Development of ACRODAT(®), a new software medical device to assess disease activity in patients with acromegaly.

Despite 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. An 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. 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 (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. The 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.

van der Lely AJ ，Gomez R ，Pleil A ，Badia X ，Brue T ，Buchfelder M ，Burman P ，Clemmons D ，Ghigo E ，Jørgensen JOL ，Luger A ，van der Lans-Bussemaker J ，Webb SM ，Strasburger CJ ... -  《-》

Acromegaly disease activity according to ACRODAT®, a cross-sectional study in Spain: ACROVAL study.

To evaluate disease activity status using the Acromegaly Disease Activity Tool (ACRODAT) in a cohort of Spanish acromegaly patients, to assess the relationship between the level of disease activity according to both ACRODAT and the physicians' clinical evaluation, and to study the potential discrepancies in the perception of symptoms between physicians and patients. Multicenter, observational, descriptive and cross-sectional study. Disease activity was assessed in adult patients with acromegaly under pharmacological treatment during at least 6 months using ACRODAT. According to ACRODAT, 48.2%, 31.8% and 20.0% of a total of 111 patients were classified as having a stable disease (S), mild disease activity (M-DA) and significant disease activity (S-DA) respectively. ACRODAT classification of disease activity significantly correlated with physicians' opinion, with a moderate inter-rater agreement and a specificity of 92.45% (PPV = 86.21%). No correlation was found between IGF-I levels and severity of symptoms or quality of life (QoL). A decision to take clinical action was significantly more frequent in S-DA and M-DA patients than S patients but no action was taken on 5 (22.7%) and 27 (77.1%) S-DA and M-DA patients, respectively CONCLUSIONS: ACRODAT detected disease activity in 51.8% of patients. Interestingly, although M-DA and S-DA patients were likely to be in the process of being controlled, action was not always taken on these patients. ACRODAT is a validated and highly specific tool that may be useful to routinely monitor acromegaly and to identify patients with non-obvious disease activity by incorporating "patient-centred" parameters like symptoms and QoL to the clinical evaluation of acromegaly.

Marazuela M ，Blanco C ，Bernabeu I ，Menendez E ，Villar R ，Paja M ，Sampedro-Nuñez M ，Samaniego ML ，Díaz-Muñoz M ，Sánchez-Cenizo L ... -  《-》

Shared decision-making and detection of comorbidities in an online acromegaly consultation with and without the Acromegaly Disease Activity Tool ACRODAT(®) using the simulated person approach.

Friedel AL ，Schock L ，Siegel S ，Fritz AH ，Unger N ，Harbeck B ，Dammann P ，Kreitschmann-Andermahr I ... -  《-》

AcroVoice: eliciting the patients' perspective on acromegaly disease activity.

To determine how patients define acromegaly disease activity and treatment success and to quantify from the patients' perspective the relative importance of each disease parameter included in the ACRODAT®. One hundred acromegaly patients on medical therapy (mean age = 47.1 years; SD = 11.96) completed an online preference study evaluating hypothetical patient profiles described in terms of insulin-like growth factor-I (IGF-I) levels, tumor size, comorbid conditions, signs/symptoms, and quality of life (QoL). Participants first completed a single-profile task experiment by rating 20 single patient profiles as exhibiting stable, mild, or significant disease activity based on treatment success. Next, participants completed a double-profile discrete choice experiment (DCE) by selecting the patient that was doing "better" from 15 profile pairs. Results were analyzed using logistic and conditional logistic models. When choosing between stable vs. mild or significant disease activity, signs/symptoms, tumor size, and IGF-I levels were weighted equally; IGF-I and signs and symptoms were valued equally when selecting mild vs. significant disease activity. The DCE showed that, statistically, all disease parameters, except comorbid conditions, predicted health status equally. Tumor size and IGF-I levels each accounted for 23% of the decision-making process; QoL, signs/symptoms, and comorbid conditions accounted for 21%, 19%, and 14%, respectively. All five ACRODAT® parameters had some influence on disease activity from the patients' perspective. To account for patients' preferences and optimize treatment and outcomes, a holistic disease management approach should be employed.

Jackson Y ，Flood E ，Rhoten S ，Janssen EM ，Lundie M ... -  《-》

SAGIT®: clinician-reported outcome instrument for managing acromegaly in clinical practice--development and results from a pilot study.

Giustina A ，Bevan JS ，Bronstein MD ，Casanueva FF ，Chanson P ，Petersenn S ，Thanh XM ，Sert C ，Houchard A ，Guillemin I ，Melmed S ，SAGIT Investigator Group ... -  《-》