Development of a novel patient-reported measure for acromegaly: the Acro-TSQ
Development of a novel patient-reported measure for acromegaly: the Acro-TSQ
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DOI：
10.1007/s11102-019-00986-4
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发表时间：
2019-12-01
期刊：
影响因子：
3.8
通讯作者：
Mathias, Susan D.
中科院分区：
文献类型：
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作者：
Fleseriu, Maria;Fogelfeld, Leon;Mathias, Susan D.
目的生长抑素类似物(SSAs)是治疗肢端肥大症的主要药物，目前可用于肌肉注射或皮下深度注射。在肢端肥大症及其治疗的研究中，患者报告的结果(PRO)作为相关结果越来越常见，但目前还没有有效的PRO措施来关注疾病负担和治疗的影响，这是专门为肢端肥大症患者设计的。我们试图开发一种新的和独特的PRO测量，Acromegaly治疗满意度问卷(Acro-TSQ)。方法对在美国接受稳定剂量SSA注射的肢端肥大症患者进行概念引出(CE)访谈，持续6个月。在这些访谈的基础上起草了一份问卷；然后进行了CE和认知情况汇报(CE/CD)的联合访谈，以确认问卷的内容、清晰度和相关性。结果19名受试者完成了访谈[9例CE，10例CE/CD；15例兰诺素库/AutoGel(Somatuline)，4例奥曲肽LAR(Sandostatin LAR)]。当被问及目前治疗的有效性时，大多数受试者的回答都是积极的；然而，突破性症状、注射部位反应和副作用是常见的报告，并对社会和情绪健康以及日常活动产生负面影响。所有参与汇报访谈的10名受试者都认为问卷很相关，很容易完成，而且回答选项很清楚。由此产生的26个条目的Acro-TSQ涵盖了症状和症状控制、胃肠道副作用及其对日常活动的影响、治疗的情感影响、便利性和易用性以及总体满意度。结论Acro-TSQ是一种新的PRO，关注疾病负担和治疗的影响；对于接受可注射SSA治疗的肢端肥大症患者，Acro-TSQ被发现是全面、明确和相关的。
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.