Oral mucositis (OM) is a frequent and debilitating complication for children undergoing hematopoietic stem cell transplantation (HSCT) that causes significant pain and increases risk of infections or multi-organ failure, and negatively impacts treatment outcomes, patients quality of life and healthcare resources. Palifermin (Keratinocyte Growth Factor, rHuKGF) decreases the incidence and duration of severe mucositis in adult patients receiving HSCT, but so far no data are available on use of Palifermin in children undergoing HSCT. In current study, Palifermin short-term safety and efficacy have been established in pediatric HSCT setting. Methods: A total number of 10 children undergoing HSCT were included into the study. All patients had suffered WHO grade 4 OM during previous chemotherapy. Five children aged 5–16 years were treated with Palifermin: 3 with relapsed ALL had matched-sibling donor allogeneic transplantation, while 2 others with advanced solid tumors (germinal tumor and neuroblastoma) underwent autologous transplantation. Palifermin was administered in the dose 60 μg/kg/day for 3 days prior to conditioning therapy and for 3 days following HSCT. The control group consisted of 5 patients, identified as closely as possible for diagnosis, conditioning, and age, who were included into matched-pair analysis. OM was graded daily according to WHO criteria and evaluated for 28 days after transplantation. All patients were followed-up until day 100 after transplantation. Results: OM WHO grade 4 was not observed in Palifermin group, while 4/5 patients in control group had OM grade 4. The median duration of mucositis of WHO grade ≥3 was 5 days (range, 3 to 8) in the Palifermin group, and 8 days (range, 6–12) in the control group. Opioid analgesic use was not necessary in Palifermin group. None of the patients in Palifermin group had blood-borne bacterial infections nor clinically documented infection. Following allo-HSCT, no patients had acute GVHD>1 in Palifermin group. All patients received total parenteral nutition (TPN) due to loss of appetite. No differences in hematological recovery were observed between groups. Palifermin treatment was associated with improvements in patient-reported outcomes (mouth and throat soreness, swallowing, eating, drinking, taking) and clinical sequelae, including incidence of febrile neutropenia, duration of hospitalization, requirements for parenteral nutrition and well-being. No serious adverse effects were observed in Palifermin group: adverse events, mainly rash, and taste alteration, were mild and transient, while 4/5 patients had white film coating mouth or tongue and taste loss in Palifermin group. Conclusion: Palifermin reduced the duration and severity of OM in pediatric patients after high-dose therapy for HSCT. It appears to reduce the incidence of WHO grade 4 OM and duration of WHO grade 3 or 4 OM and promotes faster mucosal recovery in allogeneic and autologous HSCT recipients. Use of Palifermin for the prevention of severe OM has shown no negative impact on disease outcomes in the HSCT setting for children. The ability of Palifermin therapy to reduce the breakdown of mucosal barrier could decrease the complications caused by systemic infection. Reducing the complications of oral mucositis could decrease the length of hospitalization and serious sequelae.

Disclosures: Palifermin is not registered for use in children.

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