背景:了解患者对药物耐受性的看法,除了有效性,提供患者体验的完整图片,并支持更明智的治疗决策。美国国家癌症研究所的患者报告结果版本的不良事件通用术语标准(PRO-CTCAE)的项目库被开发用于测量患者报告的频率,严重程度,和干扰与癌症治疗相关的不良事件(AE)。这项定性访谈研究评估了从PRO-CTCAE库中选择的项目的适用性,以评估selexipag的耐受性,针对肺动脉高压(PAH)患者的前列环素途径的药物。
方法:两轮共10次定性,遵循半结构化指南的网络辅助电话采访是在最近有经验的个人中进行的。每次访谈都包括概念启发,以收集参与者对症状性AE的观点(类型,频率,严重程度,和干扰)和PRO-CTCAE项目的认知汇报,解决了最常报告的口腔自语组AE。
结果:对20名患有PAH的参与者进行了访谈(平均年龄[范围]50[24-68]岁;75%为女性;世界卫生组织功能类II-III为85%),包括不同的种族/民族,教育水平,和就业状况。目前有15名参与者接受了selexipag治疗;在中止之前,有5人服用了selexipag≥6个月。最常报告的不良事件包括头痛,下巴疼痛,和恶心(n=15、12和10名参与者,分别)。腹泻和头痛被5和4名参与者确定为最麻烦的AE。分别。一些AE是暂时性的(例如,颌骨疼痛);其他人是持久的(例如,肌肉疼痛)。根据第一轮访谈的结果,在第2轮测试中,增加了冲洗项目,并修改了PRO-CTCAE一般疼痛项目,使其专门针对颌骨疼痛.访谈结果确定了以下与PAH临床试验评估相关的AE:恶心,呕吐,腹泻,冲洗,下巴疼痛,头痛,疼痛的肌肉,关节疼痛。
结论:本研究中选择的PRO-CTCAE项目和确定为患者相关的其他有症状的AE有可能被纳入评估中,在未来的selexipag和可能的其他PAH疗法研究中,了解患者对耐受性的看法。
BACKGROUND: Understanding patients\' perspectives regarding drug tolerability, in addition to effectiveness, provides a complete picture of the patient experience and supports more informed therapeutic decision-making. The item library of the National Cancer Institute\'s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) was developed to measure patient-reported frequency, severity, and interference of adverse events (AEs) associated with cancer therapies. This qualitative interview study assessed the suitability of items selected from the PRO-CTCAE library for assessing tolerability of selexipag, a medication targeting the prostacyclin pathway for patients with pulmonary arterial hypertension (PAH).
METHODS: Two rounds of 10 qualitative, web-assisted telephone interviews following a semi-structured guide were conducted in individuals with recent experience taking oral selexipag for PAH. Each interview included concept elicitation to gather participants\' perspectives on symptomatic AEs (type, frequency, severity, and interference) and cognitive debriefing of PRO-CTCAE items addressing the most frequently reported AEs of oral selexipag.
RESULTS: Interviews were conducted with 20 participants with PAH (mean [range] age 50 [24-68] years; 75% female; 85% in World Health Organization Functional Class II-III), comprising different races/ethnicities, levels of education, and employment status. Fifteen participants were currently treated with selexipag; five had taken selexipag for ≥ 6 months before discontinuing. The most frequently reported AEs included headache, jaw pain, and nausea (n = 15, 12, and 10 participants, respectively). Diarrhea and headache were identified as the most bothersome AEs by 5 and 4 participants, respectively. Some AEs were transitory (e.g., jaw pain); others were long-lasting (e.g., muscle pain). Based on findings from Round 1 interviews, a flushing item was added and the PRO-CTCAE general pain item was modified to be specific to jaw pain for testing in Round 2. Interview findings identified the following AEs as relevant to assess in a PAH clinical trial: nausea, vomiting, diarrhea, flushing, jaw pain, headache, aching muscles, and aching joints.
CONCLUSIONS: The PRO-CTCAE items selected in this study and the additional symptomatic AEs identified as patient-relevant have the potential to be included in assessments capturing the patient perspective on tolerability in future studies of selexipag and possibly other PAH therapies.