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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
13
Carboplatin intravenously (IV), 425 mg/m2/dose (or if ≤ 12kg, 14.2 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant.
On day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.
Beginning on day 0 after infusion of the PBSC, patients will receive G-CSF subcutaneously (SQ) or IV (SQ preferred) 5 micrograms/kg once daily and continuing once daily until post-nadir absolute neutrophil count (ANC) \> 2000/μL for 3 consecutive days.
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
Number of Patients with Successful Engraftment
The time to neutrophil engraftment will be assessed by standard statistical approaches.
Time frame: Day 42
Number of Patients with Disease Free Survival
The number of patients alive and disease free will be assessed using standard statistical approaches.
Time frame: 2 Years
Overall Survival
The number of patients alive will be assessed by standard statistical approaches.
Time frame: 2 Years
Number of Patients with Treatment Related Death
The rate of treatment related mortality will be assessed by cumulative incidence approach.
Time frame: 1 Year
Number of Patients with Disease Free Survival
The number of patients alive and disease free will be assessed using standard statistical approaches.
Time frame: 5 Years
Overall Survival
The number of patients alive will be assessed by standard statistical approaches.
Time frame: 5 Years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
It is suggested that patients who have a complete surgical resection of the primary tumor receive 21.6 Gy external beam radiation therapy (EBRT) to the post-induction chemotherapy, pre-operative primary tumor volume. It is suggested that patients who have an incomplete surgical resection of the primary tumor (residual soft tissue mass measuring \>1 cm3) will receive 21.6 Gy EBRT to the postinduction chemotherapy, pre-operative primary tumor volume and an additional boost of 14.4 Gy EBRT to the gross residual tumor (total dose 36 Gy to gross residual tumor volume). Radiation should be given after stem cell transplantation and should start no sooner than 28 days post transplant.
Post-transplant maintenance therapy with cis-RA daily for 14 days every 28 days repeated for 6 months. This phase of the therapy can be initiated by the BMT team and continued by the referring physician. It is recommended to begin Isotretinoin at day 66 post-transplant and no later than day 100. For patients ≤12 kg, isotretinoin (accutane) should be administered at 5.33 mg/kg/dose divided twice daily. For patients \>12 kg isotretinoin (accutane) should be administered at 160 mg/m\^2/day divided twice a day. Patients should be considered for monoclonal antibody therapy against GD2, such as ch14.18 if such trials are available.
Melphalan Intravenously (IV), 70 mg/m2/dose (or if ≤ 12 kg, 2.3 mg/kg/dose) once daily x 3 doses on days 7 through 5 pretransplant
Etoposide intravenously (IV), 338 mg/m2/dose (or if ≤ 12kg, 11.3 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant