Journal of patient-reported outcomes · 2025
Real-world burden of disease, treatment, and healthcare resource utilization in acromegaly: a quantitative survey of patient experiences.
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Koobatian Maxwell, Sisco Jill, Okeyo Janetricks C, Krasner Alan, Quock Tiffany P
Аннотация
BACKGROUND: Acromegaly is a rare endocrine disease caused by excessive growth hormone (GH) secretion typically due to a pituitary adenoma. Patients ineligible for or with an inadequate response to surgery and/or radiotherapy often require pharmacotherapy such as depot somatostatin receptor ligand (SRL) injections. This study evaluated the current management of patients with acromegaly and real-world experience of disease burden, treatment burden, and healthcare resource utilization (HCRU).
METHODOLOGY: A quantitative study was conducted among symptomatic adults with acromegaly receiving therapy, including depot SRL injections. A web-based survey captured the 3-month disease experience including the presence and severity of acromegaly-associated symptoms, treatment experience, HCRU, and impact on the ability to work (Work Productivity and Impairment Questionnaire [WPAI], assessed over prior 7 days).
RESULTS: Among 58 patients who completed the survey, 36 (62.1%) received a depot SRL injection, either as monotherapy (18 [50%]), or in combination with other agents. All patients experienced ≥ 1 symptom during the previous 3 months, with 67% having ≥ 1 symptom with a severity of ≥ 8 on a scale of 0 to 10. Patients reported that acromegaly had a moderate (59%) or high (22%) level of interference in their life overall. Of 18 patients on depot SRL injection monotherapy, 12 (67%) reported ≥ 1 breakthrough acromegaly symptom at any time prior to the next injection. WPAI scores were 51% for daily activity impairment, 38% for presenteeism, 34% for overall work impairment, and 8% for absenteeism. Patients receiving depot SRL injection (monotherapy/combination therapy) were more likely than those not receiving this injection to report moderate-to-high interference of acromegaly treatment with their life (56%/72% vs. 18%) and that their treatment was moderately to highly burdensome (67%/72% vs. 41%). HCRU due to acromegaly treatment included: a mean of 2.6 office visits, 13.8% with ≥ 1 emergency department visit, and 10.3% with ≥ 1 overnight hospitalization. When asked about treatment preferences, 60% of patients preferred oral therapy and 22% injectable mediation; 81% preferred a therapy that can be taken at home.
CONCLUSIONS: Despite current pharmacotherapies, patients reported substantial burden due to acromegaly and its treatment, which extends beyond clinical manifestations to impact activities, productivity, and HCRU.
METHODOLOGY: A quantitative study was conducted among symptomatic adults with acromegaly receiving therapy, including depot SRL injections. A web-based survey captured the 3-month disease experience including the presence and severity of acromegaly-associated symptoms, treatment experience, HCRU, and impact on the ability to work (Work Productivity and Impairment Questionnaire [WPAI], assessed over prior 7 days).
RESULTS: Among 58 patients who completed the survey, 36 (62.1%) received a depot SRL injection, either as monotherapy (18 [50%]), or in combination with other agents. All patients experienced ≥ 1 symptom during the previous 3 months, with 67% having ≥ 1 symptom with a severity of ≥ 8 on a scale of 0 to 10. Patients reported that acromegaly had a moderate (59%) or high (22%) level of interference in their life overall. Of 18 patients on depot SRL injection monotherapy, 12 (67%) reported ≥ 1 breakthrough acromegaly symptom at any time prior to the next injection. WPAI scores were 51% for daily activity impairment, 38% for presenteeism, 34% for overall work impairment, and 8% for absenteeism. Patients receiving depot SRL injection (monotherapy/combination therapy) were more likely than those not receiving this injection to report moderate-to-high interference of acromegaly treatment with their life (56%/72% vs. 18%) and that their treatment was moderately to highly burdensome (67%/72% vs. 41%). HCRU due to acromegaly treatment included: a mean of 2.6 office visits, 13.8% with ≥ 1 emergency department visit, and 10.3% with ≥ 1 overnight hospitalization. When asked about treatment preferences, 60% of patients preferred oral therapy and 22% injectable mediation; 81% preferred a therapy that can be taken at home.
CONCLUSIONS: Despite current pharmacotherapies, patients reported substantial burden due to acromegaly and its treatment, which extends beyond clinical manifestations to impact activities, productivity, and HCRU.