type II
interferon. Few studies have reported a positive response to JAK inhibitor for Anti-MDA5+ DM.
Kazuhiro et al. reported in 2018 that JAK inhibitor tofacitinib may be an effective treatment
option for high risk amyopathic dermatomyositis (ADM) -ILD patients after failure of
conventional treatment, but the number of cases is too small. And a recent paper showed that
great efficacy of tofacitinib for the improvement of survival of anti-MDA5-positive
early-stage ADM-ILD patients.The aim of this study is to evaluate the efficacy and safety of
JAK inhibitors in the treatment of anti-MDA5+ DM patients, and to evaluate the effect of JAK
inhibitors on B cells of these patients, so as to provide a new target and theoretical basis
for the treatment of anti-MDA5+ DM.
Phase:
Phase 4
Details
Lead Sponsor:
First Affiliated Hospital Xi'an Jiaotong University