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nHCM长期缺乏靶向治疗药物，随着阿夫凯泰ACACIA-HCM研究取得阳性结果，nHCM的治疗格局正面临根本性重塑。

肥厚型心肌病（HCM）是最常见的心血管疾病之一，根据是否存在静息或诱发的左心室流出道（LVOT）梗阻，HCM通常分为梗阻性HCM（oHCM）和非梗阻性HCM（nHCM）[1-4]。长期以来，临床研究和药物开发更多聚焦于oHCM，主要原因在于其梗阻机制明确、症状与血流动力学改变之间的关联相对直观，且已有成熟的药物和介入/外科治疗路径。相比之下，nHCM虽然缺乏明显的左心室流出道梗阻，但并不意味着疾病负担较轻。nHCM患者常常面临劳力性呼吸困难、运动耐量下降、舒张功能障碍、房颤、室性心律失常、心源性猝死以及进展为晚期心衰甚至终末期心肌病等风险[1-4]。

近年来，随着对HCM分子机制认识的不断加深，尤其是心肌肌球蛋白抑制剂（CMI）的问世，nHCM治疗理念正在发生变化。过去以经验性、对症性治疗为主的管理模式，正逐步向“基于病理生理机制的精准干预”过渡[1,4,5]，也使nHCM成为近年来心肌病领域最受关注的前沿方向之一。

参考文献：

[1]Desai, M, Maurizi, N, Biagini, E. et al. Pathophysiology and Therapeutic Needs in Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol HF. 2025 Nov, 13 (11) .

[2]Maron BJ, Desai MY, Nishimura RA, Spirito P, Rakowski H, Towbin JA, Rowin EJ, Maron MS, Sherrid MV. Diagnosis and Evaluation of Hypertrophic Cardiomyopathy: JACC State-of-the-Art Review. J Am Coll Cardiol. 2022 Feb 1;79(4):372-389.

[3]2024 AHA/ACC/AMSSM/HRS/PACES/SCMR Guideline for the Management of Hypertrophic Cardiomyopathy: A Report of the American Heart Association/American College of Cardiology Joint Committee on Clinical Practice Guidelines. Circulation. 2024 Jun 4;149(23):e1239-e1311.

[4]国家心血管病中心心肌病专科联盟,中国医疗保健国际交流促进会心血管病精准医学分会"中国成人肥厚型心肌病诊断与治疗指南2023"专家组. 中国成人肥厚型心肌病诊断与治疗指南2023[J]. 中国循环杂志,2023,38(1):1-33.

[5]Ordine L, Polizzi R, Canciello G, et al. Unveiling the complexity of nonobstructive hypertrophic cardiomyopathy. Heart Fail Rev. 2025 Dec; 30(6): 1141-1153.

[6]《心肌肌球蛋白抑制剂药物治疗专家共识》工作组. 心肌肌球蛋白抑制剂药物治疗专家共识[J]. 临床药物治疗杂志. Vol.24，No.3 March，2026.

[7]Ho CY, Mealiffe ME, Bach RG, et al. Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2020 Jun 2;75(21):2649-2660.

[8]ODYSSEY-HCM Investigators. Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2025 Sep 11;393(10):961-972.

[9]REDWOOD-HCM Investigators. Efficacy and Safety of Aficamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4. J Card Fail. 2024 Nov;30(11):1439-1448.

[10]Cytokinetics Announces Positive Topline Results from ACACIA-HCM, the Pivotal Phase 3 Clinical Trial of Aficamten in Patients with Non-Obstructive Hypertrophic Cardiomyopathy. https:// ir.cytokinetics.com/ press-releases/ press-release-details/2026/Cytokinetics-Announces-Positive-Topline-Results-from-ACACIA-HCM-the-Pivotal-Phase-3-Clinical-Trial-of-Aficamten-in-Patients-with-Non-Obstructive-Hypertrophic-Cardiomyopathy/default.aspx（访问日期2026年7月14日）

[11]Phase 2 randomized, double-blind, placebo controlled study to evaluate BHB/HRS-1893 in adults with symptomatic non-obstructive hypertrophic cardiomyopathy. ESC-HFA 2026.

[12]Meder B, Coats CJ, Leinwand LA, et al. EJHF expert consensus statement on the diagnosis and management of hypertrophic cardiomyopathy. Eur J Heart Fail. 2026 May 11;28(2):299-314.

[13]Maron MS, Mahmod M, Abd Samat AH, et al. Safety and Efficacy of Metabolic Modulation With Ninerafaxstat in Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2024 May 28;83(21):2037-2048.

[ MAT-CN-2604243 V3.0 批准日期：2026年8月]

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