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

Yamada, Kenji; Shiraishi, Hideaki; Oki, Eishin et al.

Molecular genetics and metabolism reports, 2018, 15, 55-63

Permalink : https://hdl.handle.net/2115/71201

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アクセス権
open access
URI
https://hdl.handle.net/2115/71201
タイトル
Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan
著者
Yamada, Kenji ; Shiraishi, Hideaki ; Oki, Eishin ; Ishige, Mika ; Fukao, Toshiyuki ; Hamada, Yusuke ; Sakai, Norio ; Ochi, Fumihiro ; Watanabe, Asami ; Kawakami, Sanae ; Kuzume, Kazuyo ; Watanabe, Kenji ; Sameshima, Koji ; Nakamagoe, Kiyotaka ; Tamaoka, Akira ; Asahina, Naoko ; Yokoshiki, Saki ; Miyakoshi, Takashi ; Ono, Kota ; Oba, Koji ; Isoe, Toshiyuki ; Hayashi, Hiroshi ; Yamaguchi, Seiji ; Sato, Norihiro
著者名
Yamada, Kenji
e-Rad_Researcher
80374411
著者名
Shiraishi, Hideaki
著者名
Oki, Eishin
著者名
Ishige, Mika
著者名
Fukao, Toshiyuki
著者名
Hamada, Yusuke
著者名
Sakai, Norio
著者名
Ochi, Fumihiro
著者名
Watanabe, Asami
著者名
Kawakami, Sanae
著者名
Kuzume, Kazuyo
著者名
Watanabe, Kenji
著者名
Sameshima, Koji
著者名
Nakamagoe, Kiyotaka
著者名
Tamaoka, Akira
著者名
Asahina, Naoko
著者名
Yokoshiki, Saki
著者名
Miyakoshi, Takashi
著者名
Ono, Kota
e-Rad_Researcher
30422926
著者名
Oba, Koji
著者名
Isoe, Toshiyuki
著者名
Hayashi, Hiroshi
著者名
Yamaguchi, Seiji
著者名
Sato, Norihiro
言語
英語
キーワード
Bezafibrate
Fatty acid oxidation disorders (FAODs)
Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency
Carnitine palmitoyltransferase-II (CPT-2) deficiency
Clinical trial
発行日
2018-06
出版者
Elsevier
収録物名
Molecular genetics and metabolism reports
巻(号)
15
ページ
55-63
抄録
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.
資源タイプ
学術雑誌論文
出版タイプ
Version of Record
権利情報
Creative Commons Attribution 4.0 International
PISSN
2214-4269
関連情報 (isIdenticalTo)
出版社版DOI
https://doi.org/10.1016/j.ymgmr.2018.02.003