Pediatric Oncology

Eltrombopag (Promacta) boosted the platelet count in a 13-year-old boy with HIV-associated thrombocytopenia, according to case study results presented at the 2018 American Society of Pediatric Hematology/Oncology Conference.

Elissa Engel, MD, pediatric resident, University of South Florida, discusses the successful use of eltrombopag (Promacta) in a pediatric patient with HIV-related thrombocytopenia.

Tazemetostat demonstrated promising antitumor activity in pediatric patients with INI1-negative solid tumors.

Susan N. Chi, MD, senior physician, director, Pediatric Brain Tumor Clinical Trials Program, Dana-Farber Cancer Institute, assistant professor of Pediatrics, Harvard Medical School, discusses a phase I multicenter trial of tazemetostat in children with INI1-negative tumors.

Kelly Bailey, MD, PhD, physician, department of Pediatric Hematology/Oncology, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, discusses key molecular pathways in pediatric bone sarcoma.

Brian D. Crompton, MD, physician, Pediatric Hematology/Oncology, Dana-Farber Cancer Institute, assistant professor of pediatrics, Harvard Medical School, discusses the utilization of liquid biopsies in pediatric patients with sarcoma.

Adolescent and young adult patients with cancer placed a high importance on prognostic information, and obtaining that knowledge was associated with improved well-being.

The FDA has halted enrollment on clinical trials of tazemetostat in patients with various solid tumors and hematologic malignancies.

Kristopher Bosse, MD, attending physician, Division of Oncology, The Children's Hospital of Philadelphia, discusses the treatment of pediatric patients with high-risk neuroblastoma.

A novel risk stratification and treatment approach led to enhanced clinical outcomes overall, as well as the successful omission of radiotherapy for some patients with stage IV favorable histology Wilms tumor and pulmonary metastases.

Rosandra N. Kaplan, MD, investigator, Pediatric Oncology Branch, Head, Tumor Microenvironment Section, National Cancer Institute, discusses the treatment of pediatric patients with solid tumors.

Ramamoorthy Nagasubramanian, MD, discusses the results of the phase I/II study of larotrectinib in pediatric patients.

Larotrectinib induced an “unprecedented” objective response rate of 93% in patients with TRK fusion–positive solid tumors.

A rolling new drug application has been completed for larotrectinib (LOXO-101) for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion.

The FDA has approved nilotinib for the first- and second-line treatment of pediatric patients aged 1 year and older with Philadelphia chromosome–positive chronic myeloid leukemia in the chronic phase.

IKZF1 deletions that co-occurred with deletions in CDKN2A, CDKN2B, PAX5, or PAR1 in the absence of ERG deletion (IKZF1plus) were associated with a dramatically reduced prognosis in pediatric patients with B-cell precursor ALL.

Larotrectinib (LOXO-101) induced durable responses in patients with TRK fusion–positive solid tumors, according to updated results for the novel pan-TRK inhibitor.

Mark E. Hatley, MD, discusses the research of endothelial progenitor cells in pediatric patients with rhabdomyosarcoma and how it can lead to thorough analyses of the tumor cells.

One out of 6 survivors of childhood cancer was unemployed, according to results from a meta-analysis of 56 studies conducted in the United States, Canada, Asia, and Europe.

At a median follow-up of 13.1 months, tisagenlecleucel (Kymriah) induced an overall remission rate of 81% in children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia.

The FDA has halted 4 US studies of BPX-501, a novel cellular immunotherapy for cancers and orphan inherited blood disorders, after 3 patients developed encephalopathy possibly related to treatment with BPX-501.

The method of treatment administration in the JACLS ALL-97 and ALL-02 trials may have contributed to a lower incidence of osteonecrosis in Japanese children with acute lymphoblastic leukemia.

The presence of a specialized pediatric palliative care team lowered the odds that a child with cancer would be admitted to an intensive care unit at the end of life by five-fold compared with those who did not receive palliative care, according to results from a retrospective study.

Ipilimumab has been approved by the European Commission for the treatment of patients aged 12 and older with unresectable or metastatic melanoma.

The FDA has granted a priority review to the CAR T-cell therapy tisagenlecleucel for adult patients with relapsed/refractory DLBCL who are ineligible for or relapse after autologous stem cell transplant.

Children with TP53 pathogenic variants were more likely to both develop acute lymphoblastic leukemia, as well as secondary cancers later on, according to a recent analysis.

In patients with stage III favorable-histology Wilms tumor, negative nodal involvement and the absence of loss of heterozygosity at chromosomes 1p and 16q were predictors for event-free survival and overall survival.

Charalambos (Babis) Andreadis, MD, MSCE, associate professor of clinical medicine, Department of Medicine, UCSF Helen Diller Family Comprehensive Cancer Center, discusses the role of chimeric antigen receptor (CAR) T-cell therapy for pediatric patients with acute lymphocytic leukemia (ALL).

Loxo Oncology has initiated a rolling submission of data for a NDA to the FDA for the novel pan-TRK inhibitor larotrectinib (LOXO-101) as a treatment for adult and pediatric patients with TRK fusion-positive advanced solid tumors.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended approval of ipilimumab for the treatment of pediatric patients aged 12 years and older with unresectable or metastatic melanoma.