Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
Status:
Unknown status
Trial end date:
2018-06-01
Target enrollment:
Participant gender:
Summary
Interstitial lung disease (ILD) is a common complication of dermatomyositis (DM) with
prevalence up to 65%, and is considered to be one of the determining factors of prognosis.
Clinical amyopathic dermatomyositis (CADM), which is a special phenotype of DM, with
characteristic cutaneous manifestations but no or only subclinical myopathy. Many studies,
mainly from Asia, including ours, have demonstrated that these patients with CADM tend to
develop a rapidly progressive ILD (RPILD) and have a poor response to conventional therapy,
such as high-dose corticosteroids and immunosuppressants, leading to lethal outcome with a
6-month survival rate of less than 50%.
Pirfenidone, a new oral antifibrotic agent, has been approved for the treatment of idiopathic
pulmonary fibrosis (IPF). Randomized controlled trials of pirfenidone in patients with IPF
suggested that it could ameliorate pulmonary function decline and improve the
progression-free survival. Its utility in connective tissue disease (CTD) related ILD has
been implicated, but no evidence has yet demonstrated its efficacy. Therefore, the
investigators conduct this study to evaluate the possible therapeutic effects of pirfenidone
on RPILD associated with CADM.