Children are not just little adults: Recent advances in understanding of diffuse intrinsic pontine glioma biology

Pediatric Research

Volumn 75, Issue 1-2, 2014, Pages 205-209

  Schroeder, Kristin M ^a   Hoeman, Christine M ^a   Becher, Oren J ^a  

^a DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; CYCLIN D; CYCLIN DEPENDENT KINASE 4; CYCLIN DEPENDENT KINASE 6; CYCLIN DEPENDENT KINASE INHIBITOR 2A; CYCLIN DEPENDENT KINASE INHIBITOR 2B; CYTOTOXIC AGENT; EPIDERMAL GROWTH FACTOR RECEPTOR; EPIDERMAL GROWTH FACTOR RECEPTOR 4; GADOLINIUM; HISTONE H3; K RAS PROTEIN; PHOSPHATIDYLINOSITOL 3 KINASE; PROTEIN KINASE B; RAS PROTEIN; SCATTER FACTOR; SCATTER FACTOR RECEPTOR; SOMATOMEDIN B; SOMATOMEDIN RECEPTOR; TOPOTECAN; ISOCITRATE DEHYDROGENASE 1; ISOCITRATE DEHYDROGENASE 2; PHOSPHATIDYLINOSITOL 4,5 BISPHOSPHATE 3 KINASE; PLATELET DERIVED GROWTH FACTOR ALPHA RECEPTOR; PLATELET DERIVED GROWTH FACTOR B; POLYCOMB REPRESSIVE COMPLEX 2; PROTEIN P53; PROTEIN TYROSINE KINASE;

ABDUCENS NERVE PARALYSIS; AMINO ACID SUBSTITUTION; AMINO TERMINAL SEQUENCE; ATAXIA; BABINSKI REFLEX; BRAIN BIOPSY; CANCER COMBINATION CHEMOTHERAPY; CANCER DIAGNOSIS; CANCER RADIOTHERAPY; CANCER SURVIVAL; CHILDHOOD CANCER; CLINICAL FEATURE; CONTRAST ENHANCEMENT; CONVECTION ENHANCED DELIVERY; DIPLOPIA; DRUG DELIVERY SYSTEM; DYSARTHRIA; DYSMETRIA; EVENT FREE SURVIVAL; FACE ASYMMETRY; GENE AMPLIFICATION; GENE DELETION; GENE EXPRESSION PROFILING; HISTONE ACETYLATION; HISTONE METHYLATION; HUMAN; HYPERREFLEXIA; INTRACRANIAL HYPERTENSION; NEUROIMAGING; NEUROLOGIC DISEASE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OBSTRUCTIVE HYDROCEPHALUS; OVERALL SURVIVAL; PHASE 1 CLINICAL TRIAL (TOPIC); PHYSICAL EXAMINATION; PONTINE GLIOMA; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; PROTEIN EXPRESSION; QUALITY OF LIFE; REVIEW; AKT3 GENE; BLOOD BRAIN BARRIER; BRAIN FOURTH VENTRICLE; BRAIN STEM; CANCER CHEMOTHERAPY; CHILD; CHROMOSOME 16Q; CHROMOSOME 17P; CHROMOSOME 20P; CHROMOSOME 2Q; CHROMOSOME 8Q; CHROMOSOME 9Q; CHROMOSOME LOSS; CRANIAL NEUROPATHY; GAIN OF FUNCTION MUTATION; GENE MUTATION; GENE OVEREXPRESSION; GENETICALLY ENGINEERED MOUSE STRAIN; GLIOMA CELL LINE; H3F3A GENE; HIST1H3B GENE; K27M GENE; LONG TERMINAL REPEAT; MOLECULARLY TARGETED THERAPY; ONCOGENE; ONCOGENE K RAS; P53 GENE; PATHOGENESIS; PHOSPHATIDYLINOSITOL 4,5 BISPHOSPHATE 3 KINASE GENE; PIKC2G GENE; PLATELET DERIVED GROWTH FACTOR ALPHA RECEPTOR GENE; SIGNAL TRANSDUCTION;

ADULT; AGE FACTORS; ANIMALS; BRAIN STEM NEOPLASMS; CHILD; CLINICAL TRIALS AS TOPIC; DISEASE MODELS, ANIMAL; GENETIC PREDISPOSITION TO DISEASE; GLIOMA; HUMANS; MUTATION; PHENOTYPE; SIGNAL TRANSDUCTION; TRANSLATIONAL MEDICAL RESEARCH; TREATMENT OUTCOME;

EID: 84900384372     PISSN: 00313998     EISSN: 15300447     Source Type: Journal    
DOI: 10.1038/pr.2013.194     Document Type: Review

References (50)

1. Kohler BA, Ward E, McCarthy BJ, et al. Annual report to the nation on the status of cancer, 1975-2007, featuring tumors of the brain and other nervous system. J Natl Cancer Inst 2011;103:714-36.
2. National Cancer Institute. Cancer Incidence and Survival among Children and Adolescents: United States SEER Program 1975-1995, 1999. (http://seer.cancer.gov/publications/childhood/cns.pdf.).
3. Minturn JE, Fisher MJ. Gliomas in children. Curr Treat Options Neurol 2013;15:316-27.
4. Freeman CR, Farmer JP. Pediatric brain stem gliomas: a review. Int J Radiat Oncol Biol Phys 1998;40:265-71.
5. Berger MS, Edwards MS, LaMasters D, Davis RL, Wilson CB. Pediatric brain stem tumors: radiographic, pathological, and clinical correlations. Neurosurgery 1983;12:298-302.
6. Littman P, Jarrett P, Bilaniuk LT, et al. Pediatric brain stem gliomas. Cancer 1980;45:2787-92.
7. Hargrave D, Bartels U, Bouffet E. Diffuse brainstem glioma in children: critical review of clinical trials. Lancet Oncol 2006;7:241-8.
8. Albright AL, Guthkelch AN, Packer RJ, Price RA, Rourke LB. Prognostic factors in pediatric brain-stem gliomas. J Neurosurg 1986;65:751-5.
9. Fisher PG, Breiter SN, Carson BS, et al. A clinicopathologic reappraisal of brain stem tumor classification. Identification of pilocystic astrocytoma and fibrillary astrocytoma as distinct entities. Cancer 2000;89:1569-76.
10. Gururangan S, McLaughlin CA, Brashears J, et al. Incidence and patterns of neuraxis metastases in children with diffuse pontine glioma. J Neurooncol 2006;77:207-12.
11. Fischbein NJ, Prados MD, Wara W, Russo C, Edwards MS, Barkovich AJ. Radiologic classification of brain stem tumors: correlation of magnetic resonance imaging appearance with clinical outcome. Pediatr Neurosurg 1996;24:9-23.
12. Albright AL, Packer RJ, Zimmerman R, Rorke LB, Boyett J, Hammond GD. Magnetic resonance scans should replace biopsies for the diagnosis of diffuse brain stem gliomas: a report from the Children's Cancer Group. Neurosurgery 1993;33:1026-9; discussion 1029-30.
13. Roujeau T, Machado G, Garnett MR, et al. Stereotactic biopsy of diffuse pontine lesions in children. J Neurosurg 2007;107(Suppl 1):1-4.
14. Geoerger B, Hargrave D, Thomas F, et al.; ITCC (Innovative Therapies for Children with Cancer) European Consortium. Innovative Therapies for Children with Cancer pediatric phase I study of erlotinib in brainstem glioma and relapsing/refractory brain tumors. Neuro Oncol 2011;13:109-18.
15. MacDonald TJ. Diffuse intrinsic pontine glioma (DIPG): time to biopsy again? Pediatr Blood Cancer 2012;58:487-8.
16. Janssens GO, Jansen MH, Lauwers SJ, et al. Hypofractionation vs conventional radiation therapy for newly diagnosed diffuse intrinsic pontine glioma: a matched-cohort analysis. Int J Radiat Oncol Biol Phys 2013;85:315-20.
17. Jansen MH, van Vuurden DG, Vandertop WP, Kaspers GJ. Diffuse intrinsic pontine gliomas: a systematic update on clinical trials and biology. Cancer Treat Rev 2012;38:27-35.
18. Hall WA, Doolittle ND, Daman M, et al. Osmotic blood-brain barrier disruption chemotherapy for diffuse pontine gliomas. J Neurooncol 2006;77:279-84.
19. Warren K, Jakacki R, Widemann B, et al. Phase II trial of intravenous lobradimil and carboplatin in childhood brain tumors: a report from the Children's Oncology Group. Cancer Chemother Pharmacol 2006;58:343-7.
20. Greenberg ML, Fisher PG, Freeman C, et al. Etoposide, vincristine, and cyclosporin A with standard-dose radiation therapy in newly diagnosed diffuse intrinsic brainstem gliomas: a pediatric oncology group phase I study. Pediatr Blood Cancer 2005;45:644-8.
21. Bobo RH, Laske DW, Akbasak A, Morrison PF, Dedrick RL, Oldfield EH. Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci USA 1994;91:2076-80.
22. Anderson RC, Kennedy B, Yanes CL, et al. Convection-enhanced delivery of topotecan into diffuse intrinsic brainstem tumors in children. J Neurosurg Pediatr 2013;11:289-95.
23. Paugh BS, Broniscer A, Qu C, et al. Genome-wide analyses identify recurrent amplifications of receptor tyrosine kinases and cell-cycle regulatory genes in diffuse intrinsic pontine glioma. J Clin Oncol 2011;29:3999-4006.
24. Zarghooni M, Bartels U, Lee E, et al. Whole-genome profiling of pediatric diffuse intrinsic pontine gliomas highlights platelet-derived growth factor receptor alpha and poly (ADP-ribose) polymerase as potential therapeutic targets. J Clin Oncol 2010;28:1337-44.
25. Puget S, Philippe C, Bax DA, et al. Mesenchymal transition and PDGFRA amplification/mutation are key distinct oncogenic events in pediatric diffuse intrinsic pontine gliomas. PLoS ONE 2012;7:e30313.
26. Verhaak RG, Hoadley KA, Purdom E, et al.; Cancer Genome Atlas Research Network. Integrated genomic analysis identifies clinically relevant subtypes of glioblastoma characterized by abnormalities in PDGFRA, IDH1, EGFR, and NF1. Cancer Cell 2010;17:98-110.
27. Grill J, Puget S, Andreiuolo F, Philippe C, MacConaill L, Kieran MW. Critical oncogenic mutations in newly diagnosed pediatric diffuse intrinsic pontine glioma. Pediatr Blood Cancer 2012;58:489-91.
28. Khuong-Quang DA, Buczkowicz P, Rakopoulos P, et al. K27M mutation in histone H3.3 defines clinically and biologically distinct subgroups of pediatric diffuse intrinsic pontine gliomas. Acta Neuropathol 2012;124:439-47.
29. Warren KE, Killian K, Suuriniemi M, Wang Y, Quezado M, Meltzer PS. Genomic aberrations in pediatric diffuse intrinsic pontine gliomas. Neuro Oncol 2012;14:326-32.
30. Wu G, Broniscer A, McEachron TA, et al.; St. Jude Children's Research Hospital-Washington University Pediatric Cancer Genome Project. Somatic histone H3 alterations in pediatric diffuse intrinsic pontine gliomas and non-brainstem glioblastomas. Nat Genet 2012;44:251-3.
31. Barski A, Cuddapah S, Cui K, et al. High-resolution profiling of histone methylations in the human genome. Cell 2007;129:823-37.
32. Reynolds N, Salmon-Divon M, Dvinge H, et al. NuRD-mediated deacetylation of H3K27 facilitates recruitment of Polycomb Repressive Complex 2 to direct gene repression. EMBO J 2012;31:593-605.
33. Lewis PW, Müller MM, Koletsky MS, et al. Inhibition of PRC2 activity by a gain-of-function H3 mutation found in pediatric glioblastoma. Science 2013;340:857-61.
34. Monje M, Mitra SS, Freret ME, et al. Hedgehog-responsive candidate cell of origin for diffuse intrinsic pontine glioma. Proc Natl Acad Sci USA 2011;108:4453-8.
35. Becher OJ, Hambardzumyan D, Walker TR, et al. Preclinical evaluation of radiation and perifosine in a genetically and histologically accurate model of brainstem glioma. Cancer Res 2010;70:2548-57.
36. Schwartzentruber J, Korshunov A, Liu XY, et al. Driver mutations in histone H3.3 and chromatin remodelling genes in paediatric glioblastoma. Nature 2012;482:226-31.
37. Paugh BS, Qu C, Jones C, et al. Integrated molecular genetic profiling of pediatric high-grade gliomas reveals key differences with the adult disease. J Clin Oncol 2010;28:3061-8.
38. Raffel C, Frederick L, O'Fallon JR, et al. Analysis of oncogene and tumor suppressor gene alterations in pediatric malignant astrocytomas reveals reduced survival for patients with PTEN mutations. Clin Cancer Res 1999;5:4085-90.
39. Barrow J, Adamowicz-Brice M, Cartmill M, et al. Homozygous loss of ADAM3A revealed by genome-wide analysis of pediatric high-grade glioma and diffuse intrinsic pontine gliomas. Neuro Oncol 2011;13:212-22.
40. Arjona D, Bello MJ, Alonso ME, et al. Molecular analysis of the EGFR gene in astrocytic gliomas: mRNA expression, quantitative-PCR analysis of non-homogeneous gene amplification and DNA sequence alterations. Neuropathol Appl Neurobiol 2005;31:384-94.
41. Pollack IF, Hamilton RL, James CD, et al.; Children's Oncology Group. Rarity of PTEN deletions and EGFR amplification in malignant gliomas of childhood: results from the Children's Cancer Group 945 cohort. J Neurosurg 2006;105(Suppl 5):418-24.
42. Louis DN, Rubio MP, Correa KM, Gusella JF, von Deimling A. Molecular genetics of pediatric brain stem gliomas. Application of PCR techniques to small and archival brain tumor specimens. J Neuropathol Exp Neurol 1993;52:507-15.
43. Wong KK, Tsang YT, Chang YM, et al. Genome-wide allelic imbalance analysis of pediatric gliomas by single nucleotide polymorphic allele array. Cancer Res 2006;66:11172-8.
44. Gilbertson RJ, Hill DA, Hernan R, et al. ERBB1 is amplified and overexpressed in high-grade diffusely infiltrative pediatric brain stem glioma. Clin Cancer Res 2003;9(10 Pt 1):3620-4.
45. Pollack IF, Hamilton RL, Sobol RW, et al.; Children's Oncology Group. IDH1 mutations are common in malignant gliomas arising in adolescents: a report from the Children's Oncology Group. Childs Nerv Syst 2011;27:87-94.
46. Yan H, Parsons DW, Jin G, et al. IDH1 and IDH2 mutations in gliomas. N Engl J Med 2009;360:765-73.
47. Pollack IF, Finkelstein SD, Burnham J, et al.; Children's Cancer Group. Age and TP53 mutation frequency in childhood malignant gliomas: results in a multi-institutional cohort. Cancer Res 2001;61:7404-7.
48. Milinkovic V, Bankovic J, Rakic M, et al. Genomic instability and p53 alterations in patients with malignant glioma. Exp Mol Pathol 2012;93:200-6.
49. Fontebasso AM, Schwartzentruber J, Khuong-Quang DA, et al. Mutations in SETD2 and genes affecting histone H3K36 methylation target hemispheric high-grade gliomas. Acta Neuropathol 2013;125:659-69.
50. Rickert CH, Sträter R, Kaatsch P, et al. Pediatric high-grade astrocytomas show chromosomal imbalances distinct from adult cases. Am J Pathol 2001;158:1525-32.