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Top advances of the year: Pediatric oncology

CANCER (2022)

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Article Oncology

Improved Outcome in Children With Newly Diagnosed High-Risk Neuroblastoma Treated With Chemoimmunotherapy: Updated Results of a Phase II Study Using hu14.18K322A

Wayne L. Furman et al.

Summary: Combining hu14.18K322A with chemotherapy improves early response and outcomes in high-risk neuroblastoma. This treatment is well-tolerated and reduces tumor volume in most patients, leading to improved end-of-induction response rates and encouraging 3-year event-free survival.

JOURNAL OF CLINICAL ONCOLOGY (2022)

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Article Oncology

Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial

D. Williams Parsons et al.

Summary: The National Cancer Institute-Children's Oncology Group Pediatric MATCH trial has successfully implemented a nationwide screening protocol to identify actionable genetic alterations in pediatric and young adult patients with cancer, assigning them to appropriate trials of molecularly targeted therapies. These findings support the early use of tumor molecular screening in childhood cancer patients who have not responded to standard treatments.

JOURNAL OF CLINICAL ONCOLOGY (2022)

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Article Hematology

Tumor-intrinsic and -extrinsic determinants of response to blinatumomab in adults with B-ALL

Yaqi Zhao et al.

Summary: This study investigated the efficacy of blinatumomab in patients with B-progenitor acute lymphoblastic leukemia (B-ALL) and found that both tumor-intrinsic and -extrinsic factors influence blinatumomab response. CD19 mutations were commonly detected in CD19(-) relapse during blinatumomab treatment, and the identification of the CD19 ex2part splice variant represents a new biomarker predictive of blinatumomab therapy failure.

BLOOD (2021)

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Article Oncology

Serial assessment of measurable residual disease in medulloblastoma liquid biopsies

Anthony P. Y. Liu et al.

Summary: The study demonstrates the clinical utility of CSF-derived cell-free DNA (cfDNA) as a biomarker of measurable residual disease (MRD) in children with medulloblastoma. Detection of MRD, particularly in patients with persistent MRD, is associated with a higher risk of disease progression. Early detection of MRD using liquid biopsies may aid in monitoring disease progression and treatment efficacy in medulloblastoma patients.

CANCER CELL (2021)

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Article Oncology

Outcomes by Clinical and Molecular Features in Children With Medulloblastoma Treated With Risk-Adapted Therapy: Results of an International Phase III Trial (SJMB03)

Amar Gajjar et al.

Summary: The study identified new risk stratification for medulloblastoma treatment through molecular classification and risk factor analysis, finding differences in PFS among patients with different genotypes and subtypes.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Article Medicine, General & Internal

Effect of Postreinduction Therapy Consolidation With Blinatumomab vs Chemotherapy on Disease-Free Survival in Children, Adolescents, and Young Adults With First Relapse of B-Cell Acute Lymphoblastic Leukemia A Randomized Clinical Trial

Patrick A. Brown et al.

Summary: In children, adolescents, and young adults with high- and intermediate-risk first relapse of B-ALL, postreinduction treatment with blinatumomab compared with chemotherapy, followed by transplant, did not result in a statistically significant difference in disease-free survival. However, study interpretation is limited by early termination with possible underpowering for the primary end point.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2021)

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Article Medicine, General & Internal

Effect of Blinatumomab vs Chemotherapy on Event-Free Survival Among Children With High-risk First-Relapse B-Cell Acute Lymphoblastic Leukemia A Randomized Clinical Trial

Franco Locatelli et al.

Summary: This study compared the effects of blinatumomab with consolidation chemotherapy as a third consolidation block before allogeneic hematopoietic stem cell transplant in children with high-risk first-relapse B-ALL. The results showed that, compared to chemotherapy, treatment with blinatumomab significantly improved event-free survival in children with high-risk first-relapse B-ALL.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2021)

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Article Oncology

Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data

Md. Jobayer Hossain et al.

Summary: Age, race, primary tumor sites, grades, and histological types are closely related to the survival of pediatric brain tumor patients. High-grade tumor types and specific tumor sites increase the risk of mortality, while poverty level and geographic region also play a role in survival rates.

CANCER EPIDEMIOLOGY (2021)

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Article Oncology

Genomes for Kids: The Scope of Pathogenic Mutations in Pediatric Cancer Revealed by Comprehensive DNA and RNA Sequencing

Scott Newman et al.

Summary: This study utilized a three-platform sequencing approach, including WGS, WES, and RNAseq, to examine tumor and germline genomes from 309 children with pediatric cancers. The study revealed the presence of diverse genetic variants in most patients, with WGS helping to detect activating gene fusions, enhancer hijacks, small intragenic deletions, and mutational signatures. The evaluation of paired tumor-normal data showed the relevance of pathogenic germline variants to tumor development in a significant portion of cases.

CANCER DISCOVERY (2021)

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Article Oncology

Efficacy of Carboplatin and Isotretinoin in Children With High-risk Medulloblastoma A Randomized Clinical Trial From the Children's Oncology Group

Sarah E. S. Leary et al.

Summary: In a randomized clinical trial focusing on high-risk group 3 medulloblastoma, intensifying therapy with carboplatin was shown to improve 5-year event-free survival by 19%. These results highlight the importance of integrated clinical and molecular risk stratification for medulloblastoma.

JAMA ONCOLOGY (2021)

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Article Hematology

Myeloablative Busulfan/Melphalan Consolidation following Induction Chemotherapy for Patients with Newly Diagnosed High-Risk Neuroblastoma: Children's Oncology Group Trial ANBL12P1

M. Meaghan Granger et al.

Summary: BuMel ASCT following COG induction for high-risk neuroblastoma in children is well tolerated, with low rates of toxicity during consolidation and no deaths during this period. The 3-year event-free survival and overall survival rates are favorable for eligible patients.

TRANSPLANTATION AND CELLULAR THERAPY (2021)

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Article Medicine, General & Internal

Effect of Tandem Autologous Stem Cell Transplant vs Single Transplant on Event-Free Survival in Patients With High-Risk Neuroblastoma A Randomized Clinical Trial

Julie R. Park et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2019)

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Article Hematology

Reducing minimal residual disease with blinatumomab prior to HCT for pediatric patients with acute lymphoblastic leukemia

Amy K. Keating et al.

BLOOD ADVANCES (2019)

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Article Hematology

Blinatumomab for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukemia

Nicola Goekbuget et al.

BLOOD (2018)

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Article Medicine, General & Internal

Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia

Hagop Kantarjian et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Oncology

Phase I/Phase II Study of Blinatumomab in Pediatric Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia

Arend von Stackelberg et al.

JOURNAL OF CLINICAL ONCOLOGY (2016)

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Review Oncology

Genomics in acute lymphoblastic leukaemia: insights and treatment implications

Kathryn G. Roberts et al.

NATURE REVIEWS CLINICAL ONCOLOGY (2015)

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Article Oncology

Pilot Induction Regimen Incorporating Pharmacokinetically Guided Topotecan for Treatment of Newly Diagnosed High-Risk Neuroblastoma: A Children's Oncology Group Study

Julie R. Park et al.

JOURNAL OF CLINICAL ONCOLOGY (2011)

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Article Medicine, General & Internal

Anti-GD2 Antibody with GM-CSF, Interleukin-2, and Isotretinoin for Neuroblastoma.

Alice L. Yu et al.

NEW ENGLAND JOURNAL OF MEDICINE (2010)

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