Effect of pirfenidone on the pulmonary fibrosis of Hermansky–Pudlak syndrome
WA Gahl, M Brantly, J Troendle, NA Avila… - Molecular genetics and …, 2002 - Elsevier
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 (800mg,
tid), 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 change in pulmonary function values as …
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 (800mg,
tid), 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 change in pulmonary function values as …
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