New Patient-Reported Outcomes Diary for Rare Acromegaly Disorder

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Acromegaly Symptom Diary 

Acromegaly is a rare but serious condition caused by pituitary tumors that result in overabundance of growth hormone and insulin-like growth factor-1. This hormonal excess results in tissue growth, including changes to facial features. As the condition progresses, many adults experience lethargy, headaches, and joint pain alongside changes to their soft tissue. People with acromegaly may also experience complications that include diabetes, sleep apnea, high blood pressure, and arthritis. 

Patient using digital device to enter information.

Researchers at RTI Health Solutions and Crinetics Pharmaceuticals collaborated on developing an Acromegaly Symptom Diary (ASD) to help patients with acromegaly capture their experiences with symptoms and treatment outcomes associated with their condition. First-hand experiences with the symptoms, treatment outcomes, and impact on quality of life of health conditions are known as patient-reported outcomes (PROs) and are a major focus of ongoing drug development. The US Food and Drug Administration (FDA) strongly encourages the use of PROs in clinical trials to accurately assess treatment benefits. These measures should be carefully developed and evaluated following the recommendations of the FDA as provided in their guidance documents. Prior to this work, no PRO measures were available for acromegaly that were developed based on FDA guidance.

Experienced qualitative interviewers spoke to patients from the Acromegaly Community patient advocacy group to determine what they felt were the key symptoms of acromegaly and what symptom improvements they would like to see under ideal treatment. Open-ended questions were posed to allow patients the freedom to describe their condition experiences fully and in their own words. Patients also reviewed the ASD and gave feedback on how well it would allow them to report their outcomes. Once researchers were satisfied the ASD reflected the feedback offered by the interviewed patients, the ASD was given to patients in two clinical trials, ACROBAT Evolve (NCT03792555) and ACROBAT Edge (NCT03789656), alongside other acromegaly PRO measures. The quality of the ASD’s measurement in the context of other PRO measures was assessed to assure it provides accurate, reliable, sensitive and interpretable scores.

Through the course of this work, and thanks to direct patient input, the ASD was found to capture the experiences of patients with acromegaly well. Read the full article here.