5-day/5-drug myeloablative outpatient regimen for resistant neuroblastoma Journal Article


Authors: Kushner, B. H.; Modak, S.; Kramer, K.; Basu, E. M.; Roberts, S. S.; Cheung, N. K.
Article Title: 5-day/5-drug myeloablative outpatient regimen for resistant neuroblastoma
Abstract: 5-day/5-drug (5D/5D) is a novel high-dose regimen administered with autologous hematopoietic SCT (HSCT). It was designed to maximize cytoreduction via high dosing of synergistically interacting agents, while minimizing morbidity in patients with resistant neuroblastoma (NB) and ineligible for clinical trials due to myelosuppression from previous therapy. 5D/5D comprises carboplatin 500 mg/m2/day on days 1-2, irinotecan 50 mg/m 2/day on days 1-3, temozolomide 250 mg/m2/day on days 1-3, etoposide 200 mg/m2/day on days 3-5 and cyclophosphamide 70 mg/kg/day on days 4-5. HSCT is on day 8. Sixteen patients received 21 courses. Treatment was in the outpatient clinic. Responses were noted against progressive disease (PD) that had developed while patients were off, or receiving only low-dose, chemotherapy but not against PD that emerged despite high-dose chemotherapy. Responses were also seen in patients with PD or stable disease after 131 I-metaiodobenzylguanidine therapy. Grade 3 toxicities were limited to transient elevations in liver enzymes (three courses) and hyponatremia (one course). Bacteremia occurred in 2/21 (10%) courses. Hematological recovery allowed patients to be enrolled on clinical trials. In conclusion, 5D/5D (including HSCT) spares vital organs, entails modest morbidity, shows activity against resistant NB and helps patients meet eligibility requirements for formal clinical trials. © 2013 Macmillan Publishers Limited All rights reserved.
Keywords: salvage therapy; neuroblastoma; novel therapeutics
Journal Title: Bone Marrow Transplantation
Volume: 48
Issue: 5
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2013-05-01
Start Page: 642
End Page: 645
Language: English
DOI: 10.1038/bmt.2012.202
PROVIDER: scopus
PUBMED: 23085829
DOI/URL:
Notes: --- - "Export Date: 3 June 2013" - "CODEN: BMTRE" - "Source: Scopus"
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MSK Authors
  1. Brian Kushner
    311 Kushner
  2. Nai-Kong Cheung
    648 Cheung
  3. Kim Kramer
    236 Kramer
  4. Shakeel Modak
    249 Modak
  5. Ellen Marlese Basu
    101 Basu
  6. Stephen Stacy Roberts
    107 Roberts