https://scholars.lib.ntu.edu.tw/handle/123456789/525182
標題: | Reversal of Cardiac Dysfunction after Enzyme Replacement in Patients with Infantile-Onset Pompe Disease | 作者: | Chen L.-R. CHUN-AN CHEN SHUENN-NAN CHIU YIN-HSIU CHIEN NI-CHUNG LEE MING-TAI LIN WUH-LIANG HWU JOU-KOU WANG MEI-HWAN WU |
公開日期: | 2009 | 出版社: | Mosby Inc. | 卷: | 155 | 期: | 2 | 起(迄)頁: | 271-275 | 來源出版物: | Journal of Pediatrics | 摘要: | Objective: To compare the effects of enzyme replacement therapy (ERT) on cardiac performance in symptomatic and symptom-free infants with Pompe disease. Study design: Patients diagnosed between 1983 and 2008 were identified. Before the initiation of ERT, systolic dysfunction appeared only in patients ?5 months; thus we used this cut-point in age to divide clinically symptomatic patients into early and late treatment groups (Clin-E and Clin-L). Newborn screening (NBS) identified symptom-free patients. Results: Among a total of 40 patients, 14 received ERT: 5 in the Clin-L, 4 in the Clin-E, and 5 in the NBS groups. All patients showed cardiomegaly, hypertrophic myocardium, and elevated B-type natriuretic peptide (measured in the Clin-E and NBS groups). ERT improved the survival and outcomes. Regressed myocardial hypertrophy and lowered B-type natriuretic peptide level occurred after 1 to 6 months of ERT. Nonetheless, there were 2 deaths and 2 survivors requiring ventilator support in the Clin-L group. Despite the regressed QRS voltage and shortened QT dispersion, life-threatening arrhythmias were still observed in 3, but none in the NBS group. Conclusion: ERT may restore the cardiac function in both symptomatic and symptom-free patients, but the beneficial effect may be unpredictable if given after the age of 5 months. ? 2009 Mosby, Inc. All rights reserved. |
URI: | https://www.scopus.com/inward/record.uri?eid=2-s2.0-67651113834&doi=10.1016%2fj.jpeds.2009.03.015&partnerID=40&md5=01974c0f6a8d07269b3d2bfb99afb940 https://scholars.lib.ntu.edu.tw/handle/123456789/525182 |
ISSN: | 0022-3476 | DOI: | 10.1016/j.jpeds.2009.03.015 | SDG/關鍵字: | adenosine; beta adrenergic receptor blocking agent; brain natriuretic peptide; dipeptidyl carboxypeptidase inhibitor; diuretic agent; propranolol; recombinant glucan 1,4 alpha glucosidase; article; artificial ventilation; cardiomegaly; clinical article; clinical feature; controlled study; enzyme replacement; glycogen storage disease type 2; health status; heart arrhythmia; heart failure; heart performance; heart ventricle hypertrophy; human; infant; mortality; newborn screening; onset age; priority journal; protein blood level; QRS complex; QT dispersion; survival rate; systolic dysfunction; tachycardia |
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。