Broderick Lynne, Foster April, Waldman Laura Tesler, Jacobs K D, Bordone Laura, Yarlas Aaron
QualityMetric, an IQVIA business, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USA.
Ionis Pharmaceuticals, Inc, 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
J Patient Rep Outcomes. 2025 Apr 12;9(1):42. doi: 10.1186/s41687-025-00876-3.
There is a lack of clear evidence pointing to a fit-for-purpose instrument to measure impacts on health-related quality of life (HRQoL) in adult and adolescent patients with hereditary angioedema (HAE). The purpose of this study was to determine whether the Angioedema Quality of Life Questionnaire (AE-QoL) is content valid and appropriate for capturing the impact of HAE attacks on HRQoL in both adults and adolescents with HAE.
This study used one-on-one, audio-recorded, cognitive debriefing interviews employing think-aloud and verbal probing approaches to evaluate the relevance, comprehensibility, and comprehensiveness of the AE-QoL in this patient population. All data were quality checked then coded and analyzed using inductive and deductive approaches.
This study included 10 adolescents and 12 adults with HAE in the United States. Overall, participants had positive impressions of the AE-QoL, finding the length and recall period appropriate, and the response options clear and easy to understand. Some reported minor concerns with the instructions, but none that prevented them from completing the instrument. Participants found the instrument relevant to their experiences with HAE, noting that items that were not personally relevant were still important to ask. Overall, participants found the AE-QoL comprehensible and comprehensive, although some participants, primarily adolescents, reported being unfamiliar with the word "leisure," making it difficult to answer the item asking about impact of attacks on "leisure time." Adolescents also reported that questions about school-related impacts were missing and would be important to ask about specifically.
This study presents evidence that supports the content validity of the AE-QoL in adult and adolescent patients with HAE. While revisions could be considered prior to using the instrument with samples of adolescent patients with HAE, in general, adolescents and adults with HAE found the measure relevant, comprehensive, and comprehensible.
缺乏明确证据表明存在一种适用于测量遗传性血管性水肿（HAE）成年和青少年患者健康相关生活质量（HRQoL）影响的适用工具。本研究的目的是确定血管性水肿生活质量问卷（AE-QoL）在内容上是否有效，以及是否适合用于捕捉HAE发作对成年和青少年HAE患者HRQoL的影响。
本研究采用一对一、录音的认知反馈访谈，运用出声思考和言语探查方法，评估AE-QoL在该患者群体中的相关性、可理解性和全面性。所有数据经过质量检查后，使用归纳和演绎方法进行编码和分析。
本研究纳入了美国10名青少年和12名成年HAE患者。总体而言，参与者对AE-QoL有积极印象，认为问卷长度和回忆期合适，回答选项清晰易懂。一些人报告对说明有轻微担忧，但都不妨碍他们完成该问卷。参与者发现该问卷与他们的HAE经历相关，并指出一些与个人无关的问题仍然值得询问。总体而言，参与者认为AE-QoL易于理解且全面，不过一些参与者，主要是青少年，报告不熟悉“休闲”一词，这使得他们难以回答关于发作对“休闲时间”影响的问题。青少年还报告说缺少关于学校相关影响的问题，专门询问这些问题会很重要。
本研究提供的证据支持AE-QoL在成年和青少年HAE患者中的内容效度。虽然在将该问卷用于青少年HAE患者样本之前可以考虑进行修订，但总体而言，成年和青少年HAE患者认为该测量方法相关、全面且易于理解。
