Pediatric Oncology

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.

Thomas E. Merchant, DO, PhD, discusses the incidence of craniopharyngioma.

Anne Angiolillo, MD, discusses the adverse effects that are associated with traditional treatment in pediatric B-cell acute lymphoblastic leukemia.

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.

Kim E. Nichols, MD, discusses the prognosis of patients with von Hippel-Lindau disease.

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.

YunZu Michele Wang, MD, discusses the potential utility of ruxolitinib in children and young adult patients with chronic graft-versus-host disease.

February 10, 2021 - Treatment with the investigative gene cell therapy betibeglogene autotemcel led to durable transfusion independence in 87% of pediatric patients less than 18 years of age with transfusion-dependent beta-thalassemia with a median average hemoglobin of 11.3 g/dL.

The landscape for pediatric oncology drugs expanded dramatically last year, with 8 new drugs or indications specifically approved for children compared with just 47 for treatment and supportive care products from the early 1950s through 2019.

February 2, 2021 - Emil J. Freireich, MD, DSc, a founding father of modern clinical cancer research and the 2015 Giants of Cancer Care award winner for Lymphoid Neoplasms, has died at age 93.

February 1, 2020 - The European Medicines Agency’s Committee of Medicinal Products for Human Use has recommended a label expansion for pembrolizumab for use as a single agent in adult and pediatric patients aged 3 years and older with relapsed/refractory classical Hodgkin lymphoma for whom autologous stem cell transplant has failed or following at least 2 previous therapies when ASCT is not an option.

Erin Murphy, MD, discusses the utility of pazopanib plus stereotactic body radiation therapy in pediatric sarcoma.

Katianne M. Howard Sharp, PhD, discusses the importance of racial and ethnic diversity in clinical trials for pediatric patients with cancer.

Joseph V. Simone, MD, a pioneering clinical investigator and institutional leader in pediatric oncology who was recognized with a 2017 Giants of Cancer Care® award, died January 21 at the age of 85.

December 23, 2020 — Findings from the International, Prospective ALCL-Relapse trial showed that allogeneic SCT after reinduction chemotherapy can lead to survival improvements in children and adolescents with high-risk relapsed or refractory anaplastic large cell lymphoma, provide more insight into how to treat this population.

The use of abdominal or pelvic radiotherapy in pediatric patients with cancer could lead to long-term negative impact on cardiovascular and metabolic health.

TP53 was found to be the only gene with a germline de novo pathogenic or likely pathogenic variant in pediatric patients with osteosarcoma.

The FDA has granted an orphan drug designation and rare pediatric disease designation to the bispecific antibody nivatrotamab for the treatment of patients with neuroblastoma.

Despite the numerous reports detailing racial differences in cancer outcomes and care over the years, the reasons underlying these disparities continue to be under deep exploration.

The FDA’s Oncologic Drugs Advisory Committee voted 8 to 2 in favor of approving remestemcel-L for the treatment of children with steroid-refractory acute graft-versus-host disease.

Numerous analyses conducted over the past few years have underscored a serious issue in clinical cancer trials that needs to be addressed: a lack of racial and ethnic diversity among participants.

The FDA has issued a complete response letter to Fennec Pharmaceuticals regarding its new drug application for a unique formulation of sodium thiosulfate (Pedmark) for the prevention of cisplatin-induced ototoxicity in patients from 1 month to less than 18 years of age with localized, nonmetastatic, solid tumors.

The FDA has granted a priority review designation to a new drug application for a unique formulation of sodium thiosulfate for the prevention of cisplatin-induced ototoxicity in patients from 1 month to less than 18 years of age with localized, nonmetastatic, solid tumors.