Background
Oral Submucous Fibrosis (OSF) significantly impacts oral function, quality of life and poses challenges for effective treatment evaluation due to heterogeneity in outcome reporting across clinical trials. To date, no core outcome set (COS) exists for OSF trials, leading to significant heterogeneity in outcome reporting and difficulty in comparing interventions. This study aimed to develop a COS for OSF (OSFCOS) to standardize outcome reporting in clinical trials evaluating OSF management.
Methods
The study followed COMET and COS-STAD guidelines and involved four stages: a systematic review of randomized controlled trials to identify reported outcomes; qualitative research using interviews and focus groups with OSF patients to capture patient-prioritized outcomes; a two-round Delphi survey with key stakeholders (patients, clinicians, researchers); and a consensus meeting to finalize core outcomes. Outcomes were rated using a 9-point GRADE scale, and consensus was predefined using standard thresholds.
Results
A total of 20 outcomes were identified from systematic review and 31 from qualitative analysis. After triangulation & refinement, 16 unique outcomes were prioritized in Delphi Round 1 (n = 45). Delphi Round 2 (n = 25) rated 23 outcomes; 10 reached “consensus in.” A consensus meeting with 17 stakeholders reviewed 13 outcomes and finalized 7 core outcomes for OSFCOS.
Conclusion
This is the first COS developed specifically for OSF that offers a robust, stakeholder-driven framework for standardized outcome reporting, comparability and enhancing data synthesis in OSF trials for better evidence generation and patient care.
The WHO Collaborating Center for Oral Cancer has classified oral submucous fibrosis (OSF) as an oral potentially malignant disorder that is associated with betel quid chewing and clinically characterized by progressive fibrosis of oral mucosa. OSF manifests as limited mouth opening, burning sensation, palpable fibrous bands, loss of elasticity of mucosa, blanching of mucosa, restricted tongue movements, and shrunken or bending of uvula. Apart from these, loss of taste sensation, hearing, and speech difficulties with differing degrees of severity have also been reported in the literature. Although many therapeutic interventions have been tried in past, none of them proved to be a superior option for the holistic management of OSF symptoms. Therefore, at present, there is considerable controversy over the standardized treatment of OSF.
Recent systematic reviews observed a lack of high-quality evidence regarding the management of OSF. This could be due to the use of poorly standardized outcomes or lack of uniform outcomes and clinical indices that are used within trials comparing the effectiveness of therapeutic methods. Even in high-quality trials, the application of such heterogeneous outcome measures or measurement methods can jeopardize the comparison of these trials’ findings and avert revealing the therapeutic benefits of a particular intervention with any certainty. Furthermore, such heterogeneity might limit the evidence synthesis and prevents the pooling of study data for systematic review or meta-analysis. Consequently, uptake of new evidence is not communicated throughout the scientific community effectively and the highest standard of care is not delivered to the patients in today’s era of evidence-based clinical practice.
Despite few studies have addressed issues of diverse outcomes in trials of OSF, no precise research has been focused on furnishing solutions to date. The problems of heterogeneous outcome measures in OSF can be tackled by systematically establishing a core outcome set (COS) representing a set of minimum agreed, standardized outcomes and reporting in all clinical trials of OSF treatment. The COS can be developed by reaching to a consensus on a standardized outcome set by utilizing accepted outcome measures for OSF. A lack of consensus regarding clinician and patient reported outcomes in the OSF literature has been highlighted in several publications. Moreover, it is important to stress that no COS is available for OSF till date. To harmonize outcome measures for OSF in clinical practice and research, future necessary steps and consensus meeting is the need of the hour to create a consensus on terminology and to define core outcome measures. Developing a COS will encourage researchers/trialists to improve the design, conduct, reporting and comparisons between trials on OSF. Therefore, the development of a COS for OSF would facilitate meta-analysis leading to the establishment of a more robust evidence (i.e. good qualitive studies with comparable outcome measures) for the management of OSF and hence improved patient care.
Shailesh M Gondivkar, Amol R Gadbail, Monal Yuwanati, Vidya Lohe, Sachin Sarode
Disease Category: Dentistry & oral health
Disease Name: oral submucous fibrosis
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (patients)
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Researchers
- Service providers
- Service users
- Statisticians
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Focus group(s)
- Interview
- Literature review
- Survey
- Systematic review
Aim:
The aim of this study is to develop a COS for OSF.
Methods:
The COS will be developed using methodology reflecting the four steps outlined by the Core Outcome Measures in Effectiveness Trials (COMET) Initiative.
Potential outcomes will be initially identified from a systematic review of the literature.
Further outcomes of importance will be determined via qualitative methods with key stakeholders (patients, clinicians, researchers, Federal healthcare and funding agencies, and industry).
The outcomes identified will subsequently be reviewed using a Delphi survey to select a COS.
A consensus meeting with representatives of all stakeholders will be held to finalise the COS for OSF.
Dissemination:
The COS will be disseminated through peer-reviewed publications, conference presentations, and workshops targeting researchers, clinicians, and policymakers in field of head, Neck, and face healthcare.