High risk neuroblastoma is incurable in at least 70% of patients.
Autologous bone marrow transplant is used so that higher doses of chemotherapy (ie- myeloablative) can be used. This has been shown to improve event free survival in high risk patients.
After myeloablative chemotherapy is given, the patient’s own bone marrow cells or stem cells are returned. This way, patient’s can receive high doses of chemotherapy directed towards the neuroblastoma without prolonged bone marrow suppression.
Preparative regimens include different drugs, but most are Melphalan based with combinations of other drugs including busulphan, Carboplatin and Etoposide.
Some centers use a thiotepa base with cyclophosphamide or etoposide or busulphan. There is no evidence to show that one preparative regimen is much better than another.
To improve event free survival (EFS), tandem (or dual-cycle) transplant regimens with PBSC support are being used after a dose intensified induction regime.
The previous COG high risk study ANBL0532 used this approach. There may be an advantage to double (tandem) transplants. ANBL0532 showed that this was possible without unacceptable toxicity.
TBI (whole body radiation therapy) is not currently part of the preparative regime prior to transplant for patients with NBL. As this has not been shown to lead to better short term outcomes and has the potential to lead to greater long-term morbidity.
