부산대학교 도서관

PURPOSE: To describe patient-reported outcomes of mouth and throat soreness (MTS) and related sequelae on daily activities from a phase III study of palifermin in the autologous hematopoietic stem-cell transplantation (HSCT) setting and to compare patient self-evaluations with cliniciansʼ assessments of oral mucositis using objective scales. PATIENTS AND METHODS: Patients (n = 212) received palifermin (60 μg/kg/d) or placebo for 3 days before total-body irradiation (12 Gy), etoposide 60 mg/kg, and cyclophosphamide 100 mg/kg, and 3 days after HSCT. Patients completed a daily questionnaire (Oral Mucositis Daily Questionnaire [OMDQ]) evaluating MTS severity and its effects on daily functional activities. Patientsʼ self-assessment data were compared with cliniciansʼ assessments of oral mucositis using the objective scales. RESULTS: Palifermin reduced the incidence and duration of severe oral mucositis, as assessed by both clinicians and patients. Comparisons between patient and clinician assessments demonstrated that the average daily scores between mucositis grade and subjective (MTS) instruments were similar, although patients reported MTS onset, peak, and resolution earlier (1 to 3 days) than cliniciansʼ assessments. Patients receiving palifermin reported statistically significant improvements (P < .001) in daily functioning activities (swallowing, drinking, eating, talking, sleeping) and required significantly less narcotic opioids (P < .001); improvement in the patientʼs overall physical and functional well-being was also reported. This was confirmed by the results of the Functional Assessment of Cancer Treatment questionnaire. CONCLUSION: These results support the clinical benefit of palifermin in the HSCT setting, providing evidence that a patientʼs self-assessment instrument (OMDQ) may serve as an alternative tool to assess oral mucositis severity in clinical trials.