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.

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.