LGMD-HI: The Limb Girdle Muscular Dystrophy Health Index

• Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO)
• Administration Mode: Self-administered
• Therapeutic Area: Limb Girdle Muscular Dystrophy
• Approximate Completion Time: ~10 minutes
• Required Supervision: None
• Recall Period: Immediate point in time

Instrument Variations

• The Limb Girdle Muscular Dystrophy Health Index Short Form (LGMD-HI-SF)
  • LGMD-HI-SF Completion Time: Under 1 minute
• The Limb Girdle Muscular Dystrophy Health Index Enhanced Design for Global Efficiency (LGMD-HI-EDGE)
  • LGMD-HI-EDGE Completion Time: ~5 minutes

Symptom Subscales

• Number of Independently Validated Symptom Subscales: 15

Ambulation, Lower Extremity Function, Hand Function, Arm & Neck Strength, Emotional Health, Embarrassment of Gait, Social Performance, Social Satisfaction, Daytime Sleepiness, Activity Participation, Fatigue, Pain, Swallowing, Breathing, & Heart Palpitations

Instrument Attributes

• The LGMD-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with limb girdle muscular dystrophy.
• Designed and validated to fully satisfy regulatory and published FDA guidance for use in drug-labelling claims and measuring changes in how a patient feels and functions.  
• The LGMD-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with limb girdle muscular dystrophy in response to therapeutic intervention during clinical trials or clinical monitoring.
• The instrument was developed using extensive patient including an analysis of 1,385 patient quotes and a large cross-sectional study involving 163 patients.
• The LGMD-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates few ceiling or floor effects.
• The LGMD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.92 for the full instrument).
• The Limb Girdle Muscular Dystrophy Health Index EDGE (LGMD-HI-EDGE) is a disease-specific patient reported outcome measure designed to measure multifactorial patient-reported burden of disease in Limb Girdle Muscular Dystrophy (LGMD) patients.  The LGMD-HI-EDGE comprises 43 items that measure 13 granular areas of patient health.  Together, the subscales can be utilized to quantify a LGMD patient’s overall disease burden.  Each question in the instrument was selected based on its high relevance to the LGMD population, its ability to be consistently understood by patients and clinicians, its reliability during test-retest evaluation, its content validity, its face validity, and its responsiveness in detecting both therapeutic change and differences between known groups with higher or lower disease severity.  Compared to the LGMD-HI, the LGMD-HI-EDGE is a shorter instrument where all questions have been specifically selected to maximize the responsiveness and usability of the instrument in detecting therapeutic gain during clinical trials.

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 LGMD-HI score (0-100) representing overall disease burden.