High Dose Chemotherapy and Autologous Transplant for Neuroblastoma
- Conditions
- Neuroblastoma
- Interventions
- Biological: Autologous stem cell infusionBiological: Granulocyte colony stimulating factorRadiation: Radiation therapy
- Registration Number
- NCT01526603
- Lead Sponsor
- Masonic Cancer Center, University of Minnesota
- Brief Summary
This is a standard of care document, outlining the therapy for children with high risk neuroblastoma who are not eligible for Children's Oncology Group (COG) studies.
- Detailed Description
This therapy involves the use of melphalan, etoposide, and carboplatin (consolidation chemotherapy); autologous stem cell rescue, post-transplant radiation therapy and a maintenance phase with Isotretinoin (Accutane, 13-cis-retinoic acid) therapy. If available, patients should also consider post-transplant therapy with cytokines and monoclonal antibody (ch14.18) on a COG or New Approaches to Neuroblastoma Therapy (NANT) trial.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 13
-
Less than 30 years of age at diagnosis of neuroblastoma
-
No evidence of disease progression: defined as increase in tumor size of >25% or new lesions
-
Recovery from last induction course of chemotherapy (absolute neutrophil count > 500 and platelet > 20,000)
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No uncontrolled infection
-
Minimum frozen peripheral blood stem cells (PBSCs) of 2 x 10^6 CD34 cells/kg for transplant are mandatory and 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of 4 x 106 CD34 cells/kg is encouraged)
-
Adequate organ function defined as:
- Hepatic: aspartate aminotransferase (AST) < 3 x upper limit of institutional normal 8 Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical congestive heart failure 8 Renal: Creatinine clearance or glomerular filtration rate (GFR) > 60 mL/min/1.73m^2 If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2
Exclusion Criteria
- Patients with progressive disease should consider participating in phase I studies since consolidation therapy using the regimen outlined in this document have not been determined to be useful.
- Patients who are delayed in consolidation chemotherapy beyond 8 weeks, and don't meet organ function criteria.
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Patients Treated for Neuroblastoma Isotretinoin (13-cis-retinoic acid) According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Granulocyte colony stimulating factor According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Autologous stem cell infusion According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Radiation therapy According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Melphalan According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Carboplatin According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy. Patients Treated for Neuroblastoma Etoposide According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy.
- Primary Outcome Measures
Name Time Method Number of Patients with Successful Engraftment Day 42 The time to neutrophil engraftment will be assessed by standard statistical approaches.
- Secondary Outcome Measures
Name Time Method Number of Patients with Disease Free Survival 5 Years The number of patients alive and disease free will be assessed using standard statistical approaches.
Overall Survival 5 Years The number of patients alive will be assessed by standard statistical approaches.
Number of Patients with Treatment Related Death 1 Year The rate of treatment related mortality will be assessed by cumulative incidence approach.
Trial Locations
- Locations (1)
Masonic Cancer Center, University of Minnesota
🇺🇸Minneapolis, Minnesota, United States