Hypertrophic Cardiomyopathy Clinic and Heart and Vascular Center, St. Luke's University Health Network, Bethlehem, Pennsylvania; Hypertrophic Cardiomyopathy Institute, Tufts Medical Center, Boston, Massachusetts.
Hypertrophic Cardiomyopathy Clinic and Heart and Vascular Center, St. Luke's University Health Network, Bethlehem, Pennsylvania; Hypertrophic Cardiomyopathy Institute, Tufts Medical Center, Boston, Massachusetts.
Am J Cardiol. 2021 Mar 1;142:130-135. doi: 10.1016/j.amjcard.2020.11.035. Epub 2020 Dec 3.
Major advances in diagnosis and treatment have emerged for hypertrophic cardiomyopathy (HCM), largely in major tertiary referral centers dedicated to this disease. Whether these therapeutic benefits are confined to patients in such highly selected cohorts, or can be implemented effectively in independent regional or community-based populations is not generally appreciated. We assessed management and clinical outcomes in a non-referral HCM center (n = 214 patients) in Eastern Pennsylvania. Over a 6.0 ± 3.2-year follow-up, the HCM-related mortality rate was 0.1% per year attributed to a single disease-related death, in a 49-year-old man with end-stage heart failure, ineligible for heart transplant. Fifteen patients (7%) with prophylactically placed implantable cardioverter-defibrillators (ICDs) experienced appropriate therapy terminating life-threatening ventricular tachyarrhythmias. In 23 other patients (11%; 5%/year), heart failure due to left ventricular outflow obstruction was reversed by surgical septal myectomy (n = 20) or percutaneous alcohol septal ablation (n = 3). This regional HCM cohort was similar to a comparison tertiary center referral population in terms of HCM-mortality: 0.1%/year vs 0.3%/year (p = 0.3) and ICD therapy (31% vs 16% of primary prevention implants), although more frequently with uncomplicated benign clinical course (62% vs 46%; p <0.01). In conclusion, effective contemporary HCM management strategies and outcomes in referral-based HCM centers can be successfully replicated in regional and/or non-referral settings. Therefore, HCM is now a highly treatable disease compatible with normal longevity when assessed in a variety of clinical venues not limited to tertiary centers.
肥厚型心肌病（HCM）的诊断和治疗取得了重大进展，主要集中在专门治疗这种疾病的大型三级转诊中心。这些治疗益处是否仅限于这些高度选择的患者群体，或者能否在独立的地区或社区人群中有效实施，人们对此认识不足。我们评估了宾夕法尼亚州东部一家非转诊 HCM 中心（n=214 例患者）的治疗管理和临床结局。在 6.0±3.2 年的随访中，HCM 相关死亡率为每年 0.1%，归因于一位 49 岁患有终末期心力衰竭、不符合心脏移植条件的男性患者的单一疾病相关死亡。15 例（7%）预防性植入植入式心律转复除颤器（ICD）的患者经历了适当的治疗，终止了危及生命的室性心动过速。在另外 23 例（11%；5%/年）患者中，左心室流出道梗阻导致的心力衰竭通过外科室间隔心肌切除术（n=20）或经皮酒精室间隔消融术（n=3）得到逆转。与转诊的三级中心比较，这个地区性 HCM 患者群体在 HCM 死亡率方面相似：0.1%/年比 0.3%/年（p=0.3）和 ICD 治疗（初级预防植入的 31%比 16%），尽管更常见于无并发症的良性临床病程（62%比 46%；p<0.01）。总之，在转诊 HCM 中心成功实施的现代 HCM 治疗管理策略和结局，也可以在地区性和/或非转诊环境中成功复制。因此，HCM 现在是一种可治疗的疾病，当在不限于三级中心的各种临床场所进行评估时，其与正常长寿是兼容的。
