https://scholars.lib.ntu.edu.tw/handle/123456789/525845
標題: | Cardiac structure and function and effects of enzyme replacement therapy in patients with mucopolysaccharidoses I, II, IVA and VI | 作者: | Lin, Hsiang-Yu Chuang, Chih-Kuang Chen, Ming-Ren Lin, Shan-Miao Hung, Chung-Lieh Chang, Chia-Ying Chiu, Pao Chin Tsai, Wen-Hui Niu, Dau-Ming Tsai, Fuu-Jen Lin, Shio Jean WUH-LIANG HWU Lin, Ju-Li SHAU-PING LIN |
公開日期: | 2016 | 出版社: | Academic Press Inc. | 卷: | 117 | 期: | 4 | 起(迄)頁: | 431-437 | 來源出版物: | Molecular Genetics and Metabolism | 摘要: | Background While enzyme replacement therapy (ERT) has been shown to improve endurance and joint mobility for patients with mucopolysaccharidoses (MPS) I, II, IVA and VI, the impact of ERT on cardiac abnormalities remains uncertain. Methods Medical records and echocardiograms of 28 Taiwanese MPS patients (9 with MPS I, 7 with MPS II, 7 with MPS IVA, and 5 with MPS VI) treated with ERT for 1–10.8?years were retrospectively reviewed. Results At start of ERT, z scores?>?2 were identified in 46% and 75% for left ventricular mass index (LVMI) and interventricular septum thickness in diastole (IVSd) in these patients, respectively. Twenty-four patients (86%) had valvular heart disease. After ERT, the mean IVSd z score of all patients decreased significantly from 3.87 to 2.57 (p?=?0.016). For 11 patients starting ERT before 12?years of age, z scores for both LVMI and IVSd decreased significantly (p?0.01) after ERT. However, the condition of valve regurgitation or stenosis did not show improvement despite ERT. Conclusions ERT was shown to be an effective therapy for reducing cardiac hypertrophy, with best results seen when ERT was started at an early age. ERT, however, had little impact on valvular heart disease. ? 2016 Elsevier Inc. |
URI: | https://www.scopus.com/inward/record.uri?eid=2-s2.0-84958580864&doi=10.1016%2fj.ymgme.2016.02.003&partnerID=40&md5=45f46b94475abd159dca066d5b6a9a26 https://scholars.lib.ntu.edu.tw/handle/123456789/525845 |
ISSN: | 1096-7192 | DOI: | 10.1016/j.ymgme.2016.02.003 | SDG/關鍵字: | elosulfase alfa; galsulfase; iduronate 2 sulfatase; laronidase; adolescent; adult; aorta stenosis; aorta valve regurgitation; Article; bi atrial enlargement; cardiac structure; cardiomegaly; cardiovascular parameters; child; clinical article; diastolic dysfunction; ECG abnormality; enzyme replacement; female; first degree atrioventricular block; heart; heart atrium enlargement; heart murmur; heart right ventricle hypertrophy; heart ventricle extrasystole; heart ventricle hypertrophy; human; infant; interventricular septum thickness in diastole; left ventricular mass index; male; Maroteaux Lamy syndrome; mitral valve prolapse; mitral valve regurgitation; mitral valve stenosis; mucopolysaccharide i; mucopolysaccharide i; mucopolysaccharide ii; mucopolysaccharide ii; mucopolysaccharide iva; mucopolysaccharide iva; mucopolysaccharidosis; priority journal; retrospective study; Taiwanese; valvular heart disease; cardiac muscle; complication; echocardiography; electrocardiography; follow up; heart; Heart Diseases; Mucopolysaccharidoses; pathology; pathophysiology; preschool child; treatment outcome; young adult; Adolescent; Adult; Child; Child, Preschool; Echocardiography; Electrocardiography; Enzyme Replacement Therapy; Female; Follow-Up Studies; Heart; Heart Diseases; Humans; Infant; Male; Mucopolysaccharidoses; Myocardium; Retrospective Studies; Treatment Outcome; Young Adult |
顯示於: | 醫學系 |
在 IR 系統中的文件,除了特別指名其著作權條款之外,均受到著作權保護,並且保留所有的權利。