Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial.

This analysis explores whether oHCM and mild symptoms patients experience similar clinical benefits with aficamten as patients with more advanced limitations.

本分析探讨了轻度症状的oHCM患者是否与症状更严重的患者一样，接受aficamten治疗后能体验到类似的临床益处。

Methods

Patients in SEQUOIA-HCM (N = 282) were grouped at baseline according to symptom severity.

SEQUOIA-HCM 研究中的患者 (N = 282) 在基线时根据症状严重程度被分组。

Mild symptoms (n = 118) were defined as New York Heart Association (NYHA) class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥ 80, and moderate to severe symptoms (n = 150) as NYHA class II/III/IV and KCCQ-CSS <80.

轻度症状 (n = 118) 被定义为纽约心脏协会 (NYHA) 心功能 II 级和堪萨斯城心肌病问卷临床总结评分 (KCCQ-CSS) ≥ 80，中度至重度症状 (n = 150) 则为 NYHA 心功能 II/III/IV 级和 KCCQ-CSS <80。

Primary endpoint was change in peak oxygen uptake (pVO2) from baseline to Week 24; secondary endpoints included change in NYHA class, KCCQ-CSS, outflow tract gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP).

主要终点是从基线到第24周时最大氧摄取量（pVO2）的变化；次要终点包括纽约心脏病协会（NYHA）心功能分级、堪萨斯城心肌病问卷临床总评分（KCCQ-CSS）、流出道梯度和N-末端前体脑钠肽（NT-proBNP）的变化。

Results

In aficamten-treated patients, change at Week 24 was not different between moderate to severe (1.8 mL/kg/min; n = 71) and mild (1.6 mL/kg/min; n = 62) symptom groups (P = .8).

在接受Aficamten治疗的患者中，第24周时，中度至重度症状组（1.8 mL/kg/min；n = 71）与轻度症状组（1.6 mL/kg/min；n = 62）之间的变化无差异（P = .8）。

Likewise, the change in secondary endpoints (NYHA class, resting or Valsalva gradients, and NT-proBNP) did not differ significantly between the two symptom groups.

同样，次要终点（纽约心脏病协会（NYHA）心功能分级、静息或Valsalva动作梯度和NT-proBNP）在两组症状患者之间的变化没有显著差异。

Both groups experienced statistically significant improvements in KCCQ-CSS, but the extent of improvement was greater in the advanced symptom group (P = .02 for interaction).

两组患者在堪萨斯城心肌病患者自评量表（KCCQ-CSS）上都经历了统计学上显著的改善，但改善程度在症状较重的组别中更大（交互作用P = .02）。

Treatment-emergent serious adverse events were infrequent in both groups.

两组中治疗后出现的严重不良事件均不常见。

Conclusions

Patients with oHCM and mild symptoms treated with aficamten achieved significant improvement across a range of clinically relevant outcomes and generally similar to patients with more advanced symptoms.

接受阿非卡门治疗的有症状的肥厚型心肌病（oHCM）患者，在一系列临床相关结果上取得了显著改善，并且通常与症状更严重的患者相似。

Less severely symptomatic patients could be considered for aficamten treatment.

症状较轻的患者可以考虑接受阿非卡门治疗。