11:30 am – 12:30 pm Saturday, September 15

Therapeutic & Prognostic Impact of Medulloblastoma Molecular Subgroups (227)

1CNE  Medulloblastoma is an embryonal tumor of the central nervous system (CNS). It is the most common malignant CNS tumor in children with peak incidence between 5–9 years of age. Patients typically present with cerebellar deficits and headaches with vomiting, specifically in the mornings. Prognosis varies according to multiple factors including histology, age at diagnosis, and metastases. Medulloblastoma treatment is an intense, multi-modality therapy that can have many resulting late effects. Recent findings have shown medulloblastoma to be a heterogeneous disease which includes multiple subgroups.

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3:15 – 4:15 pm Friday, September 14

New Hope Through Clinical Trials in Low Grade Gliomas: From Diagnosis, Standard Treatment Modalities, to BRAF/MEK Inhibitors (221)

1CNE  Low grade gliomas (LGG) are the most common type of pediatric brain tumor (Jones et al., 2017). Depending upon tumor location, many patients undergo surgical resection. If a gross total resection is achieved, patients receive MRI imaging. However, patients who have an incomplete resection or a recurrence receive additional therapy. Young patients with developing brains receive chemotherapy. Older patients may receive radiation. There are multiple agents or combination of agents that are administered in patients with LGG. Standard frontline chemotherapies are either Carboplatin and Vincristine or TPCV (Thioguanine, Lomustine, Procarbazine, and Vincristine). Other traditional single-agent therapies include Temodar, Vinblastine, or Vinorelbine. BRAF/MEK inhibitors (trametinib, dabrafenib, vemurafenib, and selumetinib) are currently being used in clinical trials (Penman, Faulkner, Lowis, & Kurian, 2015).

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3:15 – 4:15 pm Friday, September 14

Running on Empty: Inherited Bone Marrow Failure (219)

1CNE Inherited bone marrow failure syndromes are a diverse group of hematologic disorders. Despite the name, some children have new, not inherited, genetic mutations. These mutations can result in congenital abnormalities or disease in specific organs or increased cancer predisposition. Through a series of patient vignettes we will follow six patients with common syndromes highlighting clinical presentation, diagnostic evaluation, and evidence-based treatment and monitoring recommendations. We also will explore the impact of a genetic diagnosis on the psychological and social well-being of families as the parents grapple with the decision whether to pursue genetic testing for themselves or their other children.

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