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Craniospinal irradiation (CSI) is a cornerstone of pediatric brain cancer therapy, yet detailed, reproducible protocols for accurate CSI delivery in preclinical mouse models remain scarce, hindering translational research and the development of radiosensitizing strategies. We aimed to establish a clinically relevant, easily replicable radiation therapy protocol for medulloblastoma mouse models, providing a robust platform for preclinical evaluation of novel therapeutic combinations.
Allogeneic hematopoietic stem cell transplant (HSCT) is a proven curative therapy for children with high-risk myeloid malignancies. Disease relapse, transplant-related mortality and graft versus host disease (GvHD) are the main causes of treatment failure and death post-transplant. The optimum pretransplant conditioning regimen is yet to be defined. There is limited data regarding the use of busulfan, fludarabine and melphalan as a myeloablative conditioning regimen in children receiving HSCT for myeloid malignancies.
Rishi S. Kotecha MB ChB (Hons) MRCPCH FRACP PhD Co-Head, Leukaemia Translational Research rishi.kotecha@health.wa.gov.au Co-Head, Leukaemia
B-cell acute lymphoblastic leukemia (B-ALL) is the most common pediatric cancer. Current therapeutic regimens have improved 5-year event-free survival rates to 90%, however clinical outcomes for high-risk subgroups, such as BCR-ABL1+ B-ALL and relapsed ALL, remain poor. In addition, 16% of newly diagnosed children with ALL present with vertebral compression fractures. Moreover, 16% of children with ALL undergoing glucocorticoid therapy also experience a high incidence of vertebral fractures, indicating that bone health may be compromised by both leukemia progression and osteotoxicity of chemotherapy.
The RNA-binding protein IGF2BP3 is an oncofetal protein overexpressed in B-acute lymphoblastic leukemia and is critical for leukemogenesis in experimental models. With cancerspecific expression, functional dispensability for normal development, and an unleveraged prooncogenic function in mRNA homeostasis, IGF2BP3 represents an excellent target.
Britta Regli-von Ungern-Sternberg AM FAHMS MD, PhD, DEAA, FANZA Chair of Paediatric anaesthesia, University of Western Australia; Consultant
Glioblastoma, a lethal high-grade glioma, has not seen improvements in clinical outcomes in nearly 30 years. Ion channels are increasingly associated with tumorigenesis, and there are hundreds of brain-penetrant drugs that inhibit ion channels, representing an untapped therapeutic resource. The aim of this exploratory drug study was to screen an ion channel drug library against patient-derived glioblastoma cells to identify new treatments for brain cancer.
Invasive fungal disease (IFD) is a common and important complication in children with acute myeloid leukaemia (AML). We describe the epidemiology of IFD in a large multicentre cohort of children with AML.
Current immunization guidelines recommend one dose of influenza vaccine for children aged ≥9 years and two doses for younger or vaccine-naïve children. However, children receiving chemotherapy have an attenuated immune response. We performed a prospective open-label study in children undergoing treatment for cancer at Perth Children's Hospital, Western Australia, to examine the safety and efficacy of a boosted influenza schedule.
Acute lymphoblastic leukaemia (ALL) is the most common paediatric malignancy and remains one of the most common causes of cancer-related death in children and adolescents. Five-year overall survival rates now exceed 90% with current multidrug chemotherapeutic regimens.