イシガキ　ケイコ   ISHIGAKI Keiko   石垣　景子    所属   医学部 医学科（東京女子医科大学病院）    職種   准教授
論文種別	原著
言語種別	英語
査読の有無	査読なし
表題	Urinary prostaglandin D2 and E2 metabolites are elevated with disease severity in patients with Fukuyama congenital muscular dystrophy.
掲載誌名	正式名：Scientific Reports ISSNコード：20452322
掲載区分	国外
巻・号・頁	26(1),pp.6873
著者・共著者	ISHIGAKI Keiko†, TAKEUCHI Atsuko, TANIGUCHI-IKEDA Mariko, SATOU Takatoshi, MURAKAMI Terumi, SHICHIJI Minobu, ISHIGURO Kumiko, KIHARA Yuuki, NAGATA Satoru, URADE Yoshihiro
担当区分	筆頭著者,責任著者
発行年月	2025/02
概要	Treatment approaches are lacking for Fukuyama congenital muscular dystrophy (FCMD), the second most common type of pediatric muscular dystrophy after Duchenne muscular dystrophy (DMD) in the Japanese population. Recent studies demonstrating the involvement of prostaglandin (PG) D2 in DMD progression has led to the development of novel inhibitors targeting hematopoietic PGD2 synthase. This study aimed to determine the role of PGD2 in FCMD etiology in 42 patients with FCMD and 77 healthy age-matched individuals. Concentrations of tetranor-PGDM and tetranor-PGEM, the metabolites of PGD2 and PGE2, respectively, and creatinine were measured in spot urine samples. Mean tetranor-PGDM/creatinine and tetranor-PGEM/creatinine concentrations and tetranor-PGEM/tetranor-PGDM ratio were significantly higher in patients with FCMD than the healthy controls (5.3 ± 2.1 and 30.9 ± 52.3 ng/mg creatinine and 6.8 ± 2.0 vs. 3.4 ± 0.3 and 9.5 ± 0.9 ng/mg creatinine and 3.2 ± 0.3, respectively; p = 0.0011, p < 0.0001 and p < 0.0001, respectively). These metabolites were increased in patients with typical and severe FCMD phenotypes than in those with the mild FCMD phenotype, indicating their correlation with disease severity. These results implicate that PGD2 and PGE2 play important roles in FCMD pathogenesis and that novel hematopoietic PGD2 synthase inhibitors and steroids used in DMD may also have therapeutic utility in FCMD.
DOI	10.1038/s41598-025-91539-2
PMID	40011677