Mucositis is a well-known complication of both autologous and allogeneic hematopoietic stem
cell transplantation (HSCT). Many who suffer this disorder require total parental nutrition
and intravenous narcotics for pain control. Palifermin (Kepivance[TM]) is a human
keratinocyte growth factor that is produced by recombinant DNA technology in E. coli.
Palifermin is a FDA-approved, commercially available pharmacologic agent that is
manufactured by Amgen. As keratinocyte growth factor receptors have been found within the
epithelium of gastric mucosa, the use of palifermin has been proven to decrease the
frequency and duration of severe mucositis in adult studies. Whereas the appropriate dosing
regimen has been determined for adults at 60mcg/kg/day, the dosing of palifermin has not
been established in the pediatric setting. This initial pediatric study of palifermin will
determine the maximum tolerated dose, evaluating the use of this agent at three dose levels,
below, at, and above the recommended adult dose. Non-hematologic, life-threatening NCI
grade IV or grade V toxicities definitely related to the administration of palifermin from
the first infusion until day +6 after HSCT (post palifermin administration day +3) will
comprise the safety endpoints of the study.
The study is designed to evaluate palifermin at 3 dose levels. The study population will be
recipients of either a matched family member donor or matched unrelated donor HSCT. The
pharmacokinetics of palifermin at each dose level will be described to help determine the
appropriate dose for future studies, which will evaluate efficacy
Secondary objectives of this study include exploring the pharmacoeconomics of palifermin in
this particular patient population through an assessment of inpatient days, intravenous
nutrition, and analgesia requirements within 100 days post-transplantation. We will also
explore the research participants' rates of immune reconstitution, specifically T and
B-lymphocytes and NK cells, within the first year of HSCT.
- Age greater than or equal to 2 years and less than 18 years of age.
- Diagnosis of a hematologic malignancy.
- Has not received a prior autologous or allogeneic HSCT.
- Is scheduled to receive either a matched family member or MUD bone marrow stem cell
graft at St. Jude Children's Research Hospital. A matched family member donor is
defined as a sibling matched at 5 or 6 HLA loci or another related donor matched at 6
- Is scheduled to receive a myeloablative preparative regimen (cyclophosphamide/TBI
based) prior to the infusion of the allogeneic graft.
- Cardiac shortening fraction greater than or equal to 25%.
- Serum creatinine is less than twice the upper limit of normal for age.
- Bilirubin less than 3.0 mg/dl.
- Aspartate transaminase (AST) less than 500 IU/ml.
- Alanine transaminase (ALT) less than 500 IU/ml.
- Amylase less than 1.5 times the upper limits of normal for age.
- Lipase less than 1.5 times the upper limits of normal for age.
- Forced vital capacity (FVC) greater than or equal to 40% of predicted value or pulse
oximetry greater than or equal to 92% on room air.
- No known hypersensitivity to E coli-derived proteins or palifermin.
- No active or recent (within 30 days prior to enrollment) gastrointestinal bleeding.
- No active or recent (within 30 days prior to enrollment) oral ulcerations.
- No active fungal infection, bacteremia or viremia within two weeks prior to
- Female - pregnant (negative serum or urine pregnancy test within 14 days prior to
- Female - lactating.