Janssen Global Services, LLC, Horsham, PA, USA.
RTI Health Solutions, Research Triangle Park, NC, USA.
J Patient Rep Outcomes. 2023 Dec 18;7(1):134. doi: 10.1186/s41687-023-00673-w.
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).
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.
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.
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.
除了疗效之外，了解患者对药物耐受性的看法可以全面了解患者的体验，并支持更明智的治疗决策。国家癌症研究所患者报告结局版常见不良事件术语标准（PRO-CTCAE）的项目库是为了衡量与癌症治疗相关的不良事件（AE）的患者报告频率、严重程度和干扰程度而开发的。这项定性访谈研究评估了从 PRO-CTCAE 库中选择的项目是否适合评估用于治疗肺动脉高压（PAH）的塞立生帕的耐受性。
对最近服用口服塞立生帕治疗 PAH 的患者进行了两轮各 10 次的基于网络的电话定性访谈，访谈遵循半结构化指南。每次访谈均包括概念引出，以收集参与者对症状性 AE（类型、频率、严重程度和干扰）的看法，并对 PRO-CTCAE 中涉及口服塞立生帕最常报告的 AE 的项目进行认知审查。
共对 20 名 PAH 患者（平均[范围]年龄 50[24-68]岁；75%为女性；85%为世界卫生组织功能分类 II-III）进行了访谈，他们来自不同种族/民族、教育程度和就业状况。15 名参与者目前正在接受塞立生帕治疗；5 名参与者在停止治疗前至少服用了 6 个月的塞立生帕。报告频率最高的 AE 包括头痛、下颌痛和恶心（分别有 15、12 和 10 名参与者报告）。腹泻和头痛分别被 5 名和 4 名参与者认为是最困扰的 AE。一些 AE 是短暂的（例如，下颌痛）；另一些则是长期的（例如，肌肉疼痛）。基于第一轮访谈的结果，增加了潮红项目，并修改了 PRO-CTCAE 一般疼痛项目，使其专门针对下颌疼痛，用于第二轮测试。访谈结果确定了与评估 PAH 临床试验中相关的以下 AE：恶心、呕吐、腹泻、潮红、下颌痛、头痛、肌肉疼痛和关节疼痛。
本研究中选择的 PRO-CTCAE 项目和确定的其他与症状相关的 AE 具有潜在的可能性，可用于评估塞立生帕和可能的其他 PAH 治疗药物的耐受性方面的患者观点。
