Reliability and efficacy of palifermin in prevention and management of oral mucositis in patients with acute lymphoblastic leukemia: a randomized, double-blind controlled clinical trial

Aim: Oral mucositis (OM) is a frequent finding of myeloablative and Hematopoietic Stem Cells Transplantation therapy (HSCT) with no effective treatment. This randomized-controlled trial studied the efficacy of Palifermin, administered as a dose during HSCT therapy, as primary prophylaxis on paediatric patients with Acute Lymphoblastic Leukemia (ALL). Methods: Forty-six patients (8-15 years) with B-cell acute lymphoblastic leukemia (B-ALL). The patients underwent allogenic HSCT conditioned by myeloablative regimen. Subsequently to randomization, Patients in the Palifermin group were assigned to receive Palifermin, 60 μg/kg, intravenously as a single dose 3 days before and after transplant conditioning regimen cycle. The patients in the Control group received only a placebo treatment. Maximum severity of OM, incidence and duration of ulcerative OM, incidence and duration of severe OM limitations were evaluated. Results: There was a statistically significant reduction in the incidence of OM up to grade 3 in the Palifermin group compared to the Control group. There was also a reduction, confirmed at 60 days, in the degree of severity of mucositis in the Palifermin group, with an average of 1.54 grade in the Palifermin group, and of 2.16 in the Control group and in the use of opioid analgesics. Conclusions: This study indicates that a single dose of Palifermin used as primary prophylaxis during HSTC therapy can prevent severe OM in paediatric patients with ALL and used as secondary prophylaxis can prevent the recurrence of severe OM in high-risk patients with previous mucosal injury and improves the quality of life in paediatric patients with ALL.