The Impact of Pretransplant Disease Characteristics on the Outcome of Autologous Stem Cell Transplantation for Neuroblastoma with High-Risk Features: A Retrospective Model from a Limited Resources Country

Abstract

High-risk neuroblastoma still has poor survival outcome. Improvement of outcome is attributed to the consolidation of chemotherapy by autologous bone marrow transplant. Further improvement of the outcome by tandem autologous transplant is followed by immune therapy. We aimed with this study to correlate initial disease characteristics with the outcome of transplanted high-risk neuroblastoma. A retrospective analysis was done for 73 transplanted patients. Patients were treated in Children’s Cancer Hospital Egypt from July 2012 to July 2015. Seventy patients received Busulphan/Melphalan conditioning. The 3-year overall survival (OS) and event - free survival (EFS) was 63.3% and 51.3%, respectively. Disease stage did not impact the OS and EFS, P = 0.54 and 0.62 respectively. Status of MYCN did not reflect statistically on outcome for tumors with amplified compared to nonamplified (EFS, 49% and 63.1%, respectively). Response after induction chemotherapy pointed that patients who had objective response (complete response, very good partial response and partial response) were better compared to those with less response with EFS and OS of 53.3% and 64.2% compared to 49.3% and 63.5%, respectively, which may indicate that chemo-sensitive tumors have better outcome. By the end of the study, twenty-seven patients relapsed, out of them 25 patients died. Pretransplant risk features for neuroblastoma was nullified by autologous stem cell transplant. The modest outcome observed, highlights some limitations that need to be sorted out in countries with limited resources. The introduction of immune therapy and tandem transplant is needed to achieve a better outcome, yet it adds to more financial burden.