Safety, pharmacokinetics and efficacy of palifermin in children and adolescents with acute leukemias undergoing myeloablative therapy and allogeneic hematopoietic stem cell transplantation: a Pediatric Blood and Marrow Transplant Consortium Trial.
Biol Blood Marrow Transplant. 2016 Mar 8;
Authors: Morris J, Rudebeck M, Neudorf S, Moore T, Duerst R, Shah AJ, Graham M, Aquino V, Morris C, Olsson B
Abstract
Currently, there is no available effective pharmacological treatment to reduce severe oral mucositis (OM) resulting from high-dose myeloablative cytotoxic therapy in the pediatric population. Palifermin has been proven to decrease the incidence and duration of severe OM in adults with hematologic malignancies undergoing hematopoietic stem cell transplantation (HSCT). In the pediatric population the data on palifermin treatment is limited. A Phase 1 dose-escalation study of palifermin in pediatric patients with acute leukemias undergoing myeloablative HSCT with total body irradiation, etoposide and cyclophosphamide was performed to determine a safe and tolerable dose as well as to characterize the pharmacokinetic (PK) profile and efficacy of palifermin. Twenty-seven patients in three age groups (1-2, 3-11, and 12-16 years) and three dose levels (40, 60, and 80 μg/kg/day) were studied. There were no deaths, dose-limiting toxicities, or treatment-related serious adverse events. Long-term safety outcomes did not differ from what would be expected in this population. PK data showed no differences between the three age groups. Exposure did not increase with increase in dose. The maximum severity of OM (WHO Grade 4) occurred in 6 (22%) of the patients, none of whom were in the 80 μg/kg/day dosing group. This study showed that all doses were well tolerated and showed a good safety profile in all three pediatric age groups.
PMID: 26968792 [PubMed - as supplied by publisher]
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