Huang Meirong, Zhang Xu, Chen Shubao, Sun Yuping, Xiao Yanyan, Sun Jinghui, Huang Min, Chen Sun, Liu Fang
Department of Pediatric Cardiology, Shanghai Children's Medical Center, Shanghai Jiao Tong University, 1678, Dongfang Road, Shanghai, 200127, People's Republic of China.
Pediatr Cardiol. 2013 Mar;34(3):680-5. doi: 10.1007/s00246-012-0527-x. Epub 2012 Oct 30.
This study aimed to evaluate the clinical efficacy and safety of carvedilol for pediatric patients with chronic heart failure caused by dilated cardiomyopathy. Seven pediatric medical centers participated in this prospective study. Pediatric patients (n = 89) were randomly divided into an experimental group (carvedilol treatment) and a control group (conventional treatment). The analysis excluded 12 patients lost during the follow-up period. Carvedilol was added to the therapy of the experimental group after at least 1 month of basic treatment with digoxin, an angiotensin-converting-enzyme inhibitor, and diuretics. The control patients received the same basic treatment but did not receive carvedilol. The initial dose of carvedilol was 0.1 (mg/kg day), and the dose was doubled every 2 weeks until the maximum tolerated dose or 0.8 (mg/kg day) was achieved. The tolerated dose was maintained for 6 months. The Ross scales and echocardiographic parameters including left ventricular diastolic diameter, left ventricular systolic diameter, left ventricular ejection fraction, left ventricular fractional shortening, and serous brain natriuretic peptide (BNP) concentration, as well as clinical progress were compared between the two groups. The Ross scales decreased by 11.94% in the experimental group, which was more than in the control group (2.81%). In addition, changes to other echocardiographic parameters in the experimental group also were superior to those in the control group. The serous BNP concentration in the experimental group decreased by 30.1%, which also was more than the decrease (22.2%) observed in the control group. Clinical improvement was demonstrated by 40%, no change by 35%, and clinical deterioration by 25% of the patients in the experimental group, and by respectively 37.8, 27, and 35.2% of the patients in the control group. These differences were not statistically significant. Only one patient demonstrated a severe adverse event, severe pulmonary infection, and complete atrioventricular block. None of the other carvedilol-treated patients experienced drug-related side effects. Carvedilol can be safely used for treatment of chronic heart dysfunction in pediatric patients with dilated cardiomyopathy. The results in this study showed an apparent improvement in the cardiac function of these patients. Additional clinical studies are required to determine the most favorable dosing levels and regimens of carvedilol before its safety and efficacy for the pediatric population can be determined conclusively.
本研究旨在评估卡维地洛治疗扩张型心肌病所致小儿慢性心力衰竭的临床疗效及安全性。7家儿科医疗中心参与了这项前瞻性研究。小儿患者（n = 89）被随机分为实验组（卡维地洛治疗）和对照组（常规治疗）。分析排除了12例随访期间失访的患者。实验组在使用地高辛、血管紧张素转换酶抑制剂和利尿剂进行至少1个月的基础治疗后加用卡维地洛。对照患者接受相同的基础治疗，但未接受卡维地洛。卡维地洛的初始剂量为0.1（毫克/千克·天），每2周剂量加倍，直至达到最大耐受剂量或0.8（毫克/千克·天）。将耐受剂量维持6个月。比较两组的罗斯量表以及超声心动图参数（包括左心室舒张直径、左心室收缩直径、左心室射血分数、左心室缩短分数和血清脑钠肽（BNP）浓度），以及临床进展情况。实验组罗斯量表下降了11.94%，高于对照组（2.81%）。此外，实验组其他超声心动图参数的变化也优于对照组。实验组血清BNP浓度下降了30.1%，也高于对照组观察到的下降幅度（22.2%）。实验组40% 的患者临床改善，35% 无变化，25% 临床恶化；对照组分别为37.8%、27% 和35.2%。这些差异无统计学意义。仅1例患者出现严重不良事件，即严重肺部感染和完全性房室传导阻滞。其他接受卡维地洛治疗的患者均未出现与药物相关的副作用。卡维地洛可安全用于治疗扩张型心肌病小儿患者的慢性心脏功能不全。本研究结果显示这些患者的心脏功能有明显改善。在最终确定卡维地洛对小儿群体的安全性和疗效之前，还需要进行更多临床研究以确定最适宜的给药剂量和方案。
