Tandem stem cell rescue as consolidation therapy for high‐risk neuroblastoma

Background Despite aggressive treatment for high‐risk neuroblastoma (NB), event‐free survival (EFS) remains <40%. In single arm studies, intensifying therapy with high‐dose chemotherapy and tandem autologous stem cell rescue (HDC/SCR) improved outcome. We retrospectively describe our institutional experience in using HDC/SCR for patients with high‐risk NB, focusing on outcome and acute toxicities. Methods Eighty‐four patients with high‐risk NB at Children's Healthcare of Atlanta treated over a 12‐year time period underwent HDC/SCR as part of upfront therapy; 28 patients received a single HDC/SCR and 56 patients received tandem HDC/SCR. The two groups were compared in terms of EFS, overall survival (OS), and acute transplant related toxicities. Results Patients who received tandem HDC/SCR had a significantly improved EFS compared with patients who received a single HDC/SCR (4‐year EFS 59.3 ± 6.7% vs. 26.8 ± 9.2%, P  = 0.01). Similarly, the 4‐year OS was improved in patients receiving tandem HDC/SCR, though this did not reach statistical significance (70.6 ± 9.2% vs. 44.7 ± 11.2%, P  = 0.06). Multivariate regression confirmed the prognostic role of the treatment group. None of the patients who underwent a single HDC/SCR developed veno‐occlusive disease (VOD), while 17% of patients who underwent tandem HDC/SCR developed mild‐to‐severe VOD. Rates of other transplant‐related acute toxicities were similar. Conclusion Tandem HDC/SCR for patients with high‐risk NB seems to improve survival without significant increases in acute toxicities. This needs to be validated in randomized prospective trials. Pediatr Blood Cancer 2012; 58: 448–452. © 2011 Wiley Periodicals, Inc.