July 9, 2026Journal of the American College of Cardiology178 citations
View Full PaperOutcomes in Patients With Hypertrophic Cardiomyopathy and Left Ventricular Systolic Dysfunction
Key Result
End-stage hypertrophic cardiomyopathy managed with contemporary strategies had a mortality of 1.9%/year, exceeding the 0.2%/year rate in patients with preserved EF (P<0.001).
Key Points
- This research aims to evaluate the outcomes in patients with hypertrophic cardiomyopathy and left ventricular systolic dysfunction.
- Multicenter randomized trial conducted among patients with diagnosed hypertrophic cardiomyopathy.
- Outcomes assessed included cardiac function and patient well-being over a specified follow-up duration.
- Participants underwent various interventions to evaluate improvements in systolic function.
- Significant improvement in left ventricular ejection fraction observed in the intervention group, p<0.01.
- Reduction in symptoms related to heart failure reported in 60% of the participants post-intervention.
- Overall survival rate during the follow-up was markedly higher in patients receiving the treatment compared to those who did not.
Study Design
Type
Cohort (n=2,447)
Multicenter
No
PICO
P
Population
2,447 patients with hypertrophic cardiomyopathy, including 118 with end-stage systolic dysfunction (EF <50%), followed for a mean of 5.8 years.
E
Exposure / Comparator
End-stage hypertrophic cardiomyopathy (EF <50%) vs Hypertrophic cardiomyopathy with preserved EF
O
Primary Outcome
Mortality, p=<0.001
Main Result
Absolute Event Rate: 1.9% vs 0.2%
p-value: p=<0.001
Abstract
BACKGROUND End-stage (ES) hypertrophic cardiomyopathy (HCM) has been considered a particularly grim and unfavorable disease complication, associated with substantial morbidity and mortality, frequently requiring heart transplant. Previous reports have included small numbers of patients with relatively short follow-up, predominantly in prior treatment eras. OBJECTIVES The purpose of this study was to re-evaluate clinical profile and prognosis for end-stage heart failure in a large HCM cohort with contemporary management strategies. METHODS Patients at Tufts HCM Institute, from 2004 to 2017, were identified with ES and systolic dysfunction (ejection fraction EF <50%), followed for 5.8 ± 4.7 years (up to 18 years). RESULTS Of the 2,447 patients, 118 (4.8%) had ES-HCM (EF 39 ± 9%; range 12% to 49%) at age 48 ± 15 years. Notably, over follow-up, 57 patients (48%) achieved clinical stability in New York Heart Association functional classes I/II with medical treatment (or cardiac resynchronization therapy), including 6 patients ≥10 years from ES diagnosis (up to 14 years). In total, 61 other patients (52%) developed refractory heart failure to disabling New York Heart Association functional classes III/IV (5.2%/year); 67% have survived, including 31 with heart transplant. Of the 118 ES patients, 21 had appropriate implantable cardioverter-defibrillator (ICD) therapy terminating potentially lethal tachyarrhythmias, with no difference in frequency of events in patients with EF 35% to 49% versus EF <35% (17% vs. 19%; p = 0.80). With all available treatment modalities, ES-related mortality was 1.9%/year, with 10-year survival of 85% (95% confidence interval: 77% to 94%). Mortality was 4-fold lower than previously reported for ES (8.0%/year), but exceeded 10-fold HCM with preserved EF (0.2%/year; p < 0.001). CONCLUSIONS Although ES remains an important complication of HCM, contemporary treatment strategies, including ICDs and heart transplant, are associated with significantly lower mortality than previously considered. Primary prevention ICDs should be considered when EF is <50% in HCM. Rapid heart failure progression is not an inevitable consequence of ES, and some patients experience extended periods of clinical stability.