The Acromegaly Patient Experience: Burden of Treatment, Breakthrough Symptoms, and Quality of Life.

Abstract

Acromegaly is a chronic multisystem disorder in which growth hormone and insulin-like growth factor 1 excess cause progressive somatic, metabolic, psychological, and functional morbidity. Although biochemical control improves outcomes, many patients continue to experience persistent symptoms, impaired health-related quality of life (HRQoL), and substantial treatment burden. This review synthesizes data from clinical trials, longitudinal cohorts, registry studies, and patient-reported outcome (PRO) research evaluating physical symptoms, HRQoL, mood, interpersonal functioning, work productivity, and financial burden in acromegaly. We examine validated PRO instruments and the impact of medical, surgical, and radiation therapies on the patient experience. Fatigue, musculoskeletal pain, arthropathy, sleep disturbance, and body-image concerns are highly prevalent and frequently persist despite biochemical remission. HRQoL remains impaired in physical, psychological, and social domains, with depression and anxiety affecting a substantial proportion of patients. Treatment-related factors, including injection burden, breakthrough symptoms, gastrointestinal effects, and financial and surveillance demands further reduce well-being and productivity. PRO tools, including the Acromegaly Quality of Life Questionnaire, Patient-Assessed Acromegaly Symptom Questionnaire, Acromegaly Treatment Satisfaction Questionnaire, and the Acromegaly Symptom Diary, reveal discordance between biochemical control and PROs, highlighting the need for standardized PRO assessment and validated minimal important difference thresholds. New oral therapies and long-acting formulations may reduce treatment burden, but comparative PRO data are limited. Despite therapeutic advances, acromegaly remains associated with considerable symptom burden and impaired HRQoL. Patient-centered care requires systematic PRO incorporation, multidisciplinary management of comorbidities, attention to treatment burden, and shared decision-making.