HealthDay News — For children with high-risk group 3 medulloblastoma, therapy intensification with carboplatin improves event-free survival, according to a study published online July 22 in JAMA Oncology.

Sarah E. S. Leary, MD, from Seattle Children’s, and colleagues examined therapy intensification with carboplatin as a radiosensitizer and isotretinoin as a proapoptotic agent in children with high-risk medulloblastoma. Patients aged 3 to 21 years with newly diagnosed high-risk medulloblastoma were included and randomly assigned to receive 36-Gy craniospinal radiation therapy and weekly vincristine with or without daily carboplatin, followed by 6 cycles of maintenance chemotherapy with cisplatin, cyclophosphamide, and vincristine, with or without isotretinoin.

The researchers found that the 5-year event-free survival rate was 62.9% and overall survival was 73.4% for all participants. Due to futility, isotretinoin randomization was closed early. Five-year event-free survival was 66.4 and 59.2% with and without carboplatin, respectively, with a significant difference seen in group 3 subgroup patients only (73.2 vs 53.7%). The investigators observed a difference in 5-year overall survival by molecular pathway: 100, 53.6, 73.7, and 76.9% for WNT pathway activated, SHH pathway activated, group 3, and group 4, respectively.

“In addition to improved risk stratification, the development of novel therapy for high-risk medulloblastoma remains a priority to improve the quality of survival,” the authors write.


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Two authors disclosed financial ties to the pharmaceutical industry.

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