Study of Reduced Toxicity Myeloablative Conditioning Regimen for Cord Blood Transplantation in Pediatric AML

• Status: Unknown status
• Trial end date: 2012-05-01
• Target enrollment: 0
• Participant gender: All

Summary

Cord blood transplantation (CBT) is an alternative option for patients with pediatric acute leukemia that indicated stem cell transplantation. Although CBT is as affective as unrelated bone marrow transplantation with lower graft versus host disease (GVHD) severity and incidence, transplantation related mortality (TRM) has been major problems after myeloablative conditioning. To reduce TRM, CBT with non-myeloablative conditionings have been performed but not so satisfactory especially for engraftment rate. Recently reduced toxicity myeloablative conditioning regimen was developed with promising result in adult bone marrow or mobilized peripheral blood transplantation. To increase the engraftment potential with low TRM rate, reduced toxicity myeloablative conditioning composed of fludarabine, intravenous busulfan plus thymoglobulin is planned for pediatric patients with acute myeloid leukemia.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Korean Society of Pediatric Hematology Oncology
• Treatments: Busulfan; Fludarabine; Fludarabine phosphate; Thymoglobulin

Criteria

Inclusion Criteria:

1. Diagnosis of acute myeloid leukemia (FAB or WHO classification).

2. Indicated for hematopoietic stem cell transplantation.

3. Age: < 21 years old.

4. Performance status: ECOG 0-2.

5. Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases. 1. Heart: a shortening fraction > 30%, ejection fraction > 45%. 2. Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal. 3. Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.

6. Patients must lack any active viral infections or active fungal infection.

7. Appropriate cord blood unit is available: accept HLA mismatch in two of six loci (pairs of A, B, and DR). Allow 2 units cord transplantation.

8. One of parents should sign informed consent.

Exclusion Criteria:

1. Pregnant or nursing women.

2. Malignant (except acute myeloid leukemia) or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.

3. Psychiatric disorder that would preclude compliance.

4. More than three HLA type mismatch.

5. T cell depleted or ex vivo expanded cord blood.