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Interactions between acute lymphoblastic leukemia and bone marrow stromal cells influence response to therapyThe cure rate for pediatric patients with B precursor acute lymphoblastic leukemia (pre-B ALL) is steadily improving, however relapses do occur despite...
Research
Hepatic sinusoidal obstruction syndrome during chemotherapy for childhood medulloblastoma: Report of a case and review of the literatureWe report a patient with high-risk anaplastic medulloblastoma who developed severe HSOS during her second cycle of maintenance chemotherapy.
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Comparative drug screening in NUT midline carcinomaThe NUT midline carcinoma (NMC) is a rare but fatal cancer for which systematic testing of therapy options has never been performed.
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Idiosyncratic nature of voriconazole photosensitivity in children undergoing cancer therapyIn adults, the unpredictability of voriconazole pharmacokinetics, particularly in those patients receiving chemotherapy, is well recognised. A paucity of...
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Long-Term Outcome of Young Infants With Suspected Neuroblastoma following Observation as Primary Therapy: A Report From the Children's Oncology GroupSeveral studies have established that patients with localized perinatal neuroblastoma can be safely observed; however, long-term outcomes have not been previously reported. We evaluated long-term outcomes of infants with suspected perinatal neuroblastoma enrolled on the Children's Oncology Group ANBL00P2, which included an expectant observation approach.
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Comments and Controversies in Oncology: The Tribulations of Trials Developing ONC201Our international team highlights issues with efficacy reports in several studies on DMG with the new drug ONC201.
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Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trialDue to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.
Research
“If you build it, they will come”: the convergence of funding, research and collaboration in paediatric brain cancer clinical trialsEach year, approximately 1000 children in Australia and New Zealand, aged 0–14 years, are diagnosed with cancer. Despite paediatric cancer accounting for less than 1% of all cancer cases, the impact on their families and communities is profound and disproportionate.
Research
Characteristics of patients ≥10 years of age with diffuse intrinsic pontine glioma: a report from the International DIPG/DMG RegistryDiffuse intrinsic pontine gliomas generally occur in young school-age children, although can occur in adolescents and young adults. The purpose of this study was to describe clinical, radiological, pathologic, and molecular characteristics in patients ≥10 years of age with DIPG enrolled in the International DIPG Registry.
Research
PI3K/mTOR is a therapeutically targetable genetic dependency in diffuse intrinsic pontine gliomaDiffuse midline glioma (DMG), including tumors diagnosed in the brainstem (diffuse intrinsic pontine glioma; DIPG), are uniformly fatal brain tumors that lack effective treatment.