A Phase II Trial of a Chemotherapy Based Regimen of Intravenous Busulfan (Busulfex), Melphalan and Thiotepa as Myeloablative Regimen Followed by a T- Cell Depleted Allogeneic Hematopoietic Stem Cell Transplant From and HLA-Compatible Donor in the Treatment of High Risk Ewing's Sarcoma Family Tumors
- Evaluate disease-free and overall survival of patients with high-risk tumors of the
Ewing's family treated with unmodified T-cell depleted allogeneic hematopoietic stem
cell transplantation after cytoreduction comprising busulfan, melphalan, and thiotepa.
- Determine the regimen-related morbidity and mortality in these patients.
- Determine the incidence of acute and chronic graft-vs-host disease in patients treated
with this regimen.
- Determine the biologic response of minimal residual disease in patients treated with
OUTLINE: This is a prospective study.
- Myeloablative preparative regimen: Patients receive busulfan IV over 2 hours every 6
hours on days -8 to -6, melphalan IV over 20 minutes on days -5 to -3, and thiotepa IV
over 4 hours on day -2.
- Allogeneic hematopoietic stem cell transplant: Patients undergo allogeneic bone marrow
or T-cell depleted peripheral blood stem cell transplantation on day 0.
- Graft-vs-host disease (GVHD) prophylaxis: Patients receive treatment according to
institutional guidelines and are given treatment against infection.
After completion of study treatment, patients are followed periodically for at least 3
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Disease-free survival at 1 year
Susan Prockop, MD
Memorial Sloan-Kettering Cancer Center
United States: Institutional Review Board
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|