Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation
- Determine the feasibility of allogeneic hematopoietic stem cell transplantation using a
reduced-intensity conditioning regimen, in terms of whole blood engraftment rate at 100
days post transplant, in pediatric patients with hematopoietic malignancies who are at
high risk for complications with conventional transplantation.
- Determine the feasibility of donor lymphocyte infusions (DLIs), in terms of number of
patients who receive at least one DLI by 12 months post transplant, in patients treated
with this regimen.
- Determine the toxicities of the conditioning regimen, in terms of 100-day post
transplant nonrelapse-related death rate, in these patients.
- Determine the toxicity of DLI, in terms of acute and chronic graft-vs-host disease rate
and 12-month post transplant nonrelapse-related death rate, in these patients.
OUTLINE: This is a pilot study.
- Reduced-intensity conditioning regimen: Patients receive fludarabine IV on days -6 to
-2; antithymocyte globulin IV on days -5 to -2; and melphalan IV on days -3 and -2.
- Transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation
on day 0. Patients also receive filgrastim (G-CSF) IV beginning on day 5 and continuing
until blood counts recover.
- Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV or orally
beginning on day -1 and continuing until at least day 28 and methotrexate IV on days 1,
3, and 6.
- Donor lymphocyte infusion (DLI): Patients with mixed chimerism, no acute GVHD requiring
therapy, and no relapse/progression post transplant at day 90 may receive DLI. At least
30 days after discontinuation of immunosuppression, patients may receive up to 2 DLIs
at least 8-12 weeks apart in the absence of GVHD.
At the completion of study treatment, patients are followed periodically for 2 years.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Biljana Horn, MD
University of California, San Francisco
United States: Federal Government
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