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An autopsy case of Hermansky-Pudlak syndrome: a case report and review of the literature on treatment.

Authors :
Harada T
Ishimatsu Y
Nakashima S
Miura S
Tomonaga M
Kakugawa T
Hara S
Sakamoto N
Yoshii C
Mukae H
Kawabata Y
Kohno S
Source :
Internal medicine (Tokyo, Japan) [Intern Med] 2014; Vol. 53 (23), pp. 2705-9. Date of Electronic Publication: 2014 Dec 01.
Publication Year :
2014

Abstract

Hermansky-Pudlak syndrome (HPS) is a rare genetic disorder, the most common complication of which influencing the prognosis is pulmonary fibrosis. In the present report, we describe an autopsy case of a Japanese woman with HPS. The patient was diagnosed at 50 years of age based on the presence of oculocutaneous albinism, hemorrhagic diathesis, ceroid-lipofuscin accumulation and pulmonary fibrosis. Although systemic steroids, immunosuppressants and pirfenidone were administered for pulmonary involvement, she died from respiratory failure two years later. Obtaining an early diagnosis and taking into consideration the need for lung transplantation is necessary in order to improve the prognosis of HPS. We herein report this very rare Japanese case of HPS with a review of the treatment approaches for HPS complicated with pulmonary fibrosis.

Details

Language :
English
ISSN :
1349-7235
Volume :
53
Issue :
23
Database :
MEDLINE
Journal :
Internal medicine (Tokyo, Japan)
Publication Type :
Academic Journal
Accession number :
25447654
Full Text :
https://doi.org/10.2169/internalmedicine.53.2239