FRIDAY, July 23, 2021 (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, M.D., 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 six cycles of maintenance chemotherapy with cisplatin, cyclophosphamide, and vincristine, with or without isotretinoin.

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

Two authors disclosed financial ties to the pharmaceutical industry.

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