Pediatric Oncology

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.

As part of a resubmission plan for a biologics license application for omburtamab in pediatric patients with central nervous system and leptomeningeal metastasis from neuroblastoma, Y-mAbs, the manufacturer of the B7-H3–targeting monoclonal antibody, announced that it is collecting additional granularity data requested by the FDA and anticipates submitting them to the regulatory agency by the end of April 2021.

Adavosertib plus irinotecan showed early activity in a cohort of pediatric patients with neuroblastoma, thus meeting the protocol-defined efficacy end point of the 1/2 ADVL1312 trial.

Somatic KMT2A rearrangements, RAS/MAPK mutations, RUNX1 and TP53 alterations, and MECOM overexpression were all found to be common oncogenic drivers of pediatric therapy-related myeloid neoplasms, arising predominantly after exposure to cytotoxic therapy and identifiable at least 1 year prior to morphologic evidence of neoplasm.

The FDA has approved a revised label for daunorubicin/cytarabine (Vyxeos) to include the treatment of pediatric patients aged 1 year and older with newly diagnosed, therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes.

The European Commission has approved an expanded label for pembrolizumab for use as a single agent in the treatment of select adult and pediatric patients aged 3 years and older who have relapsed/refractory classical Hodgkin lymphoma.

Total body irradiation plus etoposide prior to hematopoietic stem cell transplantation resulted in improved overall survival and a lower risk of relapse in pediatric patients with high-risk acute lymphocytic leukemia compared with chemotherapy conditioning.

February 19, 2021 - The National Comprehensive Cancer Network has issued guidelines for the management of children with a rare type of kidney cancer referred to as Wilms Tumor, or nephroblastoma.

A reduced frequency of maintenance vincristine and dexamethasone led to a 5-year overall survival rate of 98.6% and improved quality of life in pediatric patients with newly diagnosed, standard-risk B-cell acute lymphoblastic leukemia.

Those diagnosed with von Hippel-Lindau disease have a resource for that complete and coordinated care in Roswell Park Comprehensive Cancer Center, which was recently named a VHL Clinical Care Center by the VHL Alliance.

VHL disease is defined as a rare, inherited disorder that causes tumors and cysts to grow in specific areas of the body, including the brain, spinal cord, eyes, inner ear, adrenal glands, pancreas, kidney, and reproductive tract.