SBMA-HI: The Spinal-Bulbar Muscular Atrophy Health Index

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

Instrument Variations

• The Spinal-Bulbar Muscular Atrophy Health Index Short Form (SBMA-HI-SF)
  • SBMA-HI Short From Completion Time: Under 1 minute

Symptom Subscales

• Number of Independently Validated Symptom Subscales: 14

Mobility & Ambulation; Activity Participation; Hip, Thigh & Knee Strength; Fatigue; Social Health; Shoulder & Arm Function; Hand & Finger Function; Swallowing Function; Sleep & Daytime Sleepiness; Pain; Numbness; Emotional Health; Breathing Function; Communication

Instrument Attributes

• The SBMA-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with spinal-bulbar muscular atrophy.
• 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 SBMA-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with spinal-bulbar muscular atrophy.
• The instrument was developed using extensive patient input, including an analysis of 729 patient quotes and a large cross-sectional study involving 223 patients.
• The SBMA-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates low floor and no ceiling effects.
• Developed in conjunction with the NIH for use in therapeutic trials.
• The SBMA-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.94 for the full instrument).

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