|Secondary Protocol No.
||MT2015-25C : Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
To evaluate 3 year progression free survival (PFS) rate of high-risk neuroblastoma patients after treatment with a tandem consolidation of Thiotepa/Cyclophosphamide and PBSC rescue followed by Carboplatin/Etoposide/ Melphalan (CEM) and PBSC rescue, as compared to historical controls of a single CEM consolidation course with PBSC rescue.
To determine key transplant outcomes as tracked by the departmental BMT database including time to engraftment, relapse, and overall survival.
||Intravenous Administration of Thiotepa, Cyclophosphamide, Mesna, Melphalan, Etoposide, Carboplatin and Infusion of mobilized stem cells.
||This is a phase II single center study to administer two courses of myeloablative consolidation
chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in
patients with high-risk neuroblastoma who have completed induction chemotherapy (independent
of this study). Ideally, patients should begin consolidation chemotherapy no later than 8 weeks
after the start of Induction Cycle #5; however it is strongly recommended to begin consolidation
within 4-6 weeks after the start of Induction Cycle #5. Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Six to 10 weeks from day 0 (rescue), consolidation course #2, consisting of melphalan, etoposide and carboplatin, is initiated followed by a 2nd PBSC rescue. It is estimated that a total of 12 patients will be enrolled in this study.
||No evidence of disease progression: defined as increase in tumor size of >25% or new lesions
Hematopoietic recovery from last induction course of chemotherapy (ANC 500 and platelets >20K).
No uncontrolled infection
Minimum frozen PBSCs of Minimum frozen PBSCs of 2 x 10^6 CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must be collected prior to starting consolidation. CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must be collected prior to starting consolidation.
Adequate organ function defined as:
Hepatic: AST < 3 x upper limit of institutional normal
Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical
congestive heart failure
Renal: creatinine clearance or GFR > 60 mL/min/1.73m^2 (If a creatinine
clearance is performed at end induction and the result is < 100
|Applicable Disease Sites
||Brain and Nervous System
Other Endocrine System
||Blood and Marrow Transplant
CYCLOPHOSPHAMIDE (CYTOXAN, NEOSAR)
||Masonic Cancer Center