Impact of Palifermin On Incidence of Mucositis and Length of Hospitalization in Children Undergoing Autologous Hematopoietic Stem-Cell Transplant for Malignant Disorders

Myeloablative chemotherapy/radiotherapy prior to autologous hematopoietic stem cell transplant (AHSCT) is a leading cause of mucositis associated with significant morbidity.  Palifermin has been demonstrated to decrease the incidence of severe mucositis in adults following total body irradiation (TBI) conditioning prior to AHSCT. The impact of palifermin on the incidence of mucositis in children following AHSCT has never been studied.  In this retrospective study, we compared the incidence of mucositis and supportive care required in children who received palifermin vs. controls (no palifermin) during non-TBI AHSCT. Mucositis was graded as per WHO criteria. The continuous variables were summarized by the mean and standard deviation; the categorical variables were summarized by percentage. The palifermin vs. control group were compared by two-sided t-test for continuous measurements and by Chi-square test for categorical measurements. From 2005-2011, 58 patients received myeloablative AHSCT, of which n=25 were in the palifermin group and n=33 were in the control group. Demographic characteristics are presented in Table. Comparing palifermin vs. the control group: the average time for neutrophil engraftment was 12.16 ±3.21 days vs. 11.5 ±1.68 (p=0.127), the incidence of ≥ grade I and III-IV mucositis was 80% vs. 90.9%, and 20% (p= 0.02) vs. 42.4% (p=0.07), the number of days with fever were 4.92±3.49 vs. 7.09±4.86 (p=0.063), the number of days patients received PCA were 8.80±8.39 vs. 8.30±8.54 (p=0.826), and the number of days patients were on TPN were 13.52±11.32 vs. 11.55±9.63 (p=0.484), respectively. The incidence of blood stream and Clostridium difficile infection was 36% vs. 27.3% (p=0.4) and 24% vs. 18.2% (p=0.5), respectively. The average length of hospital stay 31.44±7.42 vs. 28.61±10.38 (p=0.252) was not statistically different between the palifermin and control groups. In summary, we were unable to demonstrate that there was a statistical difference with incidence of mucositis and other supportive care needs or a decrease in hospital stay in the palifermin group. In children receiving AHSCT, palifermin should only be used in the setting of a large prospective study.

Variable	Control group (No Palifermin, n=33) Mean (SD)	Palifermin group (n=25) Mean (SD)	p-value
Age	6.85 (5.49)	7.96 (6.19)	0.473
Gender: Male           Female	20 (60.6%) 13(39.4%)	14(56%) 11(44%)	0.724
Weight	30.62(27.08)	35.02(25.95)	0.535
BSA	0.98(0.55)	1.05(0.51)	0.580
Brain Tumor	15(45.5%)	3(12%)	0.024
Solid Tumor	11(33.3%)	14(56%)
Lymphoma	7(21.2%)	8(32%)
Disease Status CR/PR SD/PD	29(87.9%) 4(16%)	21(84.0%) 4(12.1%)	0.671
Prior Radiation: Yes No	3(9.1%) 30(90.9%)	6(24%) 19(76%)	0.120
HSV serostatus: Positive Negative	25(75.8%) 8(24.2%)	16(64%) 9(36%)	0.330
Creatinine clearance	118.1 (42.55)	134.8 (49.31)	0.176