Pediatric Cancers

Phase 1 data may support the potential activity and safety of VCN-01 among pediatric patients with refractory retinoblastoma.

The agency has set a Prescription Drug User Fee Act date of January 7, 2025, for its decision on approving remestemcel-L in this patient population.

Upon acceptance, the FDA will review the resubmitted application between 2 and 6 months for remestemcel-L in pediatric patients.

The FDA approval of the single-agent, oral pan-RAF inhibitor tovorafenib is based on efficacy results from the phase 2 FIREFLY-1 trial.

Findings from an open-label trial support the FDA approval of inotuzumab ozogamicin as a treatment for pediatric patients with relapsed/refractory acute lymphoblastic leukemia.

Administering pegylated asparaginase continuously to pediatric patients with acute lymphoblastic leukemia appears to be safe without compromising the efficacy of treatment.

FDA initiatives such as the Food and Drug Omnibus Reform Act will set goals and specify measures for improving representation in pediatric cancer clinical studies.

Investigators report that prediabetes may prove to be a target for intervention to decrease morbidity and mortality in survivors of pediatric cancer.

Eflornithine, which was approved in adult and pediatric patients with high-risk neuroblastoma, was assessed in an externally controlled trial comparing results from Study 3b and Study ANBL0032.

Patients who are under the age of 17 with invasive mucormycosis or invasive aspergillosis, which is an AE of leukemia, can now receive isavuconazonium sulfate following FDA approval

The FDA has set a Prescription Drug User Fee Act date of April 30, 2024 for tovorafenib as a treatment for pediatric patients with relapsed or progressive low-grade glioma.

Based on findings from the phase 1/2 BCHILD trial, the FDA approved bosutinib for pediatric chronic myelogenous leukemia.

Findings from a cross-sectional study support monitoring puberal development to estimate probabilities of future fertility among male childhood cancer survivors.

Increasing radiation doses to the whole heart appear to correlate with higher risks of valvular disease, coronary artery disease, and heart failure in childhood cancer survivors.

Investigators of a prospective study of 1253 patients find that outcomes with the experimental homoharringtonine-based regimen either meet or exceed those with a classic etoposide-based regimen.

Investigators will work with the FDA to assess the root cause of a grade 5 serious adverse effect in phase 2 PLAT-08 trial evaluating SC-DARIC33 in pediatric acute myeloid leukemia.

Data from a phase 2 trial support a supplemental new drug application for isavuconazonium sulfate as a treatment for pediatric patients with invasive aspergillosis or invasive mucormycosis.

Investigators plan to launch a controlled trial assessing remestemcel-L in the highest-risk adults with steroid-refractory acute graft-versus-host-disease to meet the FDA’s request for additional data.

Data from a phase 2 trial assessing lenalidomide in patients under 22 years of age also highlight an optimal dose level for this population.

Use of the Cumulative Illness Rating Scale for Geriatrics may identify a greater comorbidity burden and a commensurate increase in risk of death among survivors of childhood cancer vs siblings and others.

Investigators pause their evaluation of SC-DARIC33 in pediatric relapsed/refractory acute myeloid leukemia following a grade 5 serious adverse effect in the phase 1 PLAT-08 trial.

Adding blinatumomab to Interfant-06 chemotherapy appears to be feasible and safe in the treatment infants with acute lymphoblastic leukemia in a phase 2 trial.

Treatment with tovorafenib appears well tolerated among pediatric patients with low-grade glioma, according to findings from the phase 2 FIREFLY trial.

Patients under the age of 3 with neuroblastoma experienced no significant negative impact on survival when their disease was reclassified from high-risk to intermediate-risk and their therapy was thusly reduced.

A phase 2 trial indicates that limited surgery and post-operative proton therapy result in a high rate of tumor control and less severe complications in young patients with craniopharyngioma.

Investigators of a phase 1/2 trial report that CAR T cells targeting disialoganglioside GD2 may result in long-lasting antitumor activity in young patients with high-risk neuroblastoma.

Investigators highlight the importance of building partnerships between CAR T-cell centers and external hospitals to increase treatment referrals and access to clinical trials for patients with B-cell acute lymphoblastic leukemia.

Findings from 2 phase 3 trials support the recommendation to approve sodium thiosulfate to reduce the risk of cisplatin-related hearing loss in pediatric patients with solid tumors in Europe.

Findings from a cohort of the Childhood Cancer Survivor Study identify a prediction model that may accurately identify childhood cancer survivors at varying risks of late kidney failure.

Results from the phase 2 CDRB436G2201 trial lead to the approval of dabrafenib plus trametinib in patients with low-grade glioma and a BRAF V600E mutation who require systemic therapy.