Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
J Card Fail. 2011 Nov;17(11):930-6. doi: 10.1016/j.cardfail.2011.07.011. Epub 2011 Sep 9.
Enzyme replacement therapy (ERT) for infantile-onset Pompe disease effectively reduces the left ventricular (LV) mass. This study sought to explore detailed process of LV reverse remodeling after ERT with the use of tissue Doppler and stain rate imaging.
Nine infants and children with Pompe cardiomyopathy undergoing ERT for ≥1 year, as well as 36 healthy control subjects, were studied. Global systolic and diastolic function was evaluated by peak systolic and early-diastolic velocity at mitral annulus. Temporal systolic and diastolic dyssynchrony was evaluated by the coefficient of variation of the time from the QRS complex to peak systolic and early-diastolic strain rate among 12 LV segments. All pre-ERT patients had impaired global systolic and diastolic function as well as increased regional dyssynchrony (P < .001 for each of all). During the regression of LV hypertrophy, all of these functional indices improved (P for trend <.001), with temporal diastolic dyssynchrony being a significant factor linking to LV mass index in multivariate analysis (P < .001).
ERT improved global LV function and dyssynchrony in Pompe patients. The relationship between LV mass and temporal diastolic dyssynchrony during reverse remodeling suggested a pathophysiologic role of dyssynchrony in Pompe cardiomyopathy.
婴儿期起病的庞贝病的酶替代疗法（ERT）可有效降低左心室（LV）质量。本研究旨在利用组织多普勒和应变率成像技术探讨 ERT 后 LV 逆重构的详细过程。
研究了 9 名接受 ERT 治疗≥1 年的庞贝心肌病婴儿和儿童，以及 36 名健康对照者。通过二尖瓣环的收缩期峰值速度和舒张早期速度评估整体收缩和舒张功能。通过 12 个 LV 节段的 QRS 综合波到收缩期峰值和舒张早期应变率的时间变化的变异系数评估收缩和舒张的时间不同步。所有 ERT 前患者均存在整体收缩和舒张功能受损以及区域不同步增加（所有均<.001）。在 LV 肥大的逆重构过程中，所有这些功能指标均得到改善（趋势 P<.001），在多变量分析中，时间性舒张不同步是与 LV 质量指数相关的重要因素（P<.001）。
ERT 改善了庞贝病患者的整体 LV 功能和不同步性。在逆重构过程中 LV 质量与时间性舒张不同步之间的关系表明，不同步在庞贝心肌病中具有病理生理作用。
