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Novel BRD4-NUT fusion isoforms increase the pathogenic complexity in NUT midline carcinomaThis study contributes to our understanding of the genetic diversity of NMC, an important step towards finding therapeutic targets for a disease that is...
Research
Molecular characterization of identical, novel MLL-EPS15 translocation and individual genomicAcute Lymphoblastic Leukemia (ALL) occurring in the first year of life is rare.
Research
Acute Lymphoblastic Leukemia in Infants: A Distinctive, High-Risk Subtype of Childhood Acute Lymphoblastic LeukemiaAcute lymphoblastic leukemia (ALL) in infants younger than 1 year of age is an aggressive, high-risk subtype of childhood ALL. Infant ALL with KMT2A-r is characteristically poorly responsive to chemotherapy and hematopoietic stem cell transplantation. New strategies, such as molecularly targeted therapies and immunotherapies, are in development and show promise in preclinical models and early phase studies.
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Exploiting the reactive oxygen species imbalance in high-risk paediatric acute lymphoblastic leukaemia through auranofinThe prognosis for high-risk childhood acute leukaemias remains dismal and established treatment protocols often cause long-term side effects in survivors. This study aims to identify more effective and safer therapeutics for these patients.
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Genome-wide association meta-analysis of single-nucleotide polymorphisms and symptomatic venous thromboembolism during therapy for ALL and lymphoma in caucasian childrenThe largest GWAS meta-analysis conducted to date associating SNPs to venous thromboembolism in children and adolescents treated on childhood ALL protocols
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Blinatumomab Added to Chemotherapy in Infant Lymphoblastic LeukemiaKMT2A-rearranged acute lymphoblastic leukemia (ALL) in infants is an aggressive disease with 3-year event-free survival below 40%. Most relapses occur during treatment, with two thirds occurring within 1 year and 90% within 2 years after diagnosis. Outcomes have not improved in recent decades despite intensification of chemotherapy.
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Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndromeChildren with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population.
Research
Pediatric pineoblastoma: A pooled outcome study of North American and Australian therapeutic dataPineoblastoma is a rare brain tumor usually diagnosed in children. Given its rarity, no pineoblastoma-specific trials have been conducted. Studies have included pineoblastoma accruing for other embryonal tumors over the past 30 years.
Research
Silencing of GATA3 defines a novel stem cell-like subgroup of ETP-ALLGATA3low ETP-ALL is a novel stem cell-like leukemia with implications for the use of myeloid-derived therapies