Effect of pirfenidone on the pulmonary fibrosis of Hermansky-Pudlak syndrome.

@article{Gahl2002EffectOP,
  title={Effect of pirfenidone on the pulmonary fibrosis of Hermansky-Pudlak syndrome.},
  author={William A Gahl and Mark L. Brantly and James Troendle and Nilo A. Avila and Antonio Padua and Carlos Montalvo and Hilda Cardona and Karim Anton Calis and Bernadette Gochuico},
  journal={Molecular genetics and metabolism},
  year={2002},
  volume={76 3},
  pages={234-42}
}
Hermansky-Pudlak syndrome (HPS) consists of oculocutaneous albinism, a platelet storage pool deficiency and, in patients with HPS1 gene mutations, a progressive, fatal pulmonary fibrosis. We investigated the safety and efficacy of an antifibrotic agent, pirfenidone (800 mg, t.i.d.), in treating 21 adult Puerto Rican HPS patients, including 20 homozygous for the same HPS1 mutation. Patients were examined every 4 months for up to 44 months in a randomized, placebo-controlled trial, with rate of… CONTINUE READING