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Safety, Pharmacokinetics, and Efficacy of Palifermin in Children and Adolescents with Acute Leukemias Undergoing Myeloablative Therapy and Allogeneic.

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Presentation on theme: "Safety, Pharmacokinetics, and Efficacy of Palifermin in Children and Adolescents with Acute Leukemias Undergoing Myeloablative Therapy and Allogeneic."— Presentation transcript:

1 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  Joan Morris, Mattias Rudebeck, Steven Neudorf, Theodore Moore, Reggie Duerst, Ami J. Shah, Michael Graham, Victor Aquino, Christopher Morris, Birgitta Olsson  Biology of Blood and Marrow Transplantation  Volume 22, Issue 7, Pages (July 2016) DOI: /j.bbmt Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

2 Figure 1 Treatment regimen.
Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

3 Figure 2 Dose-escalation schema.
Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

4 Figure 3 PFS by age group. The long-term PFS and OS did not differ substantially from the expected outcome for pediatric patients with acute leukemia undergoing high-dose chemoradiotherapy and allogeneic HSCT. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

5 Figure 4 OS by age group. The long-term PFS and OS did not differ substantially from the expected outcome for pediatric patients with acute leukemia undergoing high-dose chemoradiotherapy and allogeneic HSCT. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

6 Figure 5 PK parameters. There was a large variability in PK parameters, but data showed no indication of age having an effect on the PK of palifermin in this pediatric population. One subject (1 to 2 cohort, 80 μg/kg/day) with an extreme value of 18,200 mL/h/kg is excluded from the CL presentation. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

7 Figure 6 Maximum severity of oral mucositis by WHO score, all age cohorts combined. Severe OM (WHO grades 3 to 4) occurred in 37% of patients. The incidence of severe OM decreased with increasing dose levels. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

8 Supplementary Figure 1 Individual serum palifermin concentration-time profiles (semilogarithmic scale) after i.v. bolus administration of 80 μg/kg (12 to 16 cohort, day –10). Example of 2 typical and 1 atypical palifermin serum concentration profiles. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions


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