Pediatric Cancers

Findings from 2 phase 3 trials support the recommendation to approve sodium thiosulfate to reduce the risk of cisplatin-related hearing loss in pediatric patients with solid tumors in Europe.

Findings from a cohort of the Childhood Cancer Survivor Study identify a prediction model that may accurately identify childhood cancer survivors at varying risks of late kidney failure.

Results from the phase 2 CDRB436G2201 trial lead to the approval of dabrafenib plus trametinib in patients with low-grade glioma and a BRAF V600E mutation who require systemic therapy.

Disparities in pediatric cancer survival in Europe might be mitigated through cross-border care, twinning programs, and use of pan-Europe cancer control plans.

The FDA initially issued a complete response letter for remestemcel-L in pediatric steroid-refractory acute graft-versus-host disease in October 2020, citing a need for an additional randomized, controlled trial to confirm the agent’s efficacy.

Findings from a retrospective cohort study suggest that circulating tumor DNA may be an important tool for risk-stratified treatment strategies in rhabdomyosarcoma.

Young adult survivors of childhood cancer may be at a higher risk of experiencing psychological and physical health issues associated with loneliness.

A medical oncologist and research audiologist from St. Jude Children’s Research Hospital discuss how sodium thiosulfate injection may improve quality of life by preventing cisplatin-associated ototoxicities in pediatric patients with localized non-metastatic tumors, although more research is needed.

A biologic license application that has been resubmitted to the FDA for remestemcel-L in pediatric graft-versus-host disease includes new long-term survival data from a phase 3 clinical trial.

The FDA gives orphan drug exclusivity to sodium phosphate injection for the prevention of cisplatin-related hearing loss in pediatric patients 1 month and older with localized, non-metastatic solid tumors.

Data from the phase 2 FIREFLY-1 trial indicated that tovorafenib yielded responses among pretreated patients with recurrent or progressive pediatric low-grade glioma.

Adults and children with unresectable or metastatic alveolar soft part sarcoma—one of the rarest types of sarcoma—can now receive treatment with atezolizumab, which was given the nod of approval by the FDA.

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.

Pediatric patients with high allelic ratio FLT3/ITD–positive acute myeloid leukemia may benefit from treatment with sorafenib plus chemotherapy.

Pediatric patients with newly diagnosed pediatric T-cell lymphoblastic lymphoma experienced an improvement in survival following treatment with bortezomib and chemotherapy.

Breast cancer risk was estimated among survivors of pediatric cancer who were treated with chest radiation with a newly developed and validated breast cancer risk prediction model.

IMX-110, which was granted a rare pediatric disease designation for patients with rhabdomyosarcoma, is currently being assessed as part of a phase 1b/2a study.

The FDA has approved rituximab plus chemotherapy for previously untreated pediatric CD20-postive diffuse large B-cell lymphoma, Burkitt lymphoma, Burkitt-like lymphoma, and mature B-cell acute leukemia following results from the phase 3 Inter-B-NHL Ritux 2010 study.

In this clinical quandary, investigators determine how to best treat patients with opsoclonus-myoclonus–associated neuroblastoma with bone marrow metastases.

The addition of temozolomide to vincristine and irinotecan appears to be a new standard of care for adult and pediatric patients with relapsed/refractory rhabdomyosarcoma, according to the European Paediatric Soft Tissue Sarcoma Group.

Pediatric patients with medulloblastoma may experience benefit from 177Lu-omburtamab-DTPA, which was granted a rare pediatric disease designation by the FDA.