Faculty Collaboration Database

The Impact of Palifermin Use on Hematopoietic Cell Transplant Outcomes in Children. Biol Blood Marrow Transplant 2016 Aug;22(8):1460-1466

Date

04/20/2016

Pubmed ID

27090960

Pubmed Central ID

PMC4949099

DOI

10.1016/j.bbmt.2016.04.008

Scopus ID

2-s2.0-84973879084 (requires institutional sign-in at Scopus site)   6 Citations

Abstract

Clinical trials evaluating palifermin have enrolled few pediatric patients, precluding safety analyses in large groups of children. We compared hematopoietic cell transplantation (HCT) outcomes among pediatric patients who did or did not receive palifermin as a preventive treatment for oral mucositis. Pediatric patients and controls, matched for HCT and donor type, disease, disease status, and age, were selected from the Center for International Blood and Marrow Transplant Research database and a 1:3 matched cohort analysis was performed. Stratified Cox proportional hazards models were built and propensity score adjustments were used to compare overall and disease-free survival outcomes between palifermin-treated and untreated patients. Three controls were identified for 90% of palifermin recipients. The remaining cases were matched with 2 (8%) controls or 1 (2%) control, for a total of 210 palifermin-treated patients matched with 606 controls. Median follow-up was 31 months in cases and 36 months in controls. Fifty-seven percent of patients underwent allogeneic HCT, mostly for acute leukemia, and 43% underwent autologous HCT, mostly for solid tumors. In univariate analyses, 2-year survival and disease-free survival rates after allogeneic HCT (58% versus 66%, P = .109; 49% versus 60%, P = .06) and after autologous HCT (73% versus 77%, P = .474; 60% versus 64%, P = .637) were similar between palifermin-treated patients and matched controls. In multivariate analysis, palifermin treatment did not significantly increase the risk of mortality (relative risk [RR], 1.20; 95% confidence interval [CI], .87 to 1.66) or of relapse (RR, 1.12; 95% CI, .78 to 1.62) compared with matched controls. No significant differences in rates of acute or chronic graft-versus-host disease (GVHD) were observed between palifermin-treated patients and matched controls. Among pediatric patients undergoing HCT, overall survival, disease-free survival, neutrophil recovery, and GVHD rates were similar between palifermin-treated patients and matched controls.

Author List

Saber W, Zhang MJ, Steinert P, Chen M, Horowitz MM

Authors

Mary M. Horowitz MD, MS Professor in the Medicine department at Medical College of Wisconsin
Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin
Patricia Steinert PhD Associate Professor in the Medicine department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Case-Control Studies
Child
Child, Preschool
Databases, Factual
Female
Fibroblast Growth Factor 7
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Humans
Infant
Male
Propensity Score
Recurrence
Stomatitis
Survival Analysis
Treatment Outcome