Long-term safety outcomes in patients with hematological malignancies undergoing autologous hematopoietic stem cell transplantation treated with palifermin to prevent oral mucositis Journal Article

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Authors: Stiff, P. J.; Leinonen, M.; Kullenberg, T.; Rudebeck, M.; de Chateau, M.; Spielberger, R.
Article Title: Long-term safety outcomes in patients with hematological malignancies undergoing autologous hematopoietic stem cell transplantation treated with palifermin to prevent oral mucositis
Abstract: PURPOSE: To compare long-term safety outcomes (overall survival, disease progression, and incidence of secondary malignancies) between palifermin and placebo in the prevention of oral mucositis in patients with hematological malignancies undergoing autologous hematopoietic stem cell transplantation (HSCT). PATIENTS AND METHODS: Patients were enrolled between 1997 and 2005 into 4 phase I-III studies (3 double-blind placebo-controlled, 1 open-label) conducted at 31 sites in Australia, Europe, and the United States. Survival outcomes (overall survival, progression-free survival) were compared using hazard ratios (HRs) estimated with a Cox model that included treatment group, baseline age, disease type, Eastern Cooperative Oncology Group (ECOG) performance status, country, and presence of prior radiotherapy as covariates. The incidence of secondary malignancies was compared with a chi-squared test. RESULTS: A total of 672 patients were randomized into the studies (428 palifermin, 244 placebo). The median follow-up time for subjects alive at last visit was 7.9 years (range 0.1-14.9) for palifermin and 8.8 years (0.1-14.8) for placebo. Palifermin-treated patients had comparable overall survival (HR, 1.01; 95% confidence interval [CI] 0.78-1.31; P = .921) and progression-free survival times (HR, 1.04; 95% CI, 0.83-1.31; P = .733) compared with placebo-treated patients. Secondary malignancies were reported by 13% of palifermin-treated patients vs 11% of placebo patients (P = .477). Breakdown into secondary hematological malignancies (7% vs 6%) or solid tumors (6% vs 6%) did not suggest any differences between the treatment groups. CONCLUSION: After a follow up of up to 15 years, comparable long-term safety outcomes (overall survival, progression-free survival, and incidence of secondary malignancies) were observed for palifermin- and placebo- treated patients undergoing autologous HSCT.
Journal Title: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
ISSN: 1523-6536; 1083-8791
Publisher: Elsevier Inc  
Date Published: 2015
Language: ENG
DOI/URL:

S1083-8791(15)00547-9
Notes: LR: 20150826; CI: Copyright (c) 2015; JID: 9600628; 2015/06/17 [received]; 2015/08/13 [accepted]; aheadofprint