| 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. |
| Rights: | https://creativecommons.org/licenses/by/4.0/ |
| Type: | article |
| URI: | http://hdl.handle.net/2115/76033 |
| 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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