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

O'Brien, Kevin; Introne, Wendy; Akal, Orhan; AKAL, TÜLAY; Barbu, Adrian; McGowan, Melissa; Merideth, Melissa; Seward, Samuel; Gahl, William; Gochuico, Bernadette

doi:10.1016/j.ymgme.2018.07.012

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 (SCI-Expanded, Scopus)

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 (SCI-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
Middle East Technical University Affiliated: Yes

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.