Palifermin reduces patient-reported mouth and throat soreness and improves patient functioning in the hematopoietic stem-cell transplantation setting.
OBJECTIVE
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 clinicians' assessments of oral mucositis using objective scales.


METHODS
Patients (n = 212) received palifermin (60 microg/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. Patients' self-assessment data were compared with clinicians' 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 clinicians' 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 patient's overall physical and functional well-being was also reported. This was confirmed by the results of the Functional Assessment of Cancer Treatment questionnaire.


CONCLUSIONS
These results support the clinical benefit of palifermin in the HSCT setting, providing evidence that a patient's self-assessment instrument (OMDQ) may serve as an alternative tool to assess oral mucositis severity in clinical trials.
Stiff
Patrick J
PJ
Cardinal Bernardin Cancer Center, Maywood, IL 60153, USA. pstiff@lumc.edu
Emmanouilides
Christos
C
Bensinger
William I
WI
Gentile
Teresa
T
Blazar
Bruce
B
Shea
Thomas C
TC
Lu
John
J
Isitt
John
J
Cesano
Alessandra
A
Spielberger
Ricardo
R
D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects; D017024:Chemotherapy, Adjuvant; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000009:adverse effects; D003679:Deglutition; D019583:Dose Fractionation, Radiation; D004311:Double-Blind Method; D004326:Drinking; D004435:Eating; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D051523:Fibroblast Growth Factor 7 / Q000008:administration & dosage / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D009061:Mouth Mucosa / Q000187:drug effects*; D011788:Quality of Life; D018714:Radiotherapy, Adjuvant; D012720:Severity of Illness Index; D012890:Sleep; D013060:Speech; D013280:Stomatitis / Q000188:drug therapy* / Q000209:etiology / Q000517:prevention & control*; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome; D014916:Whole-Body Irradiation / Q000009:adverse effects
D051523:Fibroblast Growth Factor 7; D005047:Etoposide; D003520:Cyclophosphamide