Cardiac Performance Evaluation in Patients with Muscular Dystrophy

Q: What are the key findings of this study?

Ventriculoarterial coupling (V AC) ratio was significantly higher (0.8 vs 0.6; p=0.004) and ejection fraction significantly lower (57.7% vs 63.3%; p=0.0001) in pediatric muscular dystrophy patients versus controls, and worsened over 4.1 years median follow-up.

Q: What is the clinical evidence from this study?

Study design: Observational. Population: Pediatric patients (<21 years) with muscular dystrophy (Duchene or Becker phenotype) undergoing cardiac evaluation (n=46). Intervention: Observation with echocardiographic monitoring including V AC ratio assessment vs. Age-matched healthy male controls. Primary outcome: Cardiac function measured by ventriculoarterial coupling (V AC) ratio and left ventricular ejection fraction (LVEF) (V AC ratio higher in MD patients vs controls, p=0.004; EF 57.7% vs 63.3% in controls, p=0.0001, p=0.015 for longitudinal worsening of V AC).

Q: What does this research mean for the field?

Pediatric muscular dystrophy patients exhibit significantly impaired cardiac performance, characterized by a higher ventriculoarterial coupling ratio and lower ejection fraction compared to controls, which progressively worsens over time. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

Naomi Khanna; Joseph Mahgerefteh; Noah Elkins; D. Hsu; Jacqueline M. Lamour; Leslie Delfiner; Induja Gajendran; N. Bansal

doi:10.1007/s00246-026-04190-7

February 20, 2026Pediatric CardiologyOpen Access

Cardiac Performance Evaluation in Patients with Muscular Dystrophy

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Key result

Pediatric muscular dystrophy linked to worse ventriculoarterial coupling and lower LVEF, worsening over ~4 years.

V AC ratio higher in MD patients vs controls, p=0.004; EF 57.7% vs 63.3% in controls, p=0.0001
P=0.015 for longitudinal worsening of V AC
n=46

Why the study?

It was hypothesized that ventriculoarterial coupling is abnormal in patients with muscular dystrophy and worsens with disease progression.

Key Points

To determine whether ventriculoarterial coupling is abnormal in pediatric patients with muscular dystrophy and whether it deteriorates with disease progression.
Retrospective cohort analysis of 46 muscular dystrophy patients (median age at diagnosis 9.8 years; 76% Duchenne, 24% Becker) followed for a median of 4.1 years (IQR 3–6).
Compared initial and last follow-up echocardiographic parameters—arterial elastance (Ea), ventricular elastance (Ees), and ventriculoarterial coupling (VAC)—between patients and 66 age-matched male controls.
Conducted univariable and multivariable regression analyses to identify clinical factors independently associated with VAC and cardiac performance.
At diagnosis, MD patients had significantly lower EF (57.7 ± 7.6% vs. 63.3 ± 4.8%, p=0.0001) and Ees (3.3 ± 1.4 vs. 5 ± 6.2, p=0.038), and higher VAC ratio (0.8 ± 0.3 vs. 0.6 ± 0.1, p=0.004) compared to healthy controls.
Over longitudinal follow-up, MD patients demonstrated significant cardiac deterioration, with EF decreasing from 57.7 ± 7.6% to 52.8 ± 11.1% (p=0.015), Ees dropping from 3.3 ± 1.4 to 2.5 ± 1.3 (p=0.001), and VAC worsening from 0.8 ± 0.3 to 1.05 ± 0.7 (p=0.015).
Non-ambulatory status was independently associated with worse VAC (β = 0.520, SE = 0.225, p = 0.026), lower EF (β = -0.5, p = 0.003), and lower Ees (β = -0.44, p = 0.015) at last follow-up.

Study Design

Type

Observational (n=46)

Multicenter

No

Structured PICO

P

Population

46 pediatric patients (<21 years) with muscular dystrophy (76% Duchenne, 24% Becker), median age at diagnosis 9.8 years, 41% wheelchair-bound, 70% on oral steroids, 65% on ACEi/ARB, compared to 66 age-matched healthy male controls.

C

Comparator

66 age-matched healthy male controls with normal echocardiograms and no evidence of cardiac disease.

O

Outcome

Ventriculoarterial coupling (VAC) ratio, arterial elastance (Ea), and LV end-systolic elastance (Ees) measured by echocardiography at initial diagnosis and last follow-up.surrogate

Main Result

Effect estimate: V AC ratio higher in MD patients vs controls, p=0.004; EF 57.7% vs 63.3% in controls, p=0.0001

Absolute Event Rate: 1.05% vs 0.6%

p-value: p=0.015 for longitudinal worsening of V AC

Ventriculoarterial coupling is significantly impaired in pediatric patients with muscular dystrophy compared to healthy controls and worsens over time, with loss of ambulation serving as an independent marker of cardiovascular decline.

Limitations

Single-center study with small sample size
Retrospective design with variable follow-up time
Potential selection bias with medication use
Unable to control for timing of medication initiation
Echocardiograms did not uniformly coincide with diagnosis time
Inter-observer variability in echocardiographic assessments
Unable to evaluate impact of medications on cardiac function due to observational design
single-center study
small sample size
variable follow up
retrospective design
did not account for other multiple medications, airway clearance regimens and pulmonary function tests
inter-observer variability in the assessment of echocardiograms
echocardiographic studies performed at our center did not uniformly coincide with the time of initial diagnosis
inability to systematically capture medication initiation or changes between echocardiograms

Implication

Observational data in pediatric muscular dystrophy warrant no practice change; leaves open VAC as a progression biomarker for prospective trials.

Abstract

Ventriculoarterial coupling (VAC), the ratio of arterial elastance (Ea) to LV end-systolic elastance (Ees), measures the relationship between ventricular performance and arterial stiffness. We hypothesized that VAC is abnormal in muscular dystrophy (MD) patients and worsens with disease progression. MD patients < 21 years were included. Demographics, MD phenotype, medications, medical history, and clinical status were collected. Initial and last follow up echocardiograms were reviewed to determine Ea, Ees, and VAC and compared between MD and age-matched males and between the initial and last follow up studies in the MD patients. Univariable and multivariable regression analyses was performed to determine factors independently associated with VAC. Forty-six MD patients, median age at diagnosis 9.8 years (IQR 7 -12.2), followed for a median of 4.1 years (IQR 3–6) were included for study. 41% patients were wheelchair-bound, 67% were on steroids, and 65% on afterload reducing medications. Ejection Fraction (EF) < 55% was found in 28% of patients at diagnosis and in 44% at the last follow-up. EF and Ees were significantly lower and VAC ratio was significantly higher (worse) in the MD patients compared to controls & showed significant worsening from diagnosis to last follow up. Ambulation status was independently associated with EF and Ees at last follow up. Cardiac performance, as measured by VAC ratio, is impaired in patients with MD compared with a control population and worsens with longitudinal follow-up. Ambulation status is independently associated with this impairment, offering mechanistic insight into the development and progression of cardiac dysfunction.

Discussion

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Cite This Study

Khanna et al. (2026) conducted an observational in Pediatric patients (<21 years) with muscular dystrophy (Duchene or Becker phenotype) undergoing cardiac evaluation (n=46). Observation with echocardiographic monitoring including V AC ratio assessment vs. Age-matched healthy male controls was evaluated on Cardiac function measured by ventriculoarterial coupling (V AC) ratio and left ventricular ejection fraction (LVEF) (V AC ratio higher in MD patients vs controls, p=0.004; EF 57.7% vs 63.3% in controls, p=0.0001, p=0.015 for longitudinal worsening of V AC). Ventriculoarterial coupling (V AC) ratio was significantly higher (0.8 vs 0.6; p=0.004) and ejection fraction significantly lower (57.7% vs 63.3%; p=0.0001) in pediatric muscular dystrophy patients versus controls, and worsened over 4.1 years median follow-up.

synapsesocial.com/papers/699a9d8e482488d673cd3782 https://doi.org/10.1007/s00246-026-04190-7

Topics

Echocardiography

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Cardiac Performance Evaluation in Patients with Muscular Dystrophy

Key Points

Study Design

Structured PICO

Main Result

Limitations

Implication

Abstract

Discussion

Cite This Study

Topics

Cardiac Performance Evaluation in Patients with Muscular Dystrophy

Key Points

Study Design

Structured PICO

Main Result

Limitations

Implication

Abstract

Discussion

Cite This Study

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Topics

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