MITD-HI: Mitochondrial Disease Health Index (Patient-reported Outcome)

Our MITD-HI: Mitochondrial Disease Health Index is a rigorously developed patient-reported outcome (PRO) that reflects how individuals experience symptoms, functioning, and quality of life. It is used across clinical research and trials to generate meaningful, patient-centered data on disease burden and treatment effects.

• Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO)
• Administration Mode: Self-administered
• Therapeutic Area: Mitochondrial Disease
• Approximate Completion Time: <8 minutes
• Required Supervision: None
• Recall Period: Immediate point in time

Symptom Subscales

• Number of Independently Validated Symptom Subscales: 19

Muscle Strength; Mobility & Ambulation; Fatigue; Sleep & Daytime Sleepiness; Activity Participation; Pain; Gastrointestinal Health; Social Satisfaction; Coordination; Headaches; Cognitive Function; Vision; Emotional Health; Arms, Hands, & Fingers Function; Numbness; Swallowing Function; Communication; Breathing; Seizures

Instrument Attributes

• The MITD-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with mitochondrial disease.
• 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 MITD-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with mitochondrial disease.
• The instrument was developed using extensive patient input, including an analysis of 4,365 participant quotes and a large national cross-sectional study involving 145 participants with mitochondrial disease.
• Participants with over 40 types and variants of mitochondrial disease contributed to the development and validation of the MITD-HI, ensuring its applicability to a broad spectrum of individuals with mitochondrial disease. 
• The MITD-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates no floor or ceiling effects.
• The MITD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument).
• eCOA implementation guide available.

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