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Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan ; 2nd report QOL survey

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Title: Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan ; 2nd report QOL survey
Authors: Shiraishi, Hideaki Browse this author →KAKEN DB
Yamada, Kenji Browse this author
Oki, Eishin Browse this author
Ishige, Mika Browse this author →KAKEN DB
Fukao, Toshiyuki Browse this author →KAKEN DB
Hamada, Yusuke Browse this author
Sakai, Norio Browse this author
Ochi, Fumihiro Browse this author →KAKEN DB
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 →KAKEN DB
Tamaoka, Akira Browse this author →KAKEN DB
Asahina, Naoko Browse this author
Yokoshiki, Saki Browse this author
Miyakoshi, Takashi Browse this author
Oba, Koji Browse this author →KAKEN DB
Isoe, Toshiyuki Browse this author
Hayashi, Hiroshi Browse this author →KAKEN DB
Yamaguchi, Seiji Browse this author →KAKEN DB
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
Quality of life
Issue Date: Sep-2019
Publisher: Elsevier
Journal Title: Molecular genetics and metabolism reports
Volume: 20
Start Page: 100496
Publisher DOI: 10.1016/j.ymgmr.2019.100496
Abstract: Introduction: Fatty acid oxidation disorders (FAODs) are rare diseases caused by a defective mitochondrial fatty acid oxidation (FAO) enzyme. We recently reported that bezafibrate improved patient quality of life (QOL) based on the SF 36 questionnaire score in patients with FAODs during a 50-week, open-label, clinical trial. Herein we conducted further survey assessments of the trial patients to define the long-term efficacy and safety of bezafibrate. Materials and methods: This trial was an open-label, non-randomized, and multicenter study of bezafibrate treatment in five patients with very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency and one patient with carnitine palmitoyltransferase-II (CPT-2) deficiency (median age, 15.9 years; range, 5.8-26.4 years). The bezafibrate administration was continued for a further 102 174 weeks after the 24-week treatment described in our previous study. QOL was quantitated using the 36-Item Short Form Health Survey (SF 36) questionnaire, which constitutes eight components: physical functioning (PF), role limitation due to physical problems, bodily pain, general health perception, vitality, social functioning, role limitation due to emotional problems, and mental health. Results: PF was elevated in all patients and continued to rise during the study, with the total QOL scores increased from baseline in five of the six cases. In particular, three patients older than 20 years showed treatment efficacy, and all subcategories of QOL were elevated in two of these cases. Conclusion: Our findings supported one of the stated benefits of bezafibrate in improving QOL for patients with FAODs.
Type: article
Appears in Collections:国際連携研究教育局 : GI-CoRE (Global Institution for Collaborative Research and Education : GI-CoRE) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)
北海道大学病院 (Hokkaido University Hospital) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)

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