Pediatric Oncology

A rolling new drug application seeking the approval of tovorafenib monotherapy for the treatment of patients with relapsed or progressive pediatric low-grade glioma has been submitted to the FDA.

The FDA has placed a clinical hold on the phase 1 PLAT-08 trial evaluating SC-DARIC33 in pediatric and young-adult patients with relapsed/refractory CD33-positive acute myeloid leukemia.

Alex Herrera, MD, discussed key data from the SWOG S1826 trial and other important research in hematologic malignancies presented at the 2023 ASCO Annual Meeting.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended the approval of JZP458 for use in combination with multi-agent chemotherapy for the treatment of adult and pediatric patients 1 month and older with acute lymphoblastic leukemia and lymphoblastic lymphoma who developed hypersensitivity or silent inactivation to Escherichia coli–derived asparaginase.

The phase 1 PLAT-08 trial investigating SC-DARIC33 in pediatric and young-adult patients with relapsed/refractory CD33-positive acute myeloid leukemia has been paused following the report of a grade 5 serious adverse effect.

The investigational CAR T-cell therapy GD2-CART01 showed preliminary antitumor activity and safety in pediatric patients with relapsed or refractory high-risk neuroblastoma, according to data from a phase 1/2 trial.

The combination of PARP and ATR inhibition with olaparib and ceralasertib was tolerable but showed limited efficacy in pediatric patients with advanced malignancies harboring DNA replication stress and DNA repair deficiencies, according to findings from arm N of the phase 1/2 AcSé-ESMART trial.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended the approval of sodium thiosulfate injection for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to less than 18 years of age with localized, nonmetastatic solid tumors.

The FDA has granted full approval to pembrolizumab for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability–high or mismatch repair–deficient solid tumors that have progressed following previous treatment and who have no satisfactory alternative options.

Matthew J. Ehrhardt, MD, MS, discusses the importance of taking a multifactorial approach to improve long-term outcomes for survivors of childhood cancer, and expands on the findings from the study of the factors associated with risk of late mortality.

Living in a socioeconomically disadvantaged neighborhood or living with modifiable chronic health conditions were associated with an increased risk for death 5 years of more following diagnosis among survivors of childhood cancer, according to findings from a cohort study.

The FDA has approved dabrafenib with trametinib for the treatment of pediatric patients 1 year of age and older with low-grade glioma with a BRAF V600E mutation who require systemic therapy.

Fennec Pharmaceuticals announced an update to the National Comprehensive Cancer Network’s practice guidelines for Adolescent and Young Adult Oncology, calling for the use of sodium thiosulfate injection to reduce the risk for ototoxicity in pediatric patients receiving cisplatin.

The FDA has approved brentuximab vedotin (Adcetris) in combination with doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide in pediatric patients aged 2 years and older with previously untreated, high-risk classical Hodgkin lymphoma.

The FDA has granted an orphan drug designation to ET140203 for the treatment of patients with hepatoblastoma.

Nilay Shah, MD, discusses the significance of the approval of sodium thiosulfate for pediatric patients with cisplatin-treated pediatric solid tumors.

Alpelisib decreased the need for surgery and led to improvements in performance status and disease-related signs and symptoms in pediatric patients with PIK3CA-related overgrowth spectrum disease who received treatment with the PI3K inhibitor under compassionate use.

Neel S. Bhatt, MBBS, MPH, discusses the longitudinal effects of chronic diseases in childhood cancer survivors.

The National Comprehensive Cancer Network issued recommendations for providers treating children with brain cancers.

The FDA has granted an orphan drug designation to paxalisib for use as a potential therapeutic option for patients with atypical rhabdoid and teratoid tumors, a rare and highly aggressive pediatric brain cancer.

The combination of dabrafenib plus trametinib demonstrated a significant improvement in overall response rate, clinical benefit rate, and progression-free survival and fewer grade 3 or greater adverse effects and discontinuations vs carboplatin and vincristine in pediatric patients with low-grade glioma harboring a BRAF V600 mutation.

Non-White Hispanic children and those with public insurance care in a clinical trial for high-risk neuroblastoma had inferior overall survival rates compared with other children.

“I can’t conceive of anything worse than being in a situation where you can’t ask, ‘How do we get better?’ ”

A supplemental new drug application has been submitted to the FDA seeking the approval of ibrutinib for the treatment of pediatric and adolescent patients aged 1 year and older with chronic graft-vs-host disease following failure of 1 or more lines of systemic therapy.

New agents for rare cancers make up approximately 35% of drugs in the pipeline for all rare diseases, according to a report from the Pharmaceutical Research and Manufacturers of America.