Hg-75clinical, Radiological, and Histo-Genetic Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma: A Collaborative Report From the International and Siop-E Dipg Registries

Hoffman, Lindsey M.; Zanten, Sophie E M Veldhuijzen van; Colditz, Niclas; Baugh, Joshua; Chaney, Brooklyn; Lane, Adam; Fuller, Christine; Miles, Lili; Hawkins, Cynthia; Bartels, Ute; Bouffet, Éric; Goldman, Stewart; Leary, Sarah; Foreman, Nicholas; Packer, Roger J.; Warren, Katherine E.; Broniscer, Alberto; Kieran, Mark W.; Minturn, Jane E.; Comito, Melanie; Broxon, Emmett; Shih, Chie‐Schin; Khatua, Soumen; Chintagumpala, Murali; Carret, Anne‐Sophie; Hassall, Tim; Ziegler, David S.; Gottardo, Nicholas G.; Dholaria, Hetal; Lerme, Brianna; Kirkendall, Jenavieve; Doughman, Renee; Hoffmann, Marion; Wollman, Matthew; O'Keefe, Rachel; Benesch, Martin; Gerber, Nicolas U.; Bailey, Simon; Solanki, Guirish A.; Massimino, Maura; Biassoni, Veronica; Cvrlje, Filip Jadrijević; Hulleman, Esther; Drissi, Rachid; Nazarian, Javad; Jabado, Nada; Bueren, André O. von; Pietsch, Torsten; Gielen, Gerrit H.; Sturm, Dominik; Jones, David T.; Pfister, Stefan M.; Jones, Chris; Hargrave, Darren; Bison, Brigitte; Warmuth‐Metz, Monika; Leach, James; Jones, Blaise V.; Vuurden, Dannis G. van; Kramm, Christof M.; Fouladi, Maryam

doi:10.1093/neuonc/now073.71

Neuro Oncol

2016

DOI: 10.1093/neuonc/now073.71

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Hg-75clinical, Radiological, and Histo-Genetic Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma: A Collaborative Report From the International and Siop-E Dipg Registries

Lindsey M. Hoffman

Sophie E M Veldhuijzen van Zanten

Niclas Colditz

et al.

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“…While 5-year survival rates for paediatric LGG are as high as 95%, HGG prognosis remains poor with reports indicating only 15-35% 5-year survival rates and median overall survival (OS) of 10-18 months (8), despite continuous advances in surgical and adjuvant chemoradiation therapies (10,11). There is still no effective treatment for DIPG which carries only 10% 2-year survival, decreasing to 2% at 5 years (12)(13)(14). Hence, there is an evident need to develop more effective treatments than the current standard of aggressive surgery with chemoradiation, particularly owing to the considerable toxicities and subsequent longterm sequelae in developing children and adolescents.…”

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confidence: 99%

Molecular Genetics and Targeted Therapies for Paediatric High-grade Glioma

Rallis

George

Woźniak

et al. 2022

Cancer Genomics Proteomics

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Brain tumours are the leading cause of paediatric cancer-associated death worldwide. High-grade glioma (HGG) represents a main cause of paediatric brain tumours and is associated with poor prognosis despite surgical and chemoradiotherapeutic advances. The molecular genetics of paediatric HGG (pHGG) are distinct from those in adults, and therefore, adult clinical trial data cannot be extrapolated to children. Compared to adult HGG, pHGG is characterised by more frequent mutations in PDGFRA, TP53 and recurrent K27M and G34R/V mutations on histone H3. Ongoing trials are investigating novel targeted therapies in pHGG. Promising results have been achieved with BRAF/MEK and PI3K/mTOR inhibitors. Combination of PI3K/mTOR, EGFR, CDK4/6, and HDAC inhibitors are potentially viable options. Inhibitors targeting the UPS proteosome, ADAM10/17, IDO, and XPO1 are more novel and are being investigated in early-phase trials. Despite preclinical and clinical trials holding promise for the discovery of effective pHGG treatments, several issues persist. Inadequate blood-brain barrier penetration, unfavourable pharmacokinetics, dose-limiting toxicities, long-term adverse effects in the developing child, and short-lived duration of response due to relapse and resistance highlight the need for further improvement. Future pHGG management will largely depend on selecting combination therapies which work synergistically based on a sound knowledge of the underlying molecular target pathways. A systematic investigation of multimodal therapy with chemoradiotherapy, surgery, target agents and immunotherapy is paramount. This review provides a comprehensive overview of pHGG focusing on molecular genetics and novel targeted therapies. The diagnostics, genetic discrepancies with adults and their clinical implications, as well as conventional treatment approaches are discussed.Intracranial and intraspinal tumours are the most common solid tumours in paediatrics (1). They are the second leading cause of cancer-associated death in children and adolescents under the age of 19 in the USA and Canada (1), with an average annual age-adjusted incidence of 6.06 per 100,000 in the USA between 2012 and 2016 (2). Brain tumours are now the leading cause of paediatric-cancer-associated death worldwide, surpassing deaths from childhood leukaemia (3). Gliomas represent the highest proportion of childhood brain tumours (4, 5) accounting for 60% of paediatric brain tumour cases (5, 6), of which approximately half are classified as high grade (6, 7).

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mentioning

confidence: 99%

Molecular Genetics and Targeted Therapies for Paediatric High-grade Glioma

Rallis

George

Woźniak

et al. 2022

Cancer Genomics Proteomics

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H3 K27M-altered glioma and diffuse intrinsic pontine glioma: Semi-systematic review of treatment landscape and future directions

van den Bent,

Saratsis,

Geurts

et al. 2023

Neuro-Oncology

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H3 K27M-mutant diffuse glioma is a recently identified brain tumor associated with poor prognosis. As of 2016, it is classified by the World Health Organization as a distinct form of grade IV glioma. Despite recognition as an important prognostic and diagnostic feature in diffuse glioma, radiation remains the sole standard of care and no effective systemic therapies are available for H3K27M mutant tumors. This review will detail treatment interventions applied to diffuse midline glioma (DMG) and diffuse intrinsic pontine glioma (DIPG) prior to the identification of the H3 K27M mutation, the current standard-of-care for H3 K27M-mutant diffuse glioma treatment, and ongoing clinical trials listed on w w w.clinicaltrials.gov evaluating novel therapeutics in this population. Current clinical trials were identified using clinicaltrials.gov, and studies qualifying for this analysis were active or ongoing interventional trials that evaluated a therapy in at least one treatment arm or cohort comprised exclusively of patients with DIPG and H3 K27M-mutant glioma. Forty-one studies met these criteria, including trials evaluating H3 K27M vaccination, chimeric antigen-receptor T cell therapy, and small molecule inhibitors. Ongoing evaluation of novel therapeutics is necessary to identify safe and effective interventions in this underserved patient population.

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Hg-75clinical, Radiological, and Histo-Genetic Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma: A Collaborative Report From the International and Siop-E Dipg Registries

Cited by 2 publications

References 0 publications

Molecular Genetics and Targeted Therapies for Paediatric High-grade Glioma

Molecular Genetics and Targeted Therapies for Paediatric High-grade Glioma

H3 K27M-altered glioma and diffuse intrinsic pontine glioma: Semi-systematic review of treatment landscape and future directions

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