Clinician- and patient-rated outcomes and their impact on Quality of Life

Introduction Chronic Graft-versus-Host disease (cGvHD) is a complication of allogeneic hematopoietic stem cell transplantation (HSCT), which diminishes patients’ quality of life (QoL). The oral cavity is often affected, causing mucosal changes (erythema, pseudomembranous ulcerations, lichenoid and hyperkeratotic changes), oral dryness, and sclerosis. Previous studies discussed the signs and symptoms and pathophysiology of oral cGvHD and its management. However, the association between objective and subjective oral cGvHD assessment tools and the influence of oral cGvHD on patients’ QoL has hardly been studied. Aims To study any associations between objective and subjective oral cGvHD assessments and to study the influence of oral cGvHD on patients’ QoL. Methods Patients with oral cGvHD referred to the Department of Oral and Maxillofacial Surgery, Academic Medical Center Amsterdam (AMC), between September 2015 and June 2016 were included. Patients filled out questionnaires on oral symptoms and QoL. Mucosal changes were scored, salivary flow rates (stimulated and unstimulated) and mouth opening were measured. Results In total 16 patients were included. A strong correlation was found between objective and subjective oral dryness (r = -0.92, p<0.01). However, objective and subjective assessments of oral mucosal cGvHD did not correlate. A significant correlation was found between QoL ratings and experiencing pain and discomfort in the oral cavity and having trouble eating (r = -0.599 p = 0.014 and r = -0.614 p = 0.011 respectively). No significant correlation was found between oral dryness, mucosal changes, and sclerosis associated with oral cGvHD. Conclusion We found that objective and subjective oral dryness assessments were strongly associated. In contrast, objective and subjective assessments of mucosal oral cGvHD did not correlate, underlining the importance of including patient-reported outcomes. No associations could be identified between oral dryness, mucosal cGvHD and sclerosis, suggesting that these features of oral cGvHD may occur in isolation. Having trouble eating and experiencing oral pain and discomfort were associated with diminished QoL. Despite of the small sample size and the need for larger prospective studies, our results have implications for identifying oral cGvHD manifestations and its management.