Navigating Pathways to Diagnosis in Idiopathic Subglottic Stenosis: A Qualitative Study.

OBJECTIVE: Idiopathic subglottic stenosis is a rare disease, and time to diagnosis is often prolonged. In the United States, some estimate it takes an average of 9 years for patients with similar rare disease to be diagnosed. Patient experience during this period is termed the diagnostic odyssey. The aim of this study is to use qualitative methods grounded in behavioral-ecological conceptual frameworks to identify drivers of diagnostic odyssey length that can help inform efforts to improve health care for iSGS patients. METHODS: Qualitative study using semi-structured interviews. Setting consisted of participants who were recruited from those enrolled in a large, prospective multicenter trial. We use directed content analysis to analyze qualitative semi-structured interviews with iSGS patients focusing on their pathways to diagnosis. RESULTS: Overall, 30 patients with iSGS underwent semi-structured interviews. The patient-reported median time to diagnosis was 21 months. On average, the participants visited four different health care providers. Specialists were most likely to make an appropriate referral to otolaryngology that ended in diagnosis. However, when primary care providers referred to otolaryngology, patients experienced a shorter diagnostic odyssey. The most important behavioral-ecological factors in accelerating diagnosis were strong social support for the patient and providers' willingness to refer. CONCLUSION: Several factors affected time to diagnosis for iSGS patients. Patient social capital was a catalyst in decreasing time to diagnosis. Patient-reported medical paternalism and gatekeeping limited specialty care referrals extended diagnostic odysseys. Additional research is needed to understand the effect of patient-provider and provider-provider relationships on time to diagnosis for patients with iSGS. LEVEL OF EVIDENCE: 4 Laryngoscope, 134:815-824, 2024.

Multicenter Development and Validation of the Vocal Cord Paralysis Experience (CoPE), a Patient-Reported Outcome Measure for Unilateral Vocal Fold Paralysis-Specific Disability.

Importance: Unilateral vocal fold paralysis (UVFP) is a common and life-changing complication of cancer, trauma, and an estimated 500 000 head, neck, and chest surgeries performed annually in the US, among other causes (eg, idiopathic). Consequent disabilities are profound and often permanent and can include severe voice, swallowing, and breathing dysfunction and concomitant anxiety, isolation, and fear. Physiological measures often correlate poorly with patient-reported disability. The measure described herein was designed to be a comprehensive, psychometrically sound UVFP-specific patient-reported outcome measure (PROM) for use in clinical trials or at point of care. Objective: To evaluate the reliability and validity of the CoPE (vocal Cord Paralysis Experience) PROM in a nationally representative sample for both clinical and research use. Design, Setting, and Participants: This survey validation study was performed at 34 tertiary care centers across the US and included English-speaking adults with unilateral vocal fold immobility confirmed via laryngoscopy. Main Outcomes and Measures: Reliability (internal consistency, alternate form, and test-retest) and validity (convergent and known-group). Results: In total, 613 patients (mean [SD] age, 58 [15.3] years; 394 [64.5%] women) were recruited, and 555 (92.3%) completed surveys for all time points. Internal consistency was high in the overall 22-item PROM and psychosocial, swallow, and voice subscales (Cronbach α > 0.91). Intraclass correlations for individuals between the baseline and 2-week administrations were moderate for the overall score and subscales (intraclass correlations range, 0.66-0.80). There were significant differences between the online and 2-week paper administrations for the overall score and voice and psychosocial subscales (overall scale mean: 54.4 [95% CI, 49.7-59.1] vs 48.9 [95% CI, 43.7-54.0] at 2 weeks). The confirmatory model was found to be suitably fitted based on average r2 values 0.5 or greater for subscale and overall scores. Correlations between subscales and existing PROMs (Voice-Related Quality of Life, Eating Assessment Tool, and Communication Participation Item Bank) were all greater than 0.69, and mean PROM subscale scores were significantly different across known quartiles of existing PROMs. Conclusions and Relevance: The findings of this survey validation study suggest that the CoPE PROM could serve as a psychometrically sound, comprehensive measure of UVFP-attributed disability suitable for use in clinical and research settings to assess within-person changes. The results will inform a user manual to facilitate use in clinical trials comparing the effectiveness and durability of treatments including behavioral (speech therapy), temporary (eg, injection augmentation), and permanent surgical treatments for UVFP.

Study protocol to develop a patient-reported outcome measuring disability associated with unilateral vocal fold paralysis: a mixed-methods approach with the CoPE collaborative.

INTRODUCTION: Patient-reported outcome (PRO) measures are increasingly developed with multisite, representative patient populations so that they can serve as a primary endpoint in clinical trials and longitudinal studies. Creating multisite infrastructure during PRO measure development can facilitate future comparative effectiveness trials. We describe our protocol to simultaneously develop a PRO measure and create a collaborative of tertiary care centres to address the needs of patients with unilateral vocal fold paralysis (UVFP). We describe the stakeholder engagement, information technology and regulatory foundations for PRO measure development and how the process enables plans for multisite trials comparing treatments for this largely iatrogenic condition. METHODS AND ANALYSIS: The study has three phases: systematic review, measure development and measure validation. Systematic reviews and qualitative interviews (n=75) will inform the development of a conceptual framework. Qualitative interviews with patients with UVFP will characterise the lived experience of the condition. Candidate PRO measure items will be derived verbatim from patient interviews and refined using cognitive interviews and expert input. The PRO measure will be administered to a large, multisite cohort of adult patients with UVFP via the CoPE (vocal Cord Paralysis Experience) Collaborative. We will establish CoPE to facilitate measure development and to create preliminary infrastructure for future trials, including online data capture, stakeholder engagement, and the identification of barriers and facilitators to participation. Classical test theory psychometrics and grounded theory characterise our approach, and validation includes assessment of latent structure, reliability and validity. ETHICS AND DISSEMINATION: Our study is approved by the University of Wisconsin Health Sciences Institutional Review Board. Findings from this project will be published in open-access journals and presented at international conferences. Subsequent use of the PRO measure will include comparative effectiveness trials of treatments for UVFP at CoPE Collaborative sites.