期刊
INTERNATIONAL JOURNAL OF CARDIOLOGY
卷 269, 期 -, 页码 104-110出版社
ELSEVIER IRELAND LTD
DOI: 10.1016/j.ijcard.2018.07.091
关键词
Classic-infantile Pompe disease; Acid alpha-glucosidase; Enzyme replacement therapy (ERT); Hypertrophic cardiomyopathy; Tachyarrhythmia
资金
- Prinses Beatrix Spierfonds [W.OR13-21, W.OR15-10, W.OR16-07]
- TKI-Health Holland [LSHM16008]
- Tex Net
- Sophia Foundation for Medical Research (SSWO) [S17-32]
- Metakids [2016-063]
- Conselho Nacional de Desenvolvimento Cientifico e Tecnologico - National Counsil of Technological and Scientific Development, Brasil
- Colciencias
- Genzyme Corp.
Background: Cardiac failure is the main cause of death in untreated classic infantile Pompe disease, an inheritable metabolic myopathy characterized by progressive hypertrophic cardiomyopathy. Since the introduction of enzyme replacement therapy (ERT), survival has increased significantly due to reduced cardiac hypertrophy and improved cardiac function. However, little is known about ERT's long-term effects on the heart. Methods: Fourteen patients were included in this prospective study. Cardiac dimensions, function, conduction and rhythm disturbances were evaluated at baseline and at regular intervals thereafter. Results: Treatment duration ranged from 1.1 to 13.9 years (median 4.8 years). At baseline, all patients had increased left ventricular mass index (LVMI) (median LVMI 226 g/m(2), range 98 to 599 g/m(2), Z-score median 7, range 2.4-12.4). During the first four weeks, LVMI continued to increase in six patients. Normalization of LVMI was observed in 13 patients (median 30 weeks; range 3 to 660 weeks). After clinical deterioration, LVMI increased again slightly in one patient. At baseline, PR interval was shortened in all patients; it normalized in only three. A delta-wave pattern on ECG was seen in six patients and resulted in documented periods of supraventricular tachycardias (SVTs) in three patients, two of whom required medication and/or ablation. One patient had severe bradycardia (35 beats/min). Conclusion: This study shows that ERT significantly reduced LVMI, and sustained this effect over a period of 13.9 years. The risk for rhythm disturbances remains. Regular cardiac evaluations should be continued, also after initially good response to ERT. (c) 2018 The Authors. Published by Elsevier B.V.
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