Disease burden, treatment experiences and preferences in patients with acromegaly: a qualitative study

Purpose: Acromegaly is a rare disease with limited treatment options. Understanding treatment burden and patient preferences is important for evaluating new treatments and optimizing adherence. Methods: Patients with acromegaly (≥18 years) from the United States (US) participated in qualitative semi-structured interviews to explore: 1) the impact of acromegaly on quality of life, 2) patients' experiences with current treatment, 3) preferences for new treatments. Results: Fifteen US patients with acromegaly participated, reporting a range of symptoms; physical changes/swelling, fatigue/tiredness and excessive sweating. Frequently reported impacts included limited socializing (n = 6), anxiety (n = 4), embarrassment due to sweating/odor (n = 4) and clothing adaptations due to swelling (n = 4). Frequently reported impacts associated with monthly injectable treatment included unpleasantness of injections/blood tests (n = 6), clinic waiting time (n = 5), travelling to the clinic (n = 3), and treatment frequency (n = 3). Over half of patients preferred daily oral treatment options (n = 8, 53.3%). When asked about preference around hypothetical treatment frequency, 60.0% (n=9) preferred a hypothetical 3-monthly injection compared to a monthly injection if it was as efficacious as the monthly injection or recommended by their doctor. Conclusion: Patients experience a wide range of symptoms and impacts, with a high burden of treatment associated with monthly injections. Patients demonstrated preferences for less frequent treatments, with a preference for reducing their current monthly injections to three or six monthly. When considering new treatments, the efficacy and safety profile were of most importance to patients with acromegaly.