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.
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