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Children with recurrent or refractory neuroblastoma experienced an improvement in progression-free survival when treated with the combination of Hycamtin® (topotecan) plus cyclophosphamide; overall survival, however, was not improved compared with Hycamtin alone. These results from a Phase II, randomized trial were recently published in the Journal of Clinical Oncology.[1]

Neuroblastoma—a cancer that develops in nerve tissue of the adrenal gland, neck, chest, or spinal cord—most commonly occurs in infants and children, and has highly variable treatment outcomes. Some patients can be cured with relatively simple treatments, while others have poor outcomes even with very intensive treatment. Strategies to improve treatment for patients with recurrent or refractory neuroblastoma include combination chemotherapy.

In the current Phase II, randomized study, 119 children with recurrent or refractory neuroblastoma were randomized to undergo treatment with either Hycamtin alone or Hycamtin plus cyclophosphamide. Patients were assessed for response and treatment side effects, and long-term response was evaluated utilizing novel methods that accounted for additional therapy received after study participation.

  • 19% of patients receiving Hycamtin alone achieved either a partial or complete response compared with 32% of the combination group
  • Progression-free survival was better for the combination group, but there was no significant difference in overall survival
  • Adjusting for subsequent treatment, the proportion of patients alive at 2 years was similar in both groups.
  • Side effects were similar in both groups.
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The researchers concluded that the combination of Hycamtin plus cyclophosphamide resulted in an improvement in progression-free survival which did not translate to an overall survival benefit in patients with recurrent/refractory neuroblastoma.


[1] London WB, Frantz CN, Campbell LA, et al. Phase II Randomized Comparison of Topotecan Plus Cyclophosphamide Versus Topotecan Alone in Children With Recurrent or Refractory Neuroblastoma: A Children’s Oncology Group Study. Journal of Clinical Oncology. 2010;28:808-3815.