A randomized trial of 13-Cis retinoic acid in children with advanced neuroblastoma after high-dose therapy
2000

Study on 13-Cis Retinoic Acid for Neuroblastoma in Children

Sample size: 175 publication Evidence: moderate

Author Information

Author(s): Kohler J A, Imeson J, Ellershaw C, Lie S O

Primary Institution: Child Health, Southampton General Hospital

Hypothesis

Does 13-Cis retinoic acid improve event-free survival in children with advanced neuroblastoma after high-dose therapy?

Conclusion

The study found that 13-Cis retinoic acid did not show any advantage in event-free survival compared to placebo.

Supporting Evidence

  • The study involved 175 children with advanced neuroblastoma.
  • Participants were randomly assigned to receive either the treatment or a placebo.
  • The treatment was administered for up to 4 years.
  • Toxicity from the treatment was mild.

Takeaway

The study tested a medicine called 13-Cis retinoic acid on kids with a serious cancer, but it didn't help them live longer without problems compared to those who didn't take it.

Methodology

Children were randomly assigned to receive either 13-Cis retinoic acid or a placebo for up to 4 years.

Limitations

The study did not demonstrate a benefit in event-free survival despite mild toxicity.

Participant Demographics

Children with Stage 3 or 4 neuroblastoma who had a good response to conventional therapy.

Digital Object Identifier (DOI)

10.1054/bjoc.2000.1425

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