Evolution in the Management of Childhood Low-Grade Gliomas and Neuronal/Mixed Neuronal-Glial Tumors

1. Packer R.J., Pfister S., Bouffet E., Avery R., Bandopadhayay P., Bornhorst M., et al. Pediatric low-grade gliomas: implications of the biologic era. Neuro Oncol 2017; 19 (6): 750–61. PMID: 27683733

2. Jones D.T.W., Kieran M.W., Bouffet E., Alexandrescu S., Bandopadhayay P., Bornhorst M., et al. Pediatric Low-Grade Gliomas: Next Biologically Driven Steps. Neuro Oncol 2018; 20 (2): 160–73. PMID: 29016845

3. Fisher M.J., Loguidice M., Gutmann D.H., Listernick R., Ferner R.E., Ullrich N.J., et al. Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysis. Neuro Oncol 2012; 14: 790–7. PMID: 22474213

4. Franz D.N., Belousova E., Sparagana S.,Bebin E.M., Frost M., Kuperman R., et al. Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet 2013 Jan 12; 381 (9861): 125–32. PMID: 23158522

5. Krueger D.A., Care M.M., Holland K., Agricola K., Tudor C., Mangeshkar P., et al. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med 2010 Nov 4; 363 (19): 1801–11. PMID: 21047224

6. Chung T.K., Lynch E.R., Fiser C.J., Nelson D.A., Agricola K., Tudor C., et al. Psychiatric comorbidity and treatment response in patients with tuberous sclerosis complex. Ann Clin Psychiatry 2011 Nov; 23 (4): 263–9. PMID: 22073383

7. Krueger D.A., Franz D.N. Targeting mTOR complex 1 to treat neurological and psychiatric manifestations of tuberous sclerosis complex. Future Neurology 2011;6: 261–71.

8. Kieran M.W., Yao X., Macy M., Leary S., Cohen K., MacDonald T., et al. A prospective multi-institutional Phase II study of everolimus (RAD001), an mTOR inhibitor, in pediatric patients with recurrent or progressive low-grade glioma. A POETIC Consortium trial. Pediatr Blood Cancer 2013; 60 (Suppl 3): 19–20.

9. Packer R.J., Jakacki R., Horn M., Rood B., Vezina G., MacDonald T., et al. Objective response of multiply recurrent low-grade gliomas to bevacizumab and irinotecan. Ped Blood and Cancer 2009; 52: 791–5. PMID: 19165892

10. Gururangan S., Fangusaro J., Poussaint T.Y., McLendon R.E., Onar-Thomas A., Wu S., et al. Efficacy of bevacizumab plus irinotecan in children with recurrent low-grade gliomas – a Pediatric Brain Tumor Consortium study. Neuro Oncol 2014 Jan; 16 (2): 310–7. Epub 2013 Dec 4. PMID: 24311632

11. Avery R.A., Hwang E.I., Jakacki R.I., Packer R.J. Marked Recovery of Vision in Children with Optic Pathway Gliomas Treated with Bevacizumab. Archives Ophthalmology. JAMA Ophthalmo 2014 Jan 1; 132 (1): 111–4. PMID: 24232489

12. Pfister S., Janzarik W.G., Remke M., Ernst A., Werft W., Becker N., et al. BRAF gene duplication constitutes a mechanism of MAPK pathway activation in low-grade astrocytomas. J Clin Invest 2008; 118 (5): 1739–49 PMID: 18398503

13. Jones D.T., Kocialkowski S., Liu L., Pearson D.M., Bäcklund L.M., Ichimura K., et al. Tandem duplication producing a novel oncogenic BRAF fusion gene defines the majority of pilocytic astrocytomas. Cancer Res 2008; 68 (21): 8673–7.PMID: 18974108

14. Collins V.P., Jones D.T., Giannini C. Pilocytic astrocytoma: pathology, molecular mechanisms and markers. Acta Neuropathol 2015; 129 (6): 775–88. PMID: 25792358

15. D’Angelo F., Ceccarelli M., Tala, Garofano L., Zhang J., Frattini V., et al. The molecular landscape of glioma in patients with Neurofibromatosis 1. Nat Med 2019; 25 (1): 176–87. PMID: 30531922

16. Banerjee A., Jakacki R.I., OnarThomas A., Wu S., Nicolaides T., Young Poussaint T., et al. A phase I trial of the MEK inhibitor selumetinib (AZD6244) in pediatric patients with recurrent or refractory low-grade glioma: a Pediatric Brain Tumor Consortium (PBTC) study. Neuro Oncol 2017; 19 (8): 1135–44. PMID: 28339824

17. Fangusaro J., Onar-Thomas A., Young Poussaint T., Wu S., Ligon A.H., Lindeman N., et al. Selumetinib in paediatric patients with BRAF-aberrant or neurofibromatosis type 1-associated recurrent, refractory, or progressive low-grade glioma: a multicentre, phase 2 trial. Lancet Oncol 2019; 20 (7): 1011–22. PMID: 31151904

18. Bouffet E., Kieran M., Hargrave D., Roberts S., Aerts I., Broniscer A., et al. Trametinib therapy in pediatric patients with low-grade gliomas (LGG) with BRAF gene fusion; a disease-specific cohort in the first pediatric testing of trametinib. Neuro Oncol 2018; 20: i114.

19. Robison N., Pauly J., Malvar J., Gruber-Filbin M., de Mola R.L., Dorris K., et al. A phase I dose escalation trial of the MEK1/2 inhibitor MEK162 (binimetinib) in children with low-grade gliomas and other RAS/RAF pathways-activated tumors. Neuro Oncol 2018; 20: i114.

20. Kieran M., et al. in ESMO LBA19_PR. Copenhagen, Denmark, 2016.

21. Lassaletta A., Guerreiro Stucklin A., Ramaswamy V., Zapotocky M., McKeown T., et al. Profound clinical and radiological response to BRAF inhibition in a 2-month-old diencephalic child with hypothalamic/chiasmatic glioma. Pediatr Blood Cancer 2016; 63 (11): 2038–41. PMID: 27398937

22. Karajannis M.A., Legault G., Fisher M.J., Milla S.S., Cohen K.J., Wisoff J.H., et al. Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas. Neuro Oncol 2014; 16 (10): 1408–16. PMID: 24803676

23. Reinhardt A., Stichel D., Schrimpf D., Sahm F., Korshunov A., Reuss D.E., et al. Anaplastic astrocytoma with piloid features, a novel molecular class of IDH wildtype glioma with recurrent MAPK pathway, CDKN2A/B and ATRX alterations. Acta Neuropathol 2018; 136 (2): 273–91. PMID 29564591