Development of ACRODAT\xae, a new software medical device to assess disease activity in patients with acromegaly

Aart J Van Der Lely,Roy Gomez,Christian J Strasburger,Thierry Brue,Michael Buchfelder,Xavier Badia,David Clemmons,Andreas Pleil,Joli Van Der Lans-Bussemaker,Ezio Ghigo,Susan M Webb,Anton Luger,Jens Otto Lunde Jørgensen,Pia Burman

doi:10.1007/s11102-017-0835-5

Abstract

PurposeDespite availability of multimodal treatment options for acromegaly, achievement of long-term disease control is suboptimal in a significant number of patients. Furthermore, disease control as defined by biochemical normalization may not always show concordance with disease-related symptoms or patient’s perceived quality of life. We developed and validated a tool to measure disease activity in acromegaly to support decision-making in clinical practice.MethodsAn international expert panel (n = 10) convened to define the most critical indicators of disease activity. Patient scenarios were constructed based on these chosen parameters. Subsequently, a panel of 21 renowned endocrinologists at pituitary centers (Europe and Canada) categorized each scenario as stable, mild, or significant disease activity in an online validation study.ResultsFrom expert opinion, five parameters emerged as the best overall indicators to evaluate disease activity: insulin-like growth factor I (IGF-I) level, tumor status, presence of comorbidities (cardiovascular disease, diabetes, sleep apnea), symptoms, and health-related quality of life. In the validation study, IGF-I and tumor status became the predominant parameters selected for classification of patients with moderate or severe disease activity. If IGF-I level was ≤1.2x upper limit of normal and tumor size not significantly increased, the remaining three parameters contributed to the decision in a compensatory manner.ConclusionThe validation study underlined IGF-I and tumor status for routine clinical decision-making, whereas patient-oriented outcome measures received less medical attention. An Acromegaly Disease Activity Tool (ACRODAT) is in development that might assist clinicians towards a more holistic approach to patient management in acromegaly.

Highlights

Is a rare chronic disease associated with metabolic abnormalities, risk of cardiovascular complications, slowly progressive, irreversible disfigurement, and increased mortality [1]
From expert opinion, five parameters emerged as the best overall indicators to evaluate disease activity: insulin-like growth factor I (IGF-I) level, tumor status, presence of comorbidities, symptoms, and health-related quality of life
The health-related QoL (HRQoL) impairment parameter was based on the standardized total score from a validated measure of the Acromegaly Quality of Life Questionnaire (AcroQoL)

Summary

Introduction

Is a rare chronic disease associated with metabolic abnormalities, risk of cardiovascular complications, slowly progressive, irreversible disfigurement, and increased mortality [1]. Visible signs include enlarged hands and feet, enlarged jaw and facial bones, thickening of the skin, and excessive sweating. Common patient complaints include headache, joint pain, fatigue and sleep disturbances [1, 2]. Has been associated with reduced quality of life (QoL) [3], which may show improvement with treatment [4,5,6]. Patients are frequently not diagnosed until 5–10 years after onset [1] and, if disease control is not achieved, acromegaly is associated with increased mortality and risk of metabolic and cardiac complications [1, 7, 8]

Methods

Results

Conclusion