Development and evaluation of the Acromegaly Symptom Diary

Area of Science:

• Endocrinology
• Clinical Psychology
• Biostatistics

Background:

• Acromegaly is a rare, chronic condition resulting from excess growth hormone.
• Current monitoring relies on biochemical markers, lacking patient-reported outcome (PRO) measures aligned with FDA guidance.
• Developing a validated PRO measure is crucial for comprehensive treatment evaluation in acromegaly.

Purpose of the Study:

• To develop the Acromegaly Symptom Diary (ASD).
• To establish a scoring algorithm for the ASD.
• To evaluate the psychometric properties of the ASD for acromegaly patients.

Main Methods:

• Semistructured interviews with 16 acromegaly patients for concept elicitation and cognitive debriefing.
• Evaluation of draft ASD psychometric properties using data from ACROBAT Evolve and Edge clinical trials.
• Assessment included internal consistency, test-retest reliability, construct validity, and responsiveness.

Main Results:

• Patient interviews identified key symptoms like joint pain and fatigue.
• The ASD demonstrated strong psychometric properties: internal consistency (0.80-0.91), reliability (>0.70), validity (r ≥ |0.38|), and responsiveness (r=0.52-0.56).
• A 4- to 6-point change on the 70-point ASD scale indicates meaningful symptom change.

Conclusions:

• The Acromegaly Symptom Diary (ASD) is a validated instrument for assessing patient-reported outcomes in acromegaly.
• The ASD is suitable for evaluating the symptom experience of acromegaly patients in clinical trials.
• Provides both qualitative and quantitative evidence supporting the ASD's utility.