The OncLive Pediatric Oncology condition center page is a comprehensive resource for clinical news and expert insights on various types of cancers in pediatric patients, including acute lymphoblastic leukemia, acute myeloid leukemia, brain cancers, sarcomas, and more. This page features news articles, interviews in written and video format, and podcasts that focus on unmet needs, treatment advances, and ongoing research in childhood and adolescent and young adult cancers.
March 16th 2023
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.
October 17th 2022
FDA Grants Orphan Drug Designation to Paxalisib for Atypical Rhabdoid, Teratoid TumorsJune 20th 2022
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.
Dabrafenib Plus Trametinib Quadruples Response Rate in BRAF V600+ Pediatric Low-Grade Glioma
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.
FDA Approval Sought for Ibrutinib in Pediatric Chronic Graft-Versus-Host DiseaseFebruary 28th 2022
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.
NCCN Voices a Call to Action Against Health Care Inequity on World Cancer DayFebruary 8th 2022
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.
FDA Grants Rare Pediatric Disease Designation to Novel Radiopharmaceutical for OsteosarcomaFebruary 2nd 2022
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.
Activating the Immune System Could be Key to Treating Pediatric NeuroblastomaJanuary 11th 2022
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.
Yale Cancer Center Researchers Show Receptor Structure Reveals New Targets for Cancer TreatmentDecember 10th 2021
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.
FDA Approval Sought for Betibeglogene Autotemcel for Transfusion-Dependent Beta-ThalassemiaSeptember 22nd 2021
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.
NDA Resubmitted to FDA for Pedmark in Prevention of Cisplatin-Induced Ototoxicity in Solid TumorsJune 1st 2021
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.
NKT Cells Co-Expressing GD2-CAR, IL-15 Can Induce Complete Remissions in Pediatric NeuroblastomaMay 15th 2021
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.
Yale Cancer Center Study Shows Cognitive Impairment for Survivors of Many Pediatric Cancers
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.
Resubmission Planned for Omburtamab in Pediatric Metastatic NeuroblastomaApril 20th 2021
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.
Genomic Sequencing Identifies Pediatric Therapy-Related Myeloid Neoplasm Defining Variants Prior to Clinical PresentationApril 5th 2021
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.
FDA Approves Daunorubicin/Cytarabine for Pediatric Secondary AMLMarch 30th 2021
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.
Pembrolizumab Approved in Europe for Adult and Pediatric Relapsed/Refractory Classical Hodgkin LymphomaMarch 17th 2021
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 Improves Survival in Select Transplant-Eligible Pediatric High-Risk ALLFebruary 25th 2021
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.
Reduced Vincristine/Dexamethasone Dosing Schedule Shows Impressive Outcomes, Improved QoL in Pediatric B-ALLFebruary 19th 2021
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.
Roswell Park Is First Site in Region Named a Resource for Patients With Rare Genetic DiseaseFebruary 17th 2021
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: Monitoring Is Imperative, But Research Efforts Are Gaining GroundFebruary 16th 2021
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.