Heart failure & cardiomyopathies
Heart failure guideline-directed medical therapy in young adults with dystrophin-related cardiomyopathy: the HOPE-MD registry
- European, multicentre, registry
Authors' conclusion
In this European, multicentre registry, GDMT use and dose escalation were suboptimal in young patients with DMD/BMD and HFrEF.
Study context
- Population
- Adults with genetically confirmed DMD or BMD and left ventricular ejection fraction (LVEF) ≤ 40%After 24 months (n = 141)
- Participants
- 274 adults
- Exposure
- optimized guideline-directed medical therapy (GDMT) ... compared with low-dose or no GDMT
Main finding
As reported in the abstract
GDMT use at baseline · baseline
received GDMT
44 (16%)
of those receiving GDMT, at ≥50% of the target dose
only one quarter
Observational data: associations, not effects.
Further findings
| Outcome | adjusted HR (95% CI) | |
|---|---|---|
| Death or HF hospitalization · median follow-up of 2.2 (1.7-2.6) years · not associated with a statistically significant difference | 0.70 (0.28–1.74) | |
| 0.20.5125Adjusted hazard ratio (log scale) |
Use of individual drug classes at baseline
- Underutilization of individual drug classes at baseline: Mineralocorticoid receptor antagonist (MRAs) n = 140,
- 51%
- SGLT2i
- n = 60, 22%
- Renin-angiotensin system inhibitor (RASi)
- n = 246, 90%
- beta-blockers
- n = 235, 86%
GDMT use after 24 months
- received GDMT
- 40%
- received ≥50% of the target dose
- 22%
- n = 141
- primarily through MRA and SGLT2i initiation/uptitration
- Contraindications accounted for only a small proportion of undertreatment
Original publication
Heart failure guideline-directed medical therapy in young adults with dystrophin-related cardiomyopathy: the HOPE-MD registry. Eur Heart J Qual Care Clin Outcomes. Published 21 May 2026. doi:10.1093/ehjqcco/qcag088
Summarised from the published abstract; every number is checked against it