Pediatric Oncology

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.

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.

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.

Accelerating progress in childhood cancer faces three main obstacles: funding, drugs, and family support.

The National Comprehensive Cancer Network is one of many organizations that is taking a stand against inequity in health care by aligning themselves with the new 3-year campaign, entitled “Close the Care Gap,” led by the Union for International Cancer Control.

The Biden-Harris administration has announced a plan to restart the Cancer Moonshot initiative, which President Joe Biden first launched as vice president of the Obama administration in 2016.

The FDA has granted a rare pediatric disease designation to the bone-targeting radiopharmaceutical, Samarium-153-DOTMP, for use as a potential therapeutic option for patients with osteosarcoma.

Chemoimmunotherapy is still a relatively new strategy for patients with pediatric neuroblastoma, and the humanized anti-disialoganglioside monoclonal antibody hu14.18K322A could provide a breakthrough for children with high-risk disease.

Leslie S. Kersun, MD, MSCE, MSEd, discusses finding the right fellowship program; the challenge of balancing research, patient care, and education as a trainee; and finding one’s focus during fellowship.

Two new studies by Yale Cancer Center reveal the structure of the molecule known as anaplastic lymphoma kinase, which is a driver of several cancers, including pediatric neuroblastoma, B-cell lymphomas, and myofibroblast tumors.

A rolling submission of a biologic license application to the FDA has been completed for betibeglogene autotemcel for use in adult, adolescent, and pediatric patients with beta-thalassemia who require regular red blood cell transfusions, across all genotypes.

The new drug application seeking the approval of a unique formulation of sodium thiosulfate for the prevention of ototoxicity induced by cisplatin chemotherapy in patients aged 1 month to less than 18 years of age with localized, nonmetastatic solid tumors has been resubmitted to the FDA.

YoungNa Lee-Kim, MD, shares advice on how to advance as an oncology fellow.

Natural killer T cells that co-express GD2-CAR and interleukin-15 were found to be safe and to demonstrate evidence of in vivo expansion and localization to metastatic sites in patients with stage IV relapsed/refractory neuroblastoma.

In a new study led by Yale Cancer Center, researchers report many survivors of childhood cancers receive systemic therapies associated with cognitive effects and chronic health conditions that may impact long-term cognitive outcomes with downstream effects on education, employment, and income.

The FDA has granted a priority review designation to SH-111, a sterile injectable therapy, for the treatment of pediatric patients with T-cell leukemia.