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Efficacy of acute myeloid leukemia therapy without stem-cell transplantation in a child with blastic plasmacytoid dendritic cell neoplasmOur case demonstrates that AML therapy, without HSCT, can be sufficient to treat this rare disease in children.
Research
Glioma-specific Domain IV EGFR cysteine mutations promote ligand-induced covalent receptor dimerization and display enhanced sensitivityEpidermal growth factor receptor (EGFR) is over-expressed in many brain tumors. This paper examines mutations the EGFR that make the cell it is produced in...
We strive for a future where no child will die from brain cancer because we have developed new therapies that will cure their disease.
Research
The evolution of clinical trials for infant acute lymphoblastic leukemiaDespite initial improvements in survival of infants with ALL since establishment of the first pediatric cooperative group ALL trials, the poor outcome has...
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Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene FusionsTropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients.
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Precision-guided treatment in high-risk pediatric cancersRecent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment.
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Stability and change in self-reported risk and resilience factors associated with mental health of siblings of individuals with and without neurodevelopmental conditions over 15 monthsSiblings of individuals with neurodevelopmental conditions (NDCs) are a minority population at higher genetic and environmental risk of poorer neurocognitive and psychosocial outcomes compared to siblings of individuals without NDCs.
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Prognostic significance of molecular subgroups in survival outcome for children with medulloblastoma in MalaysiaAdvancements in genomic profiling led to the discovery of four major molecular subgroups in medulloblastoma (MB), which have now been incorporated into the World Health Organization classification of central nervous system tumors. The current study aimed to determine the prognostic significance of the MB molecular subgroups among children in Malaysia.
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Grandparents’ Experiences of Childhood Cancer: A Qualitative StudyA child's cancer diagnosis has a significant impact on the lives of grandparents. Grandparents experience the stress of worrying about both their adult children and their grandchildren. Our study aimed to explore the lived experience of grandparents of children diagnosed with cancer.
Research
Small-molecule screen reveals synergy of cell cycle checkpoint kinase inhibitors with DNA-damaging chemotherapies in medulloblastomaMedulloblastoma (MB) consists of four core molecular subgroups with distinct clinical features and prognoses. Treatment consists of surgery, followed by radiotherapy and cytotoxic chemotherapy. Despite this intensive approach, outcome remains dismal for patients with certain subtypes of MB, namely, MYC-amplified Group 3 and TP53-mutated SHH. Using high-throughput assays, six human MB cell lines were screened against a library of 3208 unique compounds. We identified 45 effective compounds from the screen and found that cell cycle checkpoint kinase (CHK1/2) inhibition synergistically enhanced the cytotoxic activity of clinically used chemotherapeutics cyclophosphamide, cisplatin, and gemcitabine.