Dos Santos Venda Nova, Carolina; (2023) Development of the Core Outcome Set to be used in Clinical Trials of Trigeminal Neuralgia. Doctoral thesis (Ph.D), UCL (University College London).

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Abstract

Introduction Trigeminal neuralgia (TN) is an excruciating unilateral facial pain, which can be managed medically and surgically. Due to the lack of standardized outcomes of treatment in the field, it has been difficult to compare the available treatments and to draw meaningful conclusions about their efficacy. Furthermore, patients have seldom been involved in TN research and outcomes of treatment should be meaningful to those most affected by TN. The aim of the present thesis was to reach consensus on what outcomes of treatment are important to different TN stakeholders (patients, clinicians, and researchers), and to develop the TN Core Outcome Set (COS) to be used in future clinical trials. Methodology Mixed methods were used to achieve this thesis’ aim. Two systematic reviews (SR) were conducted to (1) identify what outcomes have been used to date, and (2) to investigate the psychometric performance of patient reported outcomes (PROMs). Focus group (FG) work with TN patients identified outcomes that mattered most to them. Secondary analysis of the SR data and qualitative data analysis of the FG work were used to develop a list of outcomes to be presented to the different stakeholders during consensus processes. A three-round Delphi survey was conducted to prioritise the identified outcomes. It involved patients, clinicians, and researchers. Participants were asked to score the outcomes on scale from 1 to 9 (1– 3 not important;4– 6 important but not critical;7– 9 critical). Outcomes scored as critical by ≥70% and not important by <15% were retained. Those for which no consensus was reached were discussed at a consensus meeting, where the final COS was decided. Results Forty outcomes identified from the SR and FG work were presented during the Delphi survey. Of the 70 participants who completed the Delphi, 26 were patients, 38 were clinicians and six were researchers. Seventeen outcomes were scored as critical, and no consensus was met for 23 outcomes. Agreement was reached during a consensus meeting on 11 outcomes across six domains (pain, side effects, social impact, quality of life, global improvement, and satisfaction with treatment). Of the PROMs identified in the SR, only the Penn Facial Pain Scale Revised (PFPS-R) demonstrated moderate quality evidence for sufficient content validity. Conclusion The findings of the present thesis led to the development of an 11-item COS for TN clinical trials, through a partnership between patients, clinicians, and researchers. Implementation of the TN COS will contribute to improving data collection in future trials. Study results will be more homogeneous which will effectively allow comparison of different treatments to better inform researchers, clinicians and most importantly patients, about the effectiveness of the different treatments. Further work is needed to identify which PROMs to use with each of the 11 outcome domains.

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