Preferences for different treatment options among people living with acromegaly in the US.

OBJECTIVE: Acromegaly is a rare hormonal disorder that results in enlargement of the face, hands and feet. It is associated with comorbidities, increased mortality, reduced quality of life and economic burden. Adequate treatment is critical to alleviate symptoms; however, the treatment burden is substantial. To understand how different treatment aspects might ease the burden, this study investigated preferences for treatment options among people with acromegaly in the US, using a choice experiment (CE). METHODS: An online CE was conducted based on a similar study among the US general population. Respondents were recruited through Acromegaly Community in the US, and all eligible respondents were included. The survey assessed six treatment options that varied according to administration, frequency, storage, treatment setting, needle type and injection pain. RESULTS: 109 adults with acromegaly completed the survey between October and December 2022. On average, the population had lived with symptoms for 15 years, while the average number of years with the diagnosis was eight. Respondents preferred subcutaneous injections every fourth week and preferred them at home rather than at the hospital. Most respondents preferred subcutaneous injections right under the skin administered once every fourth week with a pen at home over oral capsules administered twice daily at home. CONCLUSION: The results indicate that new treatment options administered as subcutaneous injections right under the skin once every fourth week at home have the potential to lower the treatment burden among people with acromegaly, emphasizing the importance of taking each person's preference into consideration when choosing treatment.

Pituitary adenoma or neuroendocrine tumour: the need for an integrated prognostic classification.

In the 2022 fifth edition of the WHO Classification of Endocrine Tumours and of Central Nervous System Tumours, pituitary adenomas are reclassified as neuroendocrine tumours (NETs). This change confers an oncology label to neoplasms that are overwhelmingly benign. A comprehensive clinical classification schema is required to guide prognosis, therapy and outcomes for all patients with pituitary adenomas. Pituitary adenomas and NETs exhibit some morphological and ultrastructural similarities. However, unlike NETs, pituitary adenomas are highly prevalent, yet indolent and rarely become malignant. This Perspective presents the outcomes of an interdisciplinary international workshop that addressed the merit and clinical implications of the classification change of pituitary adenoma to NET. Many non-histological factors provide mechanistic insight and influence the prognosis and treatment of pituitary adenoma. We recommend the development of a comprehensive classification that integrates clinical, genetic, biochemical, radiological, pathological and molecular information for all anterior pituitary neoplasms.

Towards an Earlier Diagnosis of Acromegaly and Gigantism.

Acromegaly is a rare disease and the clinical features of acromegaly develop insidiously; its diagnosis is often significantly delayed. Therefore, earlier diagnosis will improve the quality of life of the patient and reduce the need for other therapies to control the initial and ongoing damage that acromegaly presents. In this chapter, we describe the view of the patient and the clinician on the importance of earlier diagnosis, as well as on what can be done to speed up this process. Earlier diagnosis will not only improve quality of life and the burden of disease in acromegaly patients, but it will also have a positive impact in the economic burden of this rare disease.

Patient reported outcome data from acromegaly patients treated with injectable somatostatin receptor ligands (SRLs) in routine clinical practice.

BACKGROUND: Acromegaly patients managed on Somatostatin receptor ligands (SRLs), the most common first-line pharmacotherapy for acromegaly, may still experience acromegaly symptoms such as headache, sweating, fatigue, soft tissue swelling, and joint pain, even those with normal IGF-1. Additionally, treatment with SRLs may cause injection site reactions and other side effects such as gastro-intestinal (GI) symptoms. This study utilized patient-reported outcome measures to examine the burden associated with acromegaly and its treatment for patients receiving a stable dose of long-acting SRLs in routine clinical practice. METHODS: US acromegaly patients on a stable dose of SRL seen by their treating healthcare provider in the past 12 months completed a one-time online survey including the Acro-TSQ, an acromegaly-specific tool for assessing symptom burden and treatment satisfaction and convenience. RESULTS: One hundred five patients were enrolled (mean age 49.9 years, 79.1% female). Patients experienced numerous symptoms, including > 80% who experienced joint pain, acro-fog, swelling of soft tissue, and fatigue/weakness. Many symptoms occurred constantly, while some occurred at the end of the injection cycle, even among those with IGF-1 < = 1.0 ULN. Injection site reactions were common. Patients were moderately satisfied with their current treatment; symptoms and side effects often affected daily activities. On average, patients reported > 3 acromegaly provider visits/year. CONCLUSIONS: Despite receiving a stable dose of SRL and regular visits with an acromegaly healthcare provider, US acromegaly patients in routine clinical practice, and even the subgroup with normal IGF-1, report significant burden of disease and treatment.

An evaluation of the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) in adult patients with acromegaly, including correlations with other patient-reported outcome measures: data from two large multicenter international studies.

PURPOSE: The Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) is a new patient-reported outcome (PRO) measure for patients with acromegaly receiving injectable somatostatin analogs (SSAs) to assess clinical symptoms and adverse drug reaction interference, treatment satisfaction, and convenience. We evaluated its scale structure, reliability, validity, responsiveness, and what constitutes clinically meaningful change. METHODS: Data from two longitudinal studies (N = 79 and 82) of patients receiving a stable injectable SSA dose for ≥ 6 months who completed the Acro-TSQ and other collateral measures (e.g., AcroQoL, AIS, WPAI:SHP, EQ-5D-5L) were analyzed. RESULTS: The first study demonstrated internal consistency of the Acro-TSQ. However, several items had high ceiling effects, responsiveness could not be established, and the minimally important difference (MID) was not estimable. In the second study, factor analysis revealed six scales: Symptom Interference, Treatment Convenience, Injection Site Interference, GI Interference, Treatment Satisfaction, and Emotional Reaction. Internal consistency and test-retest reliability were confirmed; most scales demonstrated significant differences in mean scores by disease severity. Correlations between Acro-TSQ scales and other collateral measures exceeded 0.30 in absolute value, confirming convergent validity. Responsiveness in Acro-TSQ scale scores reflected improved disease control. The MID was estimated for Symptom Interference (10-12 points), Treatment Convenience (9-11) and GI Interference (8-10). CONCLUSIONS: The Acro-TSQ is a brief, yet comprehensive tool to monitor important outcomes associated with injectable acromegaly SSA treatments. Its content reflects both disease and treatment burden as well as patient satisfaction, and its relevant for use in clinical studies.

Observed discordance between outcomes reported by acromegaly patients and their treating endocrinology medical provider.

BACKGROUND: Acromegaly patients, even those with IGF-1 values within the normal range receiving somatostatin receptor ligands (SRLs), often suffer from significant symptoms. It is not known to what extent patients' medical providers are aware of the frequency and severity of acromegaly symptoms or level of treatment satisfaction with SRLs. This study sought to examine the concordance between outcomes reported by acromegaly patients treated with long-acting SRLs and those perceived by their medical provider. METHODS: US acromegaly patients on a stable dose of SRL and seen by their medical provider in the past year completed an online survey which included the Acro-TSQ. Their medical providers were interviewed about the perception of their patient's symptoms, level of control, and general health, and completed relevant portions of the Acro-TSQ. Concordance between patient and medical provider reported data was examined. RESULTS: Medical providers reported that their patients experienced acromegaly symptoms on a regular basis, however, there was poor agreement between patients and medical providers on the frequency, severity, and pattern of symptoms, as well as on the severity of injection site reactions and multiple domains of the Acro-TSQ, with patients generally reporting symptoms and injection site reactions more often and with higher severity than medical providers. CONCLUSIONS: Medical providers were aware that their patients who were receiving a stable dose of SRL regularly experienced acromegaly symptoms. Addressing discordance in patient- and medical provider-reported frequency and severity of acromegaly symptoms and injection site reactions by facilitating better communication may improve care of acromegaly patients.

Development of a novel patient-reported measure for acromegaly: the Acro-TSQ.

PURPOSE: Somatostatin analogs (SSAs) represent a mainstay of medical treatment for acromegaly, currently available as either intramuscular or deep subcutaneous injections. Patient-reported outcomes (PROs) are increasingly common as relevant outcomes in studies of acromegaly and its treatment, but there are no validated PRO measures available that focus on the disease burden and the impact of treatment, specifically designed for use in patients with acromegaly. We sought to develop a new and unique PRO measure, the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ). METHODS: Concept elicitation (CE) interviews were conducted with acromegaly patients in the United States receiving SSA injections at a stable dose for ≥ 6 months. A questionnaire was drafted based on these interviews; combined CE and cognitive debriefing (CE/CD) interviews were then conducted to confirm the content, clarity, and relevance of the questionnaire. RESULTS: Nineteen subjects completed interviews [n = 9 CE, n = 10 CE/CD; n = 15 Lanreotide Depot/Autogel (Somatuline), n = 4 Octreotide LAR (Sandostatin LAR)]. Most subjects responded positively when asked about the effectiveness of their current treatment; however, breakthrough symptoms, injection site reactions, and side effects were commonly reported and had negative impacts on social and emotional well-being and daily activities. All 10 subjects involved in debriefing interviews found the questionnaire to be relevant, easy to complete, and found the response options to be clear. The resulting 26-item Acro-TSQ covers symptoms and symptom control, gastrointestinal side effects and their impact on daily activities, the emotional impact of treatment, convenience and ease of use, and overall satisfaction. CONCLUSIONS: The Acro-TSQ is a novel PRO, focused on both disease burden and impact of treatment; it was found to be comprehensive, clear, and relevant for patients with acromegaly receiving injectable SSA treatment.