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Center for Health + Technology [CHeT]

URMC / Center for Health + Technology / Our Expertise / CHeT Outcomes / ALD-HI: The Adrenoleukodystrophy Health Index

 

ALD-HI: The Adrenoleukodystrophy Health Index

  • Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO)
  • Administration Mode: Self-administered
  • Therapeutic Area: Adrenoleukodystrophy
  • Approximate Completion Time: <7 minutes
  • Required Supervision: None
  • Recall Period: Immediate point in time

Instrument Variations

  • The Adrenoleukodystrophy Health Index Short Form (ALD-HI-SF)
    • ALD Short From Completion Time: Under 1 minute

Symptom Subscales

  • Number of Independently Validated Symptom Subscales: 20

Mobility & Ambulation; Balance; Activity Participation; Pain; Fatigue; Bladder Function; Bowel Function; Social Satisfaction; Emotional Health; Communication; Cognition; Sleep & Daytime Sleepiness; Back, Chest & Abdominal Strength; Shoulder & Arm Function; Sensory Function; Swallowing Function; Involuntary Movements; Hand & Finger Function; Breathing; Seizures

Instrument Attributes

  • The ALD-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with adrenoleukodystrophy.
  • 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 ALD-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with adrenoleukodystrophy.
  • The instrument was developed using extensive patient input, including an analysis of 1,709 patient quotes and a large cross-sectional study involving 109 patients.
  • The ALD-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates no floor or ceiling effects.
  • Utilized in industry studies.
  • The ALD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.96 for the full instrument).
  • English

Additional translations available upon request.

  • Varma, A., Weinstein, J., Seabury, J. et al. Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD). Orphanet J Rare Dis 19, 127 (2024). https://doi.org/10.1186/s13023-024-03129-6
  • “Development of a Novel, Disease-Specific Outcome Measure for Adrenoleukodystrophy Therapeutic Trials”. Bootcamp: Women with Adrenoleukodystrophy (ALD). Virtual. August 2021.
  • “Development and Validation of Disease-Specific Patient-Reported Outcome Measure for Patients with Adrenoleukodystrophy & Adrenomyeloneuropathy”. J. Weinstein, A. Varma, C. Engebrecht, J. Seabury, S. Rosero, N. Dilek, J. Heatwole, E. Sawyer, A. Paker, G. Brooks, C. Beals, R. Gandhi, C. Heatwole. Oral Session presented at ALD Connect Annual Meeting and Patient Learning Academy; November 3-4, 2023; Houston, TX.
  • “Development and Validation of Two Patient-Reported Outcome Measures: The Adrenoleukodystrophy- Health Index (ALD-HI) and Adrenomyeloneuropathy-Health Index (AMN-HI)”. J. Weinstein, A. Varma, S. Khosa,, J. Seabury, S. Rosero, C. Engebrecht, N. Dilek, J. Heatwole, C. Irwin, C. Shupe, K. Chung, E. Sawyer, A. Paker, G. Brooks, C. Beals, R. Gandhi, C. Heatwole. The American Academy of Neurology 76th Annual Meeting; April 13-18, 2024; Denver, CO.
  • “Development and Validation of Two Patient-Reported Outcome Measures: The Adrenoleukodystrophy- Health Index (ALD-HI) and Adrenomyeloneuropathy-Health Index (AMN-HI)” J. Weinstein, A. Varma, J. Seabury, S. Rosero, C. Engebrecht, N. Dilek, J. Heatwole, C. Irwin, C. Shupe, K. Chung, P. Kanagaiah, E. Sawyer, A. Paker, G. Brooks, C. Beals, R. Gandhi, C. Heatwole. Poster presented at ALD Connect Annual Meeting and Patient Learning Academy; November 8-9, 2024; Washington, DC.

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.

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