Pediatric Hematology/Oncology and Immunopathology

Вопросы гематологии/онкологии и иммунопатологии в педиатрии

1726-17082414-9314

Fund Doctors, Innovations, Science for Children

292

10.24287/1726-1708-2019-18-4-105-108

LITERATURE REVIEW

ОБЗОР ЛИТЕРАТУРЫ

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

https://orcid.org/0000-0001-9413-7531

Packer

R. J.

United States

Correspondence: Roger J. Packer, MD, Center for Neuroscience and Behavioral Medicine, Gilbert Family Neurofibromatosis Institute, Brain Tumor Institute, Children’s National Hospital, 111 Michigan Ave, NW, Washington, DC, 20010, Phone: 202-476-5973, Fax: 202-476-2864

rpacker@childrensnational.org

Center for Neuroscience and Behavioral Medicine, Gilbert Family Neurofibromatosis Institute, Brain Tumor Institute, Children's National Hospital

31122019

184

10510831122019

2019

«D. Rogachev NMRCPHOI»

ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России

https://creativecommons.org/licenses/by/4.0

https://hemoncim.com/jour/article/view/292

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

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

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

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

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

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.

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.

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

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.

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

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.

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

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.

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

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.

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

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.

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

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.

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

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.

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

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.

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

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.

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.

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.

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.

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.

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

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

21.

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

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.

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

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.

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

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