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Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation

17 Years
Open (Enrolling)
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia,, Thalassemia, Sickle Cell Disease

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Trial Information

Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation

Fludarabine is a nucleoside analog with potent antitumor and immunosuppressive properties
used in conditioning regimens of pediatric allogeneic hematopoietic cell transplantation
(alloHCT) to promote stem cell engraftment.

This is a single-center, pharmacokinetic-pharmacodynamic (PK-PD) study investigating the
clinical pharmacology of fludarabine in 45 children undergoing alloHCT at UCSF Benioff
Children's Hospital.

Patients would receive fludarabine regardless of whether or not they decide to consent to PK

Fludarabine doses will not be adjusted based on PK data.

We will apply the combination of a D-optimality-based limited sampling strategy and
population PK methodologies to determine specific factors influencing fludarabine exposure
in pediatric alloHCT recipients and identify exposure-response relationships.

Subjects will undergo PK sampling of plasma (f-ara-a) and intracellular (f-ara-ATP) drug
concentrations over the duration of fludarabine therapy (3 to 5 days).

To evaluate sources of variability impacting fludarabine exposure clinical data will be
obtained from the patient's medical chart on each day of PK sampling.

A single blood draw for the collection of DNA and genotyping of single nucleotide
polymorphisms of genes involved in fludarabine activation, transport or elimination will
occur in all patients.

To assess exposure-response relationships neutrophil engraftment, treatment-related
toxicity, and survival data will be collected through day 100 post-transplant.

Inclusion Criteria:

- Children 0-17 years of age

- Undergoing alloHCT for the treatment of malignant or nonmalignant disorder

- Receiving fludarabine-based preparative regimen

Exclusion Criteria:

- Any child 7-17 years of age unwilling to provide assent

- Parent or guardian unwilling to provide written consent

Type of Study:


Study Design:

Time Perspective: Prospective

Outcome Measure:

Identify specific clinical markers or characteristics that impact fludarabine PK exposure.

Outcome Time Frame:

up to 100 days post transplant

Safety Issue:


Principal Investigator

Janel R Long-Boyle, PharmD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of California, San Francisco


United States: Institutional Review Board

Study ID:




Start Date:

September 2010

Completion Date:

Related Keywords:

  • Hematologic Malignancies
  • Nonmalignant Diseases
  • Immunodeficiencies
  • Hemoglobinopathies
  • Genetic Inborn Errors of Metabolism
  • Fanconi's Anemia,
  • Thalassemia
  • Sickle Cell Disease
  • fludarabine
  • pharmacokinetics
  • pediatric
  • allogeneic
  • hematopoietic cell transplantation
  • Anemia
  • Anemia, Sickle Cell
  • Neoplasms
  • Fanconi Anemia
  • Fanconi Syndrome
  • Hemoglobinopathies
  • Immunologic Deficiency Syndromes
  • Metabolism, Inborn Errors
  • Thalassemia
  • Hematologic Neoplasms



University of California, San Francisco San Francisco, California  94143