High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Rescue for Children with High-Risk Neuroblastoma. Experience of a Single Pediatric Center in Bogotá, Colombia

Abstract

Neuroblastoma (NB) is the most common extracranial solid tumor in children. Prognosis of high risk NB is extremely poor. The use of high-dose chemotherapy with autologous hematopoietic stem cell rescue in consolidation has resulted in improvements in survival and appears to have the largest impact on the survival of the high risk subset of patients although long-term event-free survival remains less than 40–50%. The aim of this retrospective study was to analyze the outcome of children with high-risk neuroblastoma who underwent Autologous Stem Cell Transplantation (ASCT) in a new pediatric stem cell transplant facility. Between August 2008 to July 2011, 16 children underwent ASCT as part of a multimodality treatment approach consisted in induction chemotherapy, surgery, HDC and autologous stem cell rescue, radiotherapy and maintenance therapy with 13-cis retinoid acid. The conditioning regimen used in all cases consisted of Carboplatin (375 mg/m2 for 4 days), Etoposide (300 mg /m2 for 4 days), and Melphalan (60 mg/m2 for 3 days). There were 16 patients with NB consisting of 6 males and 10 females. The median age was 4.7 years (range 1-14 years). The median weight was 17,4kg (range 8-40kg). 12 patients had stage IV-NB and 4 had high-risk stage III-NB. The source of stem cells was peripheral blood in 15 patients and bone marrow in 1 patient. The median time to Absolute Neutrophil Count> 0.5 x 10 9/L was 13 days (range 9-35 days). The median time to an Absolute platelet count of >20 x 10 9/L was 23 days (range 9-35 days), one patient had a graft failure who presented adequate recovery after infusion of cryopreserved bone marrow back-up. The median follow up time was 492 days (range 55 days-1127 days). 4 of the 16 recipients had relapsed. At the present time, 13 patients are alive. Relapse was the only cause of death and Transplant Related Mortality was zero. we conclude that ASCT is a feasible and effective method of treatment for patients with high risk neuroblastoma. The inclusion of high-dose chemotherapy and autologous rescue have improved the chances of survival of children with high risk neuroblastoma in developing countries allowing to reproduce the results achieved in developed countries.