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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...
Research
Germ-line and somatic DICER1 mutations in pineoblastomaThis study suggests that germ-line DICER1 mutations make a clinically significant contribution to PinB, establishing DICER1 as an important susceptibility...
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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IDH mutant high-grade gliomasGliomas are the most common type of malignant primary central nervous system (CNS) tumors, resulting in significant morbidity and mortality in children and adolescent and young adult (AYA) patients. The discovery of mutations in isocitrate dehydrogenase (IDH) genes has dramatically changed the classification and understanding of gliomas. IDH mutant gliomas have distinct clinical, pathological, and molecular features including a favorable prognosis and response to therapy compared to their wildtype counterparts.
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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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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.
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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.
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Incidence and survival for childhood cancer by endorsed non-stage prognostic indicators in AustraliaNick Gottardo MBChB FRACP PhD Head of Paediatric and Adolescent Oncology and Haematology, Perth Children’s Hospital; Co-head, Brain Tumour Research
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“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.