Myeloablative Busulfan/Melphalan Consolidation following Induction Chemotherapy for Patients with Newly Diagnosed High-Risk Neuroblastoma: Children's Oncology Group Trial ANBL12P1

•Transplantation with busulfan-melphalan (BuMel) is well tolerated in patients with neuroblastoma.•Hepatic toxicity with BuMel is acceptable, with sinusoidal obstruction syndrome (SOS) noted in 5.9% of recipients.•Busulfan pharmacokinetics shows a higher area under the curve in patients who develop SOS.•Three-year event-free survival and overall survival are promising in patients with high-risk neuroblastoma. Consolidation using high-dose chemotherapy with autologous stem cell transplantation (ASCT) is an important component of frontline therapy for children with high-risk neuroblastoma. The optimal preparative regimen is uncertain, although recent data support a role for busulfan/melphalan (BuMel). The Children's Oncology Group (COG) conducted a trial (ANBL12P1) to assess the tolerability and feasibility of BuMel ASCT following a COG induction. Patients with newly diagnosed high-risk neuroblastoma who did not progress during induction therapy and met organ function requirements received i.v. busulfan (every 24 hours for 4 doses based on age and weight) and melphalan (140 mg/m2 for 1 dose), followed by ASCT. Busulfan doses were adjusted to achieve to an average daily area under the curve (AUC)