Oluyase Adejoke O, Ghirotto Luca, Watson Harry, Costantini Massimo, Bajwah Sabrina, Normand Charles, Tanzi Silvia, Bazata Jeremias, Ryan Karen, Turola Elena, Higginson Irene J, Maddocks Matthew
Florence Nightingale Faculty of Nursing, Midwifery & Palliative Care, Cicely Saunders Institute of Palliative Care, Policy and Rehabilitation, King's College London, Bessemer Road, London, SE5 9PJ, UK.
Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy.
BMC Pulm Med. 2025 Aug 14;25(1):390. doi: 10.1186/s12890-025-03862-z.
Drug repurposing offers advantages over traditional drug development, such as shorter time and reduced costs. Understanding patient and caregiver perspectives on repurposed medicines is crucial to improving clinical trial design and conduct, especially in advanced disease. We carried out this study in the UK and Italy to explore the experiences and motivations of patients with respiratory diseases and their caregivers who participated in a trial investigating the repurposing of the antidepressant mirtazapine to alleviate severe breathlessness.
Qualitative study nested within a double-blind, placebo-controlled, randomised trial (BETTER-B: BETter TreatmEnts for Refractory Breathlessness). Purposive sampling ensured diversity in age and gender. Framework analysis was applied. Interviewed participants had Chronic Obstructive Pulmonary Disease (COPD) or Interstitial Lung Disease (ILD), experienced limiting breathlessness (grade 3 or 4 of the modified Medical Research Council breathlessness scale) and had participated in the trial.
Twenty-three patients (13 men and 10 women) and eight caregivers (4 men and 4 women) were interviewed. Of patients (15 COPD, 8 ILD), 22 had completed the trial and one had withdrawn due to adverse effects. Interviews were conducted at home or via the telephone. Two main themes were derived: (1) 'knowledge and views about antidepressants and its use' and (2) 'experience and views on joining the trial'. The patients' perceived need for relief from severe breathlessness and its impact outweighed any concerns about taking an antidepressant. Motivations for trial participation included the potential for benefit, altruism, trust in the healthcare system, and the strength of relationships between patients and healthcare professionals.
Participants willing to accept mirtazapine repurposed for another indication joined this trial regardless of their existing concerns about antidepressants. A clear explanation of trials and possible benefits, plus trust in professionals and the healthcare system, are instrumental in increasing trial participation for repurposed medicines.
ISRCTN Registry, ISRCTN10487976. Prospectively Registered on 19 November 2019.
药物重新利用相较于传统药物研发具有诸多优势，比如时间更短、成本更低。了解患者及护理人员对重新利用药物的看法对于改进临床试验设计与实施至关重要，尤其是在治疗晚期疾病时。我们在英国和意大利开展了此项研究，以探究参与一项试验的呼吸系统疾病患者及其护理人员的经历和动机，该试验旨在研究将抗抑郁药米氮平重新用于缓解严重呼吸困难。
本定性研究嵌套于一项双盲、安慰剂对照、随机试验（BETTER - B：难治性呼吸困难的更佳治疗）之中。目的抽样确保了年龄和性别的多样性。采用了框架分析法。接受访谈的参与者患有慢性阻塞性肺疾病（COPD）或间质性肺疾病（ILD），经历了受限的呼吸困难（改良医学研究理事会呼吸困难量表3级或4级）且参与了该试验。
共访谈了23名患者（13名男性和10名女性）及8名护理人员（4名男性和4名女性）。在患者中（15名COPD患者，8名ILD患者），22名完成了试验，1名因不良反应退出。访谈在患者家中或通过电话进行。得出了两个主要主题：（1）“关于抗抑郁药及其使用的知识和看法”以及（2）“参与试验的经历和看法”。患者认为缓解严重呼吸困难的需求及其影响超过了对服用抗抑郁药的任何担忧。参与试验的动机包括获益的可能性、利他主义、对医疗保健系统的信任以及患者与医疗专业人员之间关系的紧密程度。
愿意接受重新用于其他适应症的米氮平的参与者，不顾他们对现有抗抑郁药的担忧而加入了该试验。对试验及可能益处的清晰解释，加上对专业人员和医疗保健系统的信任，有助于提高对重新利用药物的试验参与度。
ISRCTN注册库，ISRCTN10487976。于2019年11月19日前瞻性注册。
