Endocrine Abstracts

Introduction: Cushing’s Syndrome (CS) requires an accurate diagnosis, patient-tailored treatments and long-term management. To define the unmet needs of patients with CS, a consensus among a panel of Italian endocrinologists was promoted. The panel involved 57 specialists with expertise in CS management.

Methods: The endocrinologists were identified by convenience sampling. The survey was built around a set of statements formulated after literature review and discussion with an experts’ advisory board. A total of 24 statements were finally included in the survey. The Delphi method was used to reach consensus on the statements using scores on a 1-to-9 scale (with 1 indicating disagreement with the statement and 9 indicating full agreement). A 70% threshold was set to define consensus, meaning that strong agreement was believed reached if at least 70% of participants had assigned scores in the range 1-3 or 7-9, respectively.

Results: Twenty-three endocrinologists (40% of the sample) working in 10 Italian regions took part into the survey. Among them, 52% reported to follow between 5 and 10 Cushing patients per year, 22% between 10 and 20 patients, 17% more than 20 patients, and 9% less than 5 patients. The Delphi process was concluded in 2 rounds. Agreement was reached on 18 of the 24 proposed statements (75%). The statements for which consensus was not reached (n=6; 25%) were mainly related to the definition of a standard pharmacological therapy for CS, the response time to the available pharmacological therapies, the achievement of a complete control of the clinical condition, the evidence base needed for CS pharmacological therapies, and the satisfaction with the safety and effectiveness profiles of the current pharmacological therapies. Some statements in particular accounted for extremely high level of agreement (values over 90%) and they were related to the need of a constant and periodic follow-up of the patients, given the possibility of mid-long term relapse; to the relevance of the escape, which can have a strong impact on the overall management of the condition; to the need to promote the development of management pathways which are specific for CS; to the need that a newer pharmacological option, with improved safety and effectiveness profile, could radically change the current management of the CS.

Conclusions: The survey revealed that the experts involved perceive some relevant unmet needs in the management of CS, mainly related to the lack of a pharmacological treatment with more favourable safety and effectiveness profile.