Longitudinal Follow-Up of Children With HLHS and Association Between Norwood Shunt Type and Long-Term Outcomes: The SVR III Study.
Longitudinal Follow-Up of Children With HLHS and Association Between Norwood Shunt Type and Long-Term Outcomes: The SVR III Study.
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DOI：
10.1161/circulationaha.123.065192
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发表时间：
2023-10-24
期刊：
影响因子：
37.8
通讯作者：
Newburger, Jane W.
中科院分区：
文献类型：
--
作者：
Goldberg, Caren S.;Trachtenberg, Felicia;Gaynor, J. William;Mahle, William T.;Ravishankar, Chitra;Schwartz, Steven M.;Cnota, James F.;Ohye, Richard G.;Gongwer, Russell;Taylor, Michael;Paridon, Stephen;Frommelt, Peter C.;Afton, Katherine;Atz, Andrew M.;Burns, Kristin M.;Detterich, Jon A.;Hill, Kevin D.;Cabrera, Antonio G.;Lewis, Alan B.;Pizarro, Christian;Shah, Amee;Sharma, Binu;Newburger, Jane W.
在SVR试验(单室重建)中，患有左心发育不良综合征的新生儿被随机分配到Norwood术中接受改良的Blalock-Taussig-Thomas分流术(MBTTS)或右室-肺动脉分流术(RVPAS)。RVPAS组1年后无移植存活率较高，但6年时与治疗组无明显差异；两组患者均有重要的并发症积累。在这个队列的第三次随访中(SVRIII[患有左心发育不良综合征的儿童的长期结局和诺伍德分流型的影响])，我们测量了12岁期间的纵向结局及其危险因素。通过记录查阅和电话访谈收集年度病史。心脏磁共振成像(CMR)，超声心动图，和周期功率计心肺运动试验在10至14岁的Fontan生理学参与者中进行。无移植存活率和并发症发生率(如心律失常或蛋白缺失性肠病)的差异在12岁期间被确定。主要研究结果是CMR的右室射血分数(RVEF)，初步分析是根据接受的分流类型进行的。通过CMR和Fontan术后无移植生存率建立RVEF的多变量线性回归模型和Cox回归模型。在登记参加SVR的549名参与者中，313名无移植幸存者中有237名(76%；60.7%是男性)参与了SVRIII。分流组CMR测定的RVEF相似(RVPAS为51±9.6[n=90]，mBTTS为52±7.4[n=75]；P=0.43)。RVPAS组和mBTTS组在12岁时的无移植存活率(163/277[59%]比144/267[54%]，P=0.11)、预测VO2峰值的年龄和性别百分比(74±18%[91]比72±18%[n=84]；P=0.71)，或预测工作率(65±20%比±19%；P=0.65)。与mBTTS组相比，RVPAS组蛋白丢失性肠病的累积发生率(5%比2%；P=0.04)和导管介入治疗的累积发生率(14%比10/100病人年；P=0.01)更高，但其他并发症的发生率相似。到Norwood手术后12年，分流术类型与RVEF、峰值VO2、并发症发生率和无移植生存期的相关性最小。在接受CMR评估的幸存者亚组中，RVEF被保留。无移植存活率低，运动能力差，以及累积的发病率突出了创新策略的必要性，以改善左心发育不良综合征患者的长期结果。网址：https://www.clinicaltrials.gov；唯一标识：nct0245531。
In the SVR trial (Single Ventricle Reconstruction), newborns with hypoplastic left heart syndrome were randomly assigned to receive a modified Blalock-Taussig-Thomas shunt (mBTTS) or a right ventricle-to-pulmonary artery shunt (RVPAS) at Norwood operation. Transplant-free survival was superior in the RVPAS group at 1 year, but no longer differed by treatment group at 6 years; both treatment groups had accumulated important morbidities. In the third follow-up of this cohort (SVRIII [Long-Term Outcomes of Children With Hypoplastic Left Heart Syndrome and the Impact of Norwood Shunt Type]), we measured longitudinal outcomes and their risk factors through 12 years of age. Annual medical history was collected through record review and telephone interviews. Cardiac magnetic resonance imaging (CMR), echocardiogram, and cycle ergometry cardiopulmonary exercise tests were performed at 10 through 14 years of age among participants with Fontan physiology. Differences in transplant-free survival and complication rates (eg, arrhythmias or protein-losing enteropathy) were identified through 12 years of age. The primary study outcome was right ventricular ejection fraction (RVEF) by CMR, and primary analyses were according to shunt type received. Multivariable linear and Cox regression models were created for RVEF by CMR and post-Fontan transplant-free survival. Among 549 participants enrolled in SVR, 237 of 313 (76%; 60.7% male) transplant-free survivors (mBTTS, 105 of 147; RVPAS, 129 of 161; both, 3 of 5) participated in SVRIII. RVEF by CMR was similar in the shunt groups (RVPAS, 51±9.6 [n=90], and mBTTS, 52±7.4 [n=75]; P=0.43). The RVPAS and mBTTS groups did not differ in transplant-free survival by 12 years of age (163 of 277 [59%] versus 144 of 267 [54%], respectively; P=0.11), percentage predicted peak Vo2 for age and sex (74±18% [n=91] versus 72±18% [n=84]; P=0.71), or percentage predicted work rate for size and sex (65±20% versus 64±19%; P=0.65). The RVPAS versus mBTTS group had a higher cumulative incidence of protein-losing enteropathy (5% versus 2%; P=0.04) and of catheter interventions (14 versus 10 per 100 patient-years; P=0.01), but had similar rates of other complications. By 12 years after the Norwood operation, shunt type has minimal association with RVEF, peak Vo2, complication rates, and transplant-free survival. RVEF is preserved among the subgroup of survivors who underwent CMR assessment. Low transplant-free survival, poor exercise performance, and accruing morbidities highlight the need for innovative strategies to improve long-term outcomes in patients with hypoplastic left heart syndrome. URL: https://www.clinicaltrials.gov; Unique identifier: NCT0245531.