Measuring the patient experience of chronic rhinosinusitis with nasal polyposis: qualitative development of a novel symptom diary

Abstract

Background: This qualitative study assessed the experience of patients with chronic rhinosinusitis with nasal polyposis (NP) to inform the development of a novel symptom diary for clinical study use. Methods:Concept elicitation and cognitive interviews were conducted with patients who had a physician-verified diagnosis of NP and a history of intranasal corticosteroid use. Concepts were identified via open-ended and follow-up questions. Relative symptom/impact disturbance level was assessed using a scale of 0 (not at all disturbing) to 10 (extremely disturbing). Results: Patients (n = 30) attributed numerous symptoms and impacts to NP; the most prevalent and disturbing were nasal congestion (identified by 100% of patients; average disturbance rating = 7.9), nasal blockage/obstruction (97%; 8.2), difficulty with sense of smell (97%; 7.6), facial pressure (90%; 6.2), postnasal drip (87%; 6.5), runny nose (87%; 6.2), facial pain (80%; 6.3), and headache (77%; 6.5). These symptoms, along with the impact of NP on sleep and daily activities, were included in the Nasal Polyposis Symptom Diary (NPSD). Cognitive interviews confirmed that patients understood the NPSD items and could select a response reflective of their experience at its worst over the past 24 hours using a four-point scale (none, mild, moderate, or severe). Conclusion:The most relevant and disturbing symptoms, according to patients with NP, were included in the NPSD. Interviews confirmed the suitability of NPSD in capturing the daily experience of patients. These findings support the content validity of the NPSD as a suitable tool for capturing NP symptoms and impacts.

Psychometric Validation and Meaningful Change Thresholds of the New Nasal Polyposis Symptom Diary

“…Following regulatory guidelines, interviews of patients with CRSwNP established the content validity of this novel PRO tool. 10 The comprehensive psychometric analyses based on OSTRO data from over 500 screened patients demonstrated the reliability, construct validity, and ability to detect change in NPSD scores measuring single (NBS, NCS, and DSS) and combined (TSS) symptoms. Test-retest ICC values for NPSD scores ranged from 0.828 to 0.960, in line with values reported for other instruments such as the SNOT-22 test-retest reliability of 0.93 in patients with CRS.…”

“…7 However, these PRO measures were not specifically developed for patients with CRSwNP. 7 Recall periods in existing tools (eg, SNOT-22) might be too long to capture daily symptom variation, [8][9][10] potentially overlooking important day-to-day changes in symptoms induced by medical therapy. [11][12][13] A literature review of existing instruments indicated a gap for a novel, brief, daily PRO questionnaire developed specifically in CRSwNP to track changes in symptoms and impacts during clinical trials.…”

“…[11][12][13] A literature review of existing instruments indicated a gap for a novel, brief, daily PRO questionnaire developed specifically in CRSwNP to track changes in symptoms and impacts during clinical trials. 10 The Nasal Polyposis Symptom Diary (NPSD), a new, content-valid daily symptom questionnaire, was developed to capture PROs for use as efficacy endpoints to evaluate the clinical benefit of investigational therapy for patients with severe CRSwNP. 10 The NPSD assesses symptoms, symptom impact on HRQOL, and medication compliance.…”

“…10 The Nasal Polyposis Symptom Diary (NPSD), a new, content-valid daily symptom questionnaire, was developed to capture PROs for use as efficacy endpoints to evaluate the clinical benefit of investigational therapy for patients with severe CRSwNP. 10 The NPSD assesses symptoms, symptom impact on HRQOL, and medication compliance. In accordance with the US Food and Drug Administration guidance on PRO measures for use in medical product development to support labeling claims, 14 qualitative research indicated that the NPSD was easy to use and measured key relevant and disturbing symptoms from the perspective of patients with severe CRSwNP.…”

“…These scores were selected based on their prevalence in published literature [16][17][18][19][20] and their reported prevalence and relevance by patients with CRSwNP interviewed for the development of the NPSD. 10…”