Efficacy of palifermin in the treatment of oral mucositis in non-hematopoietic stem cell transplant pediatric patients: experience of a single tertiary hospital.

Abstract

Background: Severe oral mucositis (OM) is one of the adverse events post-chemotherapy, radiation, and stem cell transplantation with major clinical and economic impact. The management of severe OM remains challenging. This study aimed to look for the benefit and clinical impact of palifermin for mucositis among the non-transplanted pediatric cancer population.

Methods: This is a descriptive retrospective study extended from 2016 to 2020 at Prince Sultan Military Medical City (PSMMC), in Saudi Arabia. During this period all pediatric patients (< 14 years of age) on chemotherapy and complicated by severe OM that required palifermin (35 courses), as off-labeled medication, were analyzed looking for the clinical demographics, primary diagnosis, chemotherapy agents used, effectiveness, and tolerability of palifermin.

Results: A total of 29 patients with severe OM received 35 palifermin courses. All of them received 60 mcg/kg/day IV for 3 consecutive days. 20% of them have acute lymphoblastic leukemia (ALL). We noticed that 60% of severe OM required palifermin post anthracycline while high-dose methotrexate aggravates the risk in 40% of them. Only 25.7% of the patients required TPN for a median duration of 5 days and 54.3% of them received opioids for a median duration of 4 days. Twenty patients (57.1%) had used antibiotics, 4 patients were on antifungal medication and 1 patient was on anti-viral medication concomitant with antibiotics.

Conclusion: Palifermin is safe and well tolerated and shows some effect in non-hematopoietic stem cell transplant pediatric patients with severe oral mucositis post-intensified chemotherapy or radiation therapy.