A systematic review of reported clinical cases and treatment strategies was performed to better understand the prognostic factors and to develop the best possible treatment option for a 16-year-old patient diagnosed with a malignant triton tumor in the lower extremity with distant metastases in the lungs.
Patients with previously untreated high-risk pediatric lymphoma can now receive treatment with brentuximab vedotin with the FDA’s nod of approval.
Findings from a phase 3 trial assessing nomacopan in patients with hematopoietic stem cell transplant–related thrombotic microangiopathy helped to support a rare pediatric designation from the FDA for the agent.
Members of the FDA’s Oncologic Drugs Advisory Committee indicated there isn’t enough evidence to definitively confirm the overall survival benefit of 131I-omburtamab in pediatric patients with central nervous system or leptomeningeal metastases stemming from neuroblastoma.
The FDA has given orphan drug designation to ET140203 ARTEMIS, an investigational therapy for hepatoblastoma, the safety and potential efficacy of which are being evaluated in the phase 1/2 ARYA-1 and ARYA-2 studies.
Sodium thiosulfate, which appears to be effective in decreasing the risk for hearing loss related to treatment with cisplatin, received FDA approval in pediatric patients with localized, non-metastatic solid tumors.
BRAF V600 mutation–positive pediatric low-grade glioma positively responds to combination treatment with dabrafenib plus trametinib, according to data presented at 2022 ASCO.
Non-White Hispanic pediatric patients with neuroblastoma appear to have worse overall survival outcomes vs other patient subgroups.
Based on the results of the AZA-JMML-001 trial, the FDA has approved azacitidine for use in newly diagnosed juvenile myelomonocytic leukemia.
A biologics license application for omburtamab was resubmitted following a refusal to file letter in 2020 for pediatric patients with central nervous system/leptomeningeal metastatic from neuroblastoma.