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.

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.

Treatment Pathways and Health Outcomes of German Patients with Chronic Graft-Versus-Host Disease After Allogeneic Hematopoietic Cell Transplantation: A Retrospective Health Claims Data Analysis.

BACKGROUND: Although chronic graft-versus-host-disease (cGvHD) is an important long-term complication after allogenic hematopoietic cell transplantation (allo-HCT) and is associated with increased healthcare resource utilization, real-world evidence is scarce. OBJECTIVES: The aim of the study was to evaluate survival of patients with cGvHD in Germany and to analyze hospitalization and treatment patterns. PATIENTS AND METHODS: Based on a German claims database with 4.9 million enrollees, a retrospective longitudinal analysis covering a 6-year period between 2013 and 2018 was conducted. Patients with allo-HCT in 2014 or 2015 (index date) and no record of transplantation or documentation of GvHD 365 days prior to index were included. Patients who subsequently developed a cGVHD were compared with those who did not develop a cGVHD within 3 years after index date. cGVHD cases were identified based on documented International Classification of Diseases, Tenth Revision (ICD-10) diagnosis and treatment algorithms. Since the onset of cGvHD is defined at 100 days after allo-HCT, only those alive beyond day 100 were considered in the survival analysis. Patients who did not survive the first 100 days after allo-HCT were censored to prevent a selection bias due to early mortality within patients without GvHD. Survival rates were plotted using the Kaplan-Meier estimator. The number of hospitalizations and average lengths of stay as well as treatment patterns were descriptively examined. RESULTS: Overall, 165 cGvHD patients were identified and compared with 43 patients without cGVHD. Short-term survival rates were better for patients with cGvHD; the 6-month survival probability was 95.8% for patients with cGVHD and 83.7% for patients without cGVHD. However, long-term survival was better in patients without GvHD; The 30-month survival probability was 65.5% for patients with cGVHD and 76.7% for patients without cGVHD. While overall 90% of cGvHD patients were hospitalized at least once, the share was only half for patients without GvHD (44%). 78.2% of patients with cGVHD received corticosteroids in combination with other predefined immunosuppressants. CONCLUSION: Findings from this study reveal a high disease burden associated with cGvHD. This underlines the high medical need for new interventional strategies to improve survival and morbidity after allo-HCT.