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URMC / Center for Health + Technology / Our Expertise / CHeT Outcomes / DMD-HI: The Duchenne Muscular Dystrophy Health Index

 

DMD-HI: The Duchenne Muscular Dystrophy Health Index

  • Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO) and Caregiver or observer-reported outcome (CR/ObsRO)
  • Administration Mode: Self-administered
  • Therapeutic Area: Duchenne muscular dystrophy
  • Approximate Completion Time: <12 minutes
  • Required Supervision: None
  • Recall Period: Immediate point in time

Instrument Variations

  • Duchenne Muscular Dystrophy Health Index Short Form (DMD-HI-SF)
    • DMD-HI Short Form Completion Time: Under 1 minute
  • Duchenne Muscular Dystrophy Caregiver Reported Health Index (DMDCR-HI)  
    • DMDCR-HI Completion Time: <9 minutes; can be used for DMD patients of any age. (completed by a caregiver on behalf of a patient)
  • Duchenne Muscular Dystrophy Caregiver Reported Health Index Short Form (DMDCR-HI-SF)
    • DMDCR-HI Short Form Completion Time: Under 1 minute; can be used for DMD patients of any age. (completed by a caregiver on behalf of a patient)

Symptom Subscales

  • DMD-HI & DMDCR-HI Number of Independently Validated Symptom Subscales: 16

Ambulation; Mobility; Core & Truncal Strength; Hand & Finger Function; Shoulder & Arm Function; Emotional Health; Cognition; Sleep & Daytime Sleepiness; Heart Health; Activity Participation; Fatigue; Pain; Swallowing Function; Gastrointestinal (Stomach) Health; Communication; Breathing; Supplemental Scale for fully ambulatory patients

Instrument Attributes

  • The DMD-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with Duchenne muscular dystrophy.
  • Designed and validated to fully satisfy regulatory and published FDA guidance for use in drug-labeling claims and measuring changes in how a patient feels and functions.
  • The DMD-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with Duchenne muscular dystrophy in response to therapeutic intervention during clinical trials or clinical monitoring.
  • The DMD-HI was developed using extensive patient input, including an analysis of 1,138 patient quotes and a large cross-sectional study involving 87 patients.
  • The DMD-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates low floor and no ceiling effects.
  • The DMDCR-HI is a caregiver-reported outcome (CRO/ObsRO) measures designed to assess symptoms and health-related quality-of-life from the perspective of caregivers of individuals with Duchenne muscular dystrophy.
  • The DMDCR-HI was developed using extensive caregiver input, including an analysis of 1,657 caregiver quotes and a large cross-sectional study involving 113 caregivers.
  • The DMDCR-HI is highly relevant to patients using the perspective of the caregiver, has low patient burden, correlates with markers of disease severity, and demonstrates no floor or ceiling effects.
  • The DMD-HI and DMDCR-HI can be used for both ambulatory and non-ambulatory patients. In this way, these instruments can facilitate involvement of non-ambulatory DMD patients in therapeutic trials.
  • Developed through partnerships with the Parent Project Muscular Dystrophy (PPMD), The Muscular Dystrophy Association (MDA), and Duchenne UK.
  • Utilized in foundation studies.
  • English
  • English (UK)

Additional translations available upon request.

  • Rosero S, Weinstein J, Seabury J, et al. Patient- and caregiver-reported impact of symptoms in Duchenne muscular dystrophy. Muscle & Nerve. 2024; 1-10. doi:10.1002/mus.28102.
  • “The DMD-HI & DMDCR-HI: Development and Validation of Two Novel Duchenne Muscular Dystrophy Outcome Measures.” J. Weinstein, S. Rosero, J. Seabury, N. Dilek, A. Varma, C. Zizzi, B. Cohen, D. Alexandrou, D. Guntrum, E. Ciafaloni, C. Heatwole. Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. Nashville, TN. 3/16/22.
  • “The DMD-HI & DMDCR-HI: Development and Validation of Two Novel Duchenne Muscular Dystrophy Outcome Measures.” A. Varma, J. Weinstein, S. Rosero, J. Seabury, N. Dilek, C. Zizzi, B. Cohen, D. Alexandrou, D. Guntrum, E. Ciafaloni, C. Heatwole. PPMD 2022 Annual Conference. Scottsdale, AZ. 6/23/22.
  • “The DMD-HI & DMDCR-HI: Development, Validation, and Translation of Two Duchenne Muscular Dystrophy Patient and Caregiver Reported Outcome Measures”. A. Varma, J. Weinstein, S. Rosero, J. Seabury, C. Engebrecht, N. Dilek, C. Zizzi, J. Heatwole, B. Cohen, D. Alexandrou, D. Guntrum, E. Ciafaloni, C. Heatwole. Poster session presented at: 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference; Dallas, TX; March 19-22, 2023.
  • “The DMD-HI & DMDCR-HI: Development, Validation and Translation of Two Duchenne Muscular Dystrophy Patient and Caregiver-Reported Outcome Measure”.  S. Khosa, A. Varma, J. Weinstein, S. Rosero, J. Seabury, N. Dilek, C. Zizzi, C. Engebrecht, J. Heatwole, B. Cohen, D. Alexandrou, D. Guntrum, E. Ciafaloni, C. Heatwole. Poster session presented at The World Congress of Neurology; Montreal, Canada; October 15-19, 2023.

Instrument Scoring

All subscales are scored on a scale of 0 to 100 with 0 representing no disease burden and 100 representing the maximum level of disease burden. Symptom questions within each subscale are weighted based on participant-reported prevalence and average impact as identified through the cross-sectional study. Subscale scores are also weighted to generate a total DMD-HI or DMDCR-HI score (0-100) representing overall disease burden.

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