Prolonged treatment with open-label pirfenidone in Hermansky-Pudlak syndrome pulmonary fibrosis


O'Brien K. J. , Introne W. J. , Akal O., AKAL T., Barbu A., McGowan M. P. , ...More

MOLECULAR GENETICS AND METABOLISM, vol.125, pp.168-173, 2018 (Peer-Reviewed Journal) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 125
  • Publication Date: 2018
  • Doi Number: 10.1016/j.ymgme.2018.07.012
  • Journal Name: MOLECULAR GENETICS AND METABOLISM
  • Journal Indexes: Science Citation Index Expanded, Scopus
  • Page Numbers: pp.168-173
  • Keywords: Hermansky-Pudlak syndrome, pirfenidone, pulmonary fibrosis, INTERSTITIAL LUNG-DISEASE, SYNDROME TYPE-2, MUTATIONS, GENE, FORM, CT, IMMUNODEFICIENCY, ORGANELLES, ADAPTER

Abstract

Purpose: Limited information is available regarding chronic treatment with pirfenidone, an anti-fibrotic drug. Effects of long-term open-label pirfenidone were evaluated in a small cohort with Hermansky-Pudlak syndrome (HPS), a rare autosomal recessive disorder with highly penetrant pulmonary fibrosis.