Pilot Study of High-Dose Chemotherapy With Busulfan, Melphalan, and Topotecan Followed by Autologous Hematopoietic Stem Cell Transplant in Advanced Stage and Recurrent Tumors
I. To assess the feasibility of a novel combination conditioning therapy with
busulfan/melphalan and topotecan followed by autologous hematopoietic stem cell
transplantation (HSCT) in patients with relapsed, refractory and/or poor risk pediatric
II. To determine within the confines of this pilot study, myeloid and platelet engraftment,
overall survival and disease-free survival in patients with relapsed, refractory pediatric
solid tumors and in patients who have solid tumors with poor risk factors at the time of
III. To determine the pharmacokinetics of topotecan.
AUTOLOGOUS HEMATOPOIETIC STEM CELL OR AUTOLOGOUS BONE MARROW COLLECTION: Patients undergo
stem cell mobilization per institutional guidelines with G-CSF IV or subcutaneously,
continuing until the completion of leukapheresis. Patients undergo apheresis after
mobilization and continue until a minimum of 2.0 x 10^6 CD34 cells/kg or more are collected.
Cells are processed and cryopreserved following institutional guidelines. Patients who
collect > 2.0 x 10^6 CD34+ cells/kg may proceed to high-dose chemotherapy.
HIGH-DOSE CHEMOTHERAPY: Patients receive topotecan hydrochloride IV continuously over 24
hours on days -8 to -4, busulfan IV every 6 hours on days -8 to -4, and melphalan IV over 30
minutes on days -3 and -2.
AUTOLOGOUS HEMATOPOIETIC STEM CELL OR AUTOLOGOUS BONE MARROW REINFUSION: Patients undergo
autologous hematopoietic stem cell transplantation or autologous bone marrow transplantation
on day 0. Patients also receive G-CSF IV daily beginning on day +5 and continuing until
blood counts recover.
After completion of study treatment, patients are followed every 3 months for 1 year and
then annually thereafter.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Treatment feasibility in terms of investigational agent-related adverse events of a novel treatment combination followed by peripheral blood stem cell rescue
Day 100 post stem cell rescue
Anna Pawlowska, MD
City of Hope Medical Center
United States: Institutional Review Board
|City of Hope||Duarte, California 91010|