Efficacy of palifermin in the hematopoietic stem cell transplant setting.

Palifermin is a recombinant human keratinocyte growth factor that stimulates proliferation and differentiation of epithelial cells. Palifermin's biological activity exerts cytoprotective and healing effects that decrease cell injury caused by chemotherapy and radiation therapy. In randomized, placebo-controlled trials, palifermin significantly reduced the incidence and duration of severe oral mucositis. Based on these findings, the US Food and Drug Administration approved palifermin for patients with hematologic malignancies undergoing myeloablative therapy followed by hematopoietic stem cell transplant (HSCT). However, researchers testing the efficacy of palifermin in postapproval studies using various conditioning regimens have debated the extrapolation of palifermin dosage and dosing frequency used in the registration study as inappropriate for less mucotoxic agents. In addition, modifying the dosing intervals and frequency of palifermin has been proposed to decrease adverse events and achieve the highest clinical benefits for less mucotoxic regimens. The incidence and severity of oral mucositis vary significantly across different conditioning regimens. Hence, cost-effectiveness and the clinical benefits of palifermin among various conditioning regimens have also been debated. This article reviews the published literature on the efficacy of palifermin and makes evidence-based recommendations for the use of palifermin in the HSCT setting.