PMID- 38108945 OWN - NLM STAT- MEDLINE DCOM- 20231219 LR - 20231221 IS - 2509-8020 (Electronic) IS - 2509-8020 (Linking) VI - 7 IP - 1 DP - 2023 Dec 18 TI - Use of the National Cancer Institute Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events to assess treatment tolerability in pulmonary arterial hypertension: qualitative patient research findings in current and former users of oral selexipag. PG - 134 LID - 10.1186/s41687-023-00673-w [doi] LID - 134 AB - 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. CI - (c) 2023. The Author(s). FAU - Davis, Stacy AU - Davis S AD - Janssen Global Services, LLC, Horsham, PA, USA. sdavis26@its.jnj.com. FAU - Edwards, Teresa AU - Edwards T AD - RTI Health Solutions, Research Triangle Park, NC, USA. FAU - Norcross, Lindsey AU - Norcross L AD - RTI Health Solutions, Research Triangle Park, NC, USA. FAU - Fehnel, Sheri AU - Fehnel S AD - RTI Health Solutions, Research Triangle Park, NC, USA. FAU - Beaudet, Amelie AU - Beaudet A AD - Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, Allschwil, Switzerland. FAU - Eckart, Marie AU - Eckart M AD - Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, Allschwil, Switzerland. FAU - Fastenau, John AU - Fastenau J AD - Janssen Global Services, LLC, Horsham, PA, USA. LA - eng GR - Not applicable/Janssen Pharmaceuticals, Inc./ GR - Not applicable/Actelion Pharmaceuticals Ltd/ PT - Journal Article DEP - 20231218 PL - Germany TA - J Patient Rep Outcomes JT - Journal of patient-reported outcomes JID - 101722688 RN - 5EXC0E384L (selexipag) SB - IM MH - United States MH - Humans MH - Female MH - Middle Aged MH - Male MH - *Pulmonary Arterial Hypertension MH - National Cancer Institute (U.S.) MH - Familial Primary Pulmonary Hypertension MH - Pain MH - Diarrhea MH - Headache/chemically induced MH - Nausea MH - *Neoplasms PMC - PMC10728389 OTO - NOTNLM OT - Adverse events OT - PRO-CTCAE OT - Patient-reported outcomes OT - Pulmonary arterial hypertension OT - Selexipag OT - Side effects OT - Tolerability COIS- SD is an employee of Janssen Pharmaceuticals, Inc. At the time of this research, JF was an employee of Janssen. AB is an employee of Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson. At the time of this research, ME was an employee of Actelion. TE, LN, and SF are employees of RTI Health Solutions, which received funding from Janssen for conducting this research. EDAT- 2023/12/18 12:41 MHDA- 2023/12/19 06:41 PMCR- 2023/12/18 CRDT- 2023/12/18 11:09 PHST- 2023/08/02 00:00 [received] PHST- 2023/12/07 00:00 [accepted] PHST- 2023/12/19 06:41 [medline] PHST- 2023/12/18 12:41 [pubmed] PHST- 2023/12/18 11:09 [entrez] PHST- 2023/12/18 00:00 [pmc-release] AID - 10.1186/s41687-023-00673-w [pii] AID - 673 [pii] AID - 10.1186/s41687-023-00673-w [doi] PST - epublish SO - J Patient Rep Outcomes. 2023 Dec 18;7(1):134. doi: 10.1186/s41687-023-00673-w.