Pediatric Oncology

The FDA’s Oncologic Drugs Advisory Committee has scheduled a date to review data supporting the biologics license application for remestemcel-L as a treatment for children with steroid-refractory acute graft versus host disease.

Following the arrival of the coronavirus disease 2019 pandemic, St. Jude Children's Research Hospital launched the first Global COVID-19 Observatory and Resource Center for Childhood Cancer.

The FDA has granted a priority review designation to a biologics license application for the investigational, humanized monoclonal antibody naxitamab (Danyelza) for the treatment of patients with relapsed/refractory high-risk neuroblastoma.

The use of real-time comprehensive profiling provides valuable diagnostic information and identifies potential therapeutic targets in adults with malignancies, but this process remains widely underutilized for pediatric patients and represents a significant unmet need.

A Biologics License Application has been filed with the FDA for naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma.

Higher doses of anthracyclines are associated with increased risk of breast cancer in women who survived childhood cancer, regardless of whether they have mutations that predispose them to cancer.

Cornelis M. van Tilburg, MD, PhD, pediatric oncologist, Hopp Children’s Cancer Center Heidelberg, discusses the activity of larotrectinib (Vitrakvi) in patients with TRK fusion–positive cancers who have brain metastases or primary central nervous system tumors.

The efficacy of larotrectinib is supported in distinct patient populations with TRK fusion cancer—specifically pediatric patients and adult or pediatric patients with brain metastases or primary central nervous system tumors.

John Sweetenham, MD, associate director for clinical affairs, Harold G. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, discusses unanswered questions with CAR T-cell therapy in pediatric acute lymphoblastic leukemia.

Entrectinib induced objective responses in 100% of pediatric patients with tumors harboring NTRK1/2/3, ROS1, or ALK gene fusions or mutations.

About a quarter of pediatric patients with cancer were able to be screened for targetable alterations in their tumor and directed to a treatment targeting that alteration of pathway in the screening protocol of the National Cancer Institute-Children’s Oncology Group Pediatric MATCH trial.

Christine N. Duncan, MD, discusses the current components of CAR T-cell therapy in pediatric acute lymphoblastic leukemia.

Yadav Sapkota, PhD, clinical research scientist at St. Jude Children’s Research Hospital, discusses the relationship between a genetic variant and the risk of stroke in childhood cancer survivors treated with cranial radiation therapy.

The development of high-grade acute graft-versus-host disease following hematopoietic stem cell transplantation poses the highest risk for transplant-associated thrombotic microangiopathy in pediatric patients.

Childhood cancer survivors who had lived 5 or more years from their primary malignancy are at an increased risk of developing skin cancer compared with the general population.

Childhood cancer is significantly underdiagnosed—especially in south Asia and sub-Saharan Africa—underscoring the need for stronger health systems and the expansion of universal health coverage, according to an analysis published in Lancet Oncology.

Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses the implications of a matched targeted therapy approach in pediatric leukemia.

The European Commission has approved dasatinib in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.

The relative risk of developing a solid subsequent malignant neoplasm remained elevated in survivors of childhood Hodgkin lymphoma, according to extended follow-up of more than 25 years from the Late Effects Study Group cohort.

The FDA has approved dasatinib tablets in combination with chemotherapy for the treatment of pediatric patients ≥1 year of age with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.

Corinne Summers, MD, pediatric oncologist, Seattle Children's Hospital, assistant member, Clinical Research Division, University of Washington Fred Hutchinson Cancer Research Center, discusses the role of CAR T cells in pediatric cancer.

Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses a trial attempting to match pediatric patients with more precise therapy for acute leukemia.

The FDA has approved a longer-acting version of calaspargase pegol-mknl (Asparlas) as a component of a multiagent chemotherapy regimen for pediatric and young adult patients aged 1 month to 21 years with acute lymphoblastic leukemia.

Yana Pikman, MD, discusses the clinical implications of using an approach to match targeted therapy to pediatric patients with leukemia.

Stephan Grupp, MD, PhD, director, Cancer Immunotherapy Program, director, Translational Research for the Center for Childhood Cancer Research, medical director, Stem Cell Laboratory, Children’s Hospital of Philadelphia, discusses the findings of the ELIANA trial.

The European Medicines Agency's Committee for Medicinal Products for Human Use has granted a positive opinion to dasatinib in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.

The FDA has approved romiplostim for the treatment of pediatric patients aged ≥1 year with immune thrombocytopenia for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Santhosh Upadhyaya, MD, neuro-oncologist, St. Jude Children’s Research Hospital, discusses drug development for pediatric patients with brain tumors.

Briana Patterson, MD, discusses the off-target effects of tyrosine kinase inhibitors in pediatric patients with chronic myeloid leukemia.

Michael Pulsipher, MD, director of cellular therapy and stem cell transplantation, professor of pediatrics, USC Keck School of Medicine, discusses the role of MRD testing in pediatric patients treated with CAR T-cell therapy.