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Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan

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Title: Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan
Authors: Yamada, Kenji Browse this author
Shiraishi, Hideaki Browse this author →KAKEN DB
Oki, Eishin Browse this author
Ishige, Mika Browse this author
Fukao, Toshiyuki Browse this author
Hamada, Yusuke Browse this author
Sakai, Norio Browse this author
Ochi, Fumihiro Browse this author
Watanabe, Asami Browse this author
Kawakami, Sanae Browse this author
Kuzume, Kazuyo Browse this author
Watanabe, Kenji Browse this author
Sameshima, Koji Browse this author
Nakamagoe, Kiyotaka Browse this author
Tamaoka, Akira Browse this author
Asahina, Naoko Browse this author
Yokoshiki, Saki Browse this author
Miyakoshi, Takashi Browse this author
Ono, Kota Browse this author
Oba, Koji Browse this author →KAKEN DB
Isoe, Toshiyuki Browse this author
Hayashi, Hiroshi Browse this author
Yamaguchi, Seiji Browse this author
Sato, Norihiro Browse this author
Keywords: Bezafibrate
Fatty acid oxidation disorders (FAODs)
Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency
Carnitine palmitoyltransferase-II (CPT-2) deficiency
Clinical trial
Issue Date: Jun-2018
Publisher: Elsevier
Journal Title: Molecular genetics and metabolism reports
Volume: 15
Start Page: 55
End Page: 63
Publisher DOI: 10.1016/j.ymgmr.2018.02.003
Abstract: Introduction: Fatty acid oxidation disorders (FAODs) are rare diseases caused by defects in mitochondrial fatty acid oxidation (FAO) enzymes. While the efficacy of bezafibrate, a peroxisome proliferator-activated receptor agonist, on the in vitro FAO capacity has been reported, the in vivo efficacy remains controversial. Therefore, we conducted a clinical trial of bezafibrate in Japanese patients with FAODs. Materials and methods: This trial was an open-label, non-randomized, and multicenter study of bezafibrate treatment in 6 patients with very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency and 2 patients with carnitine palmitoyltransferase-II (CPT-2) deficiency (median age, 8.2 years; ranging from 5.8 to 26.4 years). Bezafibrate was administered for 6 months following a 6-month observation period. The primary endpoint was the frequency of myopathic attacks, and the secondary endpoints were serum acylcarnitines (ACs, C14:1 or C16 + C18:1), creatine kinase (CK) levels, degree of muscle pain (VAS; visual analog scale) during myopathic attacks, and quality of life (QOL; evaluated using validated questionnaires). Results: The frequency of myopathic attacks after bezafibrate administration decreased in 3 patients, increased in 3, and did not change in 2. The CK, AC, and VAS values during attacks could be estimated in only three or four patients, but a half of the patients did not experience attacks before or after treatment. Changes in CK, AC, and VAS values varied across individuals. In contrast, three components of QOL, namely, physical functioning, role limitation due to physical problems (role physical), and social functioning, were significantly elevated. No adverse drug reactions were observed. Conclusion: In this study, the frequency of myopathic attacks and CK, AC, and VAS values during the attacks could not be evaluated due to several limitations, such as a small trial population. Our findings indicate that bezafibrate improves the QOL of patients with FAODs, but its efficacy must be examined in future investigations.
Type: article
Appears in Collections:北海道大学病院 (Hokkaido University Hospital) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)

Submitter: 佐藤 典宏

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