title: Left ventricular geometry, global function, and dyssynchrony in infants and children with pompe cardiomyopathy undergoing enzyme replacement therapy.
abstract: BACKGROUND
  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.
  
  
  RESULTS
  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).
  
  
  CONCLUSIONS
  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.
authors: Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
authors: Chun-An
authors: CA
authors: Chen
authors: Yin-Hsiu
authors: YH
authors: Chien
authors: Wuh-Liang
authors: WL
authors: Hwu
authors: Ni-Chung
authors: NC
authors: Lee
authors: Jou-Kou
authors: JK
authors: Wang
authors: Lei-Ru
authors: LR
authors: Chen
authors: Chun-Wei
authors: CW
authors: Lu
authors: Ming-Tai
authors: MT
authors: Lin
authors: Shuenn-Nan
authors: SN
authors: Chiu
authors: Hsin-Hui
authors: HH
authors: Chiu
authors: Mei-Hwan
authors: MH
authors: Wu
mesh_terms: D000319:Adrenergic beta-Antagonists / Q000627:therapeutic use; D000367:Age Factors; D001145:Arrhythmias, Cardiac / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy*; D002312:Cardiomyopathy, Hypertrophic / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy*; D016022:Case-Control Studies; D002675:Child, Preschool; D003971:Diastole; D018450:Disease Progression; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D006009:Glycogen Storage Disease Type II / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy*; D006801:Humans; D017379:Hypertrophy, Left Ventricular / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D015999:Multivariate Analysis; D011446:Prospective Studies; D018709:Statistics, Nonparametric; D013599:Systole; D013997:Time Factors; D018608:Ultrasonography, Doppler
chemical_list: D000319:Adrenergic beta-Antagonists