POMPE-FIT: The Pompe-Functional Impact Tool (Patient-reported Outcome)

Our POMPE-FIT: The Pompe-Functional Impact Tool is a rigorously developed patient-reported outcome (PRO) that reflects how individuals with Pompe disease experience symptoms, functioning, and quality of life. It can be 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: Pompe disease
• Approximate Completion Time: <5 minutes
• Required Supervision: None
• Recall Period: Immediate point in time

Instrument Variations

• The Pompe-Functional Impact Tool Short Form (POMPE-FIT-SF)
  • POMPE-FIT-SF Completion Time: Under one minute
• The Pompe Caregiver Reported-Functional Impact Tool (POMPE-CR-FIT) (age 0+)
  • POMPE-CR-FIT (age 0+) Completion Time: <5 minutes
• The Pompe Caregiver Reported-Functional Impact Tool (POMPE-CR-FIT) Short Form (age 0+)
  • POMPE-CR-FIT Short Form (age 0+) Completion Time: Under one minute
• The Pompe Caregiver Reported-Functional Impact Tool (POMPE-CR-FIT) (age 2+)
  • POMPE-CR-FIT (age 2+) Completion Time: <5 minutes
• The Pompe Caregiver Reported-Functional Impact Tool (POMPE-CR-FIT) Short Form (age 2+)
  • POMPE-CR-FIT Short Form (age 2+) Completion Time: Under one minute

Symptom Subscales

• Number of Independently Validated Symptom Subscales: 18

Mobility & Ambulation; Fatigue; Breathing Function; Shoulder & Arm Function; Lower Extremity Function; Activity Participation; Back, Chest & Abdominal Strength; Pain; Emotional Health; Sleep; Daytime Sleepiness; Heart Health; Numbness; Social Satisfaction; Communication; Cognition; Hand & Finger Function; Swallowing Function

Instrument Attributes

• The POMPE-FIT is a valid and responsive patient-reported outcome (PRO) measure designed to assess disease symptoms and measure changes in health in patients with Pompe disease.
• Designed and validated for use in drug-labeling claims and measuring changes in how a patient feels and functions.
• The instrument was developed using extensive patient input, including an analysis of 3,509 patient quotes.
• The POMPE-FIT is highly relevant to patients and has low patient burden.
• 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 POMPE-FIT score (0-100) representing overall disease burden.