慢性呼吸疾病臨床試驗潛在受試者參與試驗相關因素之探討

Abstract

背景：慢性呼吸疾病是全球主要的健康負擔之一，雖臨床試驗是發展創新治療的基礎，但受試者招募及遵從率常面臨許多挑戰。了解慢性呼吸疾病病人參與臨床試驗的決策過程，可提供改善受試者招募及提升遵從率的實證參考。本研究旨在探討影響慢性呼吸疾病病人參與臨床試驗的相關因素、知識現況與主觀經驗。 方法：本研究採量性質性混合方法設計，於台灣北部某醫學中心，針對慢性呼吸疾病病人進行調查及訪談。量性部分使用結構式問卷，包含基本資料、臨床試驗知識量表、聖喬治呼吸問卷(St. George’s Respiratory Questionnaire，SGRQ)、慢性呼吸道評估問卷(Chronic airway assessment test，CAAT)及病人健康狀況問卷-9 (The Patient Health Questionnaire -9，PHQ-9)。質性部分則對慢性呼吸疾病病人進行半結構式深度一對一訪談。 結果：量化分析結果顯示，慢性呼吸疾病病人的疾病生活品質(SGRQ/CAT分數)、憂鬱程度(PHQ-9分數)以及臨床試驗知識分數均與其是否參與試驗無顯著相關。但憂鬱症狀之「食慾不振」(p = 0.024)、「事物專注困難」(p = 0.005)、「自殺意念」(p = 0.024)、「覺得自己很糟」(p = 0.048)在參與試驗及未參與試驗的組別之間存在顯著差異。多元逐步線性迴歸分析結果指出當診斷為淋巴血管平滑肌增生症、自殺意念愈強烈、事物專注有困難時，參與試驗的意願會降低，而當潛在受試者的疲倦程度增加時，參與試驗的意願則會上升(Adjusted R2 = 0.337)。另一方面，質性訪談發現慢性呼吸疾病病人基於對研究團隊及醫療機構的信任，對臨床試驗的潛在風險與副作用表現出低度關切，而病人參加試驗動力包含個人健康需求、利他主義的價值實現以及醫療權威的外在推力共同構成，其中，「對研究團隊的信任」是極為重要的影響因素。 結論：根據本研究的發現，建議未來招募受試者時，應留意病人是否有食慾、專注力或情緒低落的嚴重困擾，除此之外，研究團隊應將資源重點投入於建立與維護長期的醫病關係，而非單向提供試驗資訊，以促進更高效且合乎倫理的臨床研究。

Background: Chronic respiratory diseases (CRDs) is a major global health burden. While clinical trials are fundamental to developing novel therapies, subject recruitment remains a significant challenge. Effective recruitment strategies depend on a deep understanding of the patient's decision-making process. This study aimed to explore the factors, knowledge status, and subjective experiences influencing patients with CRDs to participate in clinical trials. Methods: A mixed-methods design was used. The study was conducted at a medical center in northern Taiwan, recruiting patients with CRDs. Quantitative data were collected using structured questionnaires, including demographics, a 7-item clinical trial knowledge scale, the St. George's Respiratory Questionnaire (SGRQ), the Chronic Airway Assessment Test (CAAT), and the Patient Health Questionnaire-9 (PHQ-9). Qualitative data were obtained through semi-structured, in-depth interviews with CRDs. Results: Quantitative analysis results showed that for patients with chronic respiratory diseases, their quality of life (SGRQ/CAAT scores), depression level (PHQ-9 score), and 7-item knowledge score were not significantly correlated with their participation in clinical trials. However, significant differences were observed in specific depressive symptoms between participants and non-participants, including poor appetite (p = 0.024), difficulty concentrating (p = 0.005), suicidal ideation (p = 0.024), and feelings of worthlessness (p = 0.048). Stepwise multiple linear regression analysis indicated that having a diagnosis of lymphangioleiomyomatosis, stronger suicidal ideation, and concentration difficulties were associated with lower willingness to participate in clinical trials. In contrast, greater fatigue was positively associated with willingness to participate (Adjusted R² = 0.337). On the other hand, qualitative results revealed that patients with chronic respiratory diseases showed minimal concern about the potential risks or side effects of clinical trials, primarily due to their trust in the research team and medical institution. Motivations for participation were driven by personal health needs, a sense of altruism, and external influences from medical authorities, with trust in the research team emerging as a particularly influential factor. Conclusion: Based on the findings of this study, future recruitment efforts should carefully consider whether patients are experiencing significant issues with appetite, concentration, or mood. In addition, rather than relying solely on one-way communication of trial information, research teams are encouraged to invest in establishing and sustaining long-term relationships with CRDs patients to facilitate more effective and ethically grounded clinical research.