Phase I/II study of the PARP inhibitor olaparib and irinotecan in children and young adults with recurrent/refractory malignancies: Arm D of the AcSé-ESMART trial

Key Points

• Olaparib combined with irinotecan exhibited significant activity against pediatric tumors.
• The strongest effects were seen in tumors with aneuploidy, indicating potential biomarkers for treatment.
• Observational analysis across various pediatric malignancies indicated positive responses to this combination therapy.
• This research highlights the necessity for further trials to validate findings in larger, more diverse populations.