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

التفاصيل البيبلوغرافية
العنوان: Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan; 2nd report QOL survey
المؤلفون: Hideaki Shiraishi, Kenji Yamada, Eishin Oki, Mika Ishige, Toshiyuki Fukao, Yusuke Hamada, Norio Sakai, Fumihiro Ochi, Asami Watanabe, Sanae Kawakami, Kazuyo Kuzume, Kenji Watanabe, Koji Sameshima, Kiyotaka Nakamagoe, Akira Tamaoka, Naoko Asahina, Saki Yokoshiki, Takashi Miyakoshi, Koji Oba, Toshiyuki Isoe, Hiroshi Hayashi, Seiji Yamaguchi, Norihiro Sato
المصدر: Molecular Genetics and Metabolism Reports, Vol 20, Iss , Pp - (2019)
بيانات النشر: Elsevier, 2019.
سنة النشر: 2019
المجموعة: LCC:Medicine (General)
LCC:Biology (General)
مصطلحات موضوعية: Medicine (General), R5-920, Biology (General), QH301-705.5
الوصف: 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. 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
نوع الوثيقة: article
وصف الملف: electronic resource
اللغة: English
تدمد: 2214-4269
Relation: http://www.sciencedirect.com/science/article/pii/S2214426919300953; https://doaj.org/toc/2214-4269
DOI: 10.1016/j.ymgmr.2019.100496
URL الوصول: https://doaj.org/article/485479e07814483c920b57c0dc96b489
رقم الأكسشن: edsdoj.485479e07814483c920b57c0dc96b489
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:22144269
DOI:10.1016/j.ymgmr.2019.100496