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Tandem stem cell rescue as consolidation therapy for high‐risk neuroblastoma
Author(s) -
Qayed Muna,
Chiang KuangYueh,
Ricketts Richard,
Alazraki Adina,
Tahvildari Ali,
Haight Ann,
George Brad,
Esiashvili Natia,
Katzenstein Howard M.
Publication year - 2012
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.23155
Subject(s) - medicine , chemotherapy , surgery , oncology , multivariate analysis
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.