Background <p>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).</p> Methodology <p>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).</p> Results <p>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.</p> Conclusions <p>Despite current pharmacotherapies, patients reported substantial burden due to acromegaly and its treatment, which extends beyond clinical manifestations to impact activities, productivity, and HCRU.</p>

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Real-world burden of disease, treatment, and healthcare resource utilization in acromegaly: a quantitative survey of patient experiences

  • Maxwell Koobatian,
  • Jill Sisco,
  • Janetricks C. Okeyo,
  • Alan Krasner,
  • Tiffany P. Quock

摘要

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.