Left Ventricular Systolic Dysfunction in Patients Diagnosed With Hypertrophic Cardiomyopathy During Childhood: Insights From the SHaRe Registry.
Left Ventricular Systolic Dysfunction in Patients Diagnosed With Hypertrophic Cardiomyopathy During Childhood: Insights From the SHaRe Registry.
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DOI：
10.1161/circulationaha.122.062517
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发表时间：
2023-08
期刊：
影响因子：
37.8
通讯作者：
Ho, Carolyn Y. Y.
中科院分区：
文献类型：
--
作者：
Alaiwi, Sarah Abou;Roston, Thomas M.;Marstrand, Peter;Claggett, Brian Lee;Parikh, Victoria N.;Helms, Adam S.;Ingles, Jodie;Lampert, Rachel;Lakdawala, Neal K.;Michels, Michelle;Owens, Anjali T.;Rossano, Joseph W.;Saberi, Sara;Abrams, Dominic J.;Ashley, Euan A.;Semsarian, Christopher;Stendahl, John C.;Ware, James S.;Miller, Erin;Ryan, Thomas D.;Russell, Mark W.;Day, Sharlene M.;Olivotto, Iacopo;Vissing, Christoffer R.;Ho, Carolyn Y. Y.
肥厚型心肌病（HCM）患者发生左心室收缩功能障碍（LVSD）的情况很少见，但严重且与成人不良结局相关。对儿童HCM患者LVSD的患病率、预测因素和预后知之甚少。分析了国际多中心SHaRe（肉瘤性人类心肌病登记）中HCM患者的数据。LVSD定义为超声心动图报告中左心室射血分数<50%。通过死亡、心脏移植和左心室辅助装置植入的复合指标评估预后。采用考克斯比例风险模型评估发生LVSD和随后LVSD预后的预测因素。我们研究了1010例儿童期（<18岁）诊断为HCM的患者，并将其与6741例成人诊断为HCM的患者进行了比较。在儿科HCM队列中，诊断HCM时的中位年龄为12.7岁（四分位距，8.0-15.3），393例（36%）患者为女性。在最初的SHaRe临床试验机构评估中，56例（5.5%）儿童期诊断的HCM患者存在LVSD，92例（9.1%）在中位随访5.5年期间发生LVSD。总体LVSD患病率为14.7%，而成人诊断HCM患者为8.7%。儿童队列发生LVSD时的中位年龄为32.6岁（四分位距，21.3-41.6），成人队列为57.2岁（四分位距，47.3-66.5）。儿童期诊断HCM发生LVSD的预测因素包括HCM诊断时年龄<12岁（风险比[HR]，1.72 [CI，1.13-2.62），男性（HR，3.1 [CI，1.88-5.2），携带致病性肌节变异（HR，2.19 [CI，1.08-4.4]）、既往间隔缩小术（HR，2.34 [CI，1.42-3.9]）和较低的初始左心室射血分数（HR，1.53 [CI，1.38-1.69]每降低5%）。40%的儿童期诊断的LVSD和HCM患者符合复合结局，女性参与者（HR，2.60 [CI，1.41-4.78]）和左心室射血分数<35%的患者（HR，3.76 [2.16-6.52]）的比率更高。儿童期诊断的HCM患者发生LVSD的终生风险明显更高，并且LVSD的出现早于成人诊断的HCM患者。无论诊断为HCM或LVSD时的年龄如何，LVSD的预后都很差，需要对LVSD进行仔细监测，特别是当HCM儿童过渡到成人护理时。
The development of left ventricular systolic dysfunction (LVSD) in hypertrophic cardiomyopathy (HCM) is rare but serious and associated with poor outcomes in adults. Little is known about the prevalence, predictors, and prognosis of LVSD in patients diagnosed with HCM as children. Data from patients with HCM in the international, multicenter SHaRe (Sarcomeric Human Cardiomyopathy Registry) were analyzed. LVSD was defined as left ventricular ejection fraction <50% on echocardiographic reports. Prognosis was assessed by a composite of death, cardiac transplantation, and left ventricular assist device implantation. Predictors of developing incident LVSD and subsequent prognosis with LVSD were assessed using Cox proportional hazards models. We studied 1010 patients diagnosed with HCM during childhood (<18 years of age) and compared them with 6741 patients with HCM diagnosed as adults. In the pediatric HCM cohort, median age at HCM diagnosis was 12.7 years (interquartile range, 8.0–15.3), and 393 (36%) patients were female. At initial SHaRe site evaluation, 56 (5.5%) patients with childhood-diagnosed HCM had prevalent LVSD, and 92 (9.1%) developed incident LVSD during a median follow-up of 5.5 years. Overall LVSD prevalence was 14.7% compared with 8.7% in patients with adult-diagnosed HCM. Median age at incident LVSD was 32.6 years (interquartile range, 21.3–41.6) for the pediatric cohort and 57.2 years (interquartile range, 47.3–66.5) for the adult cohort. Predictors of developing incident LVSD in childhood-diagnosed HCM included age <12 years at HCM diagnosis (hazard ratio [HR], 1.72 [CI, 1.13–2.62), male sex (HR, 3.1 [CI, 1.88–5.2), carrying a pathogenic sarcomere variant (HR, 2.19 [CI, 1.08–4.4]), previous septal reduction therapy (HR, 2.34 [CI, 1.42–3.9]), and lower initial left ventricular ejection fraction (HR, 1.53 [CI, 1.38–1.69] per 5% decrease). Forty percent of patients with LVSD and HCM diagnosed during childhood met the composite outcome, with higher rates in female participants (HR, 2.60 [CI, 1.41–4.78]) and patients with a left ventricular ejection fraction <35% (HR, 3.76 [2.16–6.52]). Patients with childhood-diagnosed HCM have a significantly higher lifetime risk of developing LVSD, and LVSD emerges earlier than for patients with adult-diagnosed HCM. Regardless of age at diagnosis with HCM or LVSD, the prognosis with LVSD is poor, warranting careful surveillance for LVSD, especially as children with HCM transition to adult care.