{Reference Type}: Case Reports
{Title}: Long-term nintedanib treatment for progressive pulmonary fibrosis associated with Hermansky-Pudlak syndrome type 1 followed by lung transplantation.
{Author}: Itoh T;Kawasaki T;Kaiho T;Shikano K;Naito A;Abe M;Suzuki H;Ota M;Yoshino I;Suzuki T;
{Journal}: Respir Investig
{Volume}: 62
{Issue}: 1
{Year}: 2024 Jan 27
暂无{DOI}: 10.1016/j.resinv.2023.12.007
{Abstract}: Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disease that often causes progressive pulmonary fibrosis (HPS-PPF) in some genetic types with high mortality rates. No effective treatment for HPS-PPF other than lung transplantation has been established. Herein, we report a case of HPS type 1 with progressive pulmonary fibrosis treated with long-term nintedanib administration followed by lung transplantation. The resected lungs revealed diffuse interstitial lung lesions, including fibroblastic foci, suggesting the potential beneficial effects of anti-fibrotic drugs in HPS-PPF. Together with previous reports, the present case suggests that nintedanib might be a safe and effective drug for HPS-PPF.