Children's oncology Group's 2013 blueprint for research: Central nervous system tumors

Pediatric Blood and Cancer

Volumn 60, Issue 6, 2013, Pages 1022-1026

  Gajjar, Amar ^a   Packer, Roger J ^b   Foreman, N K ^c   Cohen, Kenneth ^d   Haas Kogan, Daphne ^e   Merchant, Thomas E ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^b CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^c UNIVERSITY OF COLORADO   (United States)

^d JOHNS HOPKINS UNIVERSITY   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Ependymoma; High grade glioma and diffuse intrinsic pontine glioma; Medulloblastoma

Indexed keywords

BEVACIZUMAB; CARBOPLATIN; CISPLATIN; CYCLOPHOSPHAMIDE; GADOLINIUM; LOMUSTINE; MOTEXAFIN; TEMOZOLOMIDE; VINCRISTINE; VISMODEGIB;

BRAIN TUMOR; CANCER ADJUVANT THERAPY; CANCER INCIDENCE; CANCER RADIOTHERAPY; CANCER RECURRENCE; CANCER RESEARCH; CANCER STAGING; CANCER SURGERY; CANCER SURVIVAL; CRANIOSPINAL IRRADIATION; EPENDYMOMA; GLIOMA; HUMAN; MEDULLOBLASTOMA; PONTINE GLIOMA; PRIORITY JOURNAL; REVIEW;

ANTINEOPLASTIC AGENTS; CENTRAL NERVOUS SYSTEM NEOPLASMS; CHEMORADIOTHERAPY; CHILD; CLINICAL TRIALS AS TOPIC; HUMANS; NEUROSURGICAL PROCEDURES; RESEARCH;

EID: 84876430481     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.24427     Document Type: Review

References (66)

1. Gurney JG, Smith MA, Bunnin GR. Chapter III: CNS and miscellaneous intracranial and intraspinal neoplasms. In: Ries LAG, Smith MA, Gurney JG, et al., editors. Cancer incidence and survival among children and adolescents: United States SEER Program 1975-1995. National Cancer Institute, SEER Program. Bethesda, MD: NIH Publication 99-4649; 1999.
2. Gottardo NG, Gajjar A. Current therapy for medulloblastoma. Curr Treat Options Neurol 2006; 8: 319-334.
3. Eberhart CG, Kepner JL, Goldthwaite PT, et al. Histopathologic grading of medulloblastomas: A Pediatric Oncology Group study. Cancer 2002; 94: 552-560.
4. Northcott PA, Korshunov A, Witt H, et al. Medulloblastoma comprises four distinct molecular variants. J Clin Oncol 2010; 29: 1408-1414.
5. Thompson MC, Fuller C, Hogg TL, et al. Genomics identifies medulloblastoma subgroups that are enriched for specific genetic alterations. J Clin Oncol 2006; 24: 1924-1931.
6. Kool M, Koster J, Bunt J, et al. Integrated genomics identifies five medulloblastoma subtypes with distinct genetic profiles, pathway signatures and clinicopathological features. PLoS ONE 2008; 3: e3088.
7. Northcott PA, Korshunov A, Pfister SM, et al. The clinical implications of medulloblastoma subgroups. Nat Rev Neurol 2012; 8: 340-351.
8. Kool M, Korshunov A, Remke M, et al. Molecular subgroups of medulloblastoma: An international meta-analysis of transcriptome, genetic aberrations, and clinical data of WNT, SHH, Group 3, and Group 4 medulloblastomas. Acta Neuropathol 2012; 123: 473-484.
9. Ris MD, Packer R, Goldwein J, et al. Intellectual outcome after reduced-dose radiation therapy plus adjuvant chemotherapy for medulloblastoma: A Children's Cancer Group study. J Clin Oncol 2001; 19: 3470-3476.
10. Packer RJ, Gajjar A, Vezina G, et al. Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma. J Clin Oncol 2006; 24: 4202-4208.
11. Jakacki RI, Burger PC, Zhou T, et al. Outcome of children with metastatic medulloblastoma treated with carboplatin during craniospinal radiotherapy: A Children's Oncology Group Phase I/II Study. J Clin Oncol 2012; 30: 2648-2653.
12. Donaldson SS, Laningham F, Fisher PG. Advances toward an understanding of brainstem gliomas. J Clin Oncol 2006; 24: 1266-1272.
13. Laigle-Donadey F, Doz F, Delattre JY. Brainstem gliomas in children and adults. Curr Opin Oncol 2008; 20: 662-667.
14. Hargrave D, Bartels U, Bouffet E. Diffuse brainstem glioma in children: Critical review of clinical trials. Lancet Oncol 2006; 7: 241-248.
15. Cohen KJ, Heideman RL, Zhou T, et al. Temozolomide in the treatment of children with newly diagnosed diffuse intrinsic pontine gliomas: A report from the Children's Oncology Group. Neuro Oncol 2011; 13: 410-416.
16. Cohen KJ, Pollack IF, Zhou T, et al. Temozolomide in the treatment of high-grade gliomas in children: A report from the Children's Oncology Group. Neuro Oncol 2011; 13: 317-323.
17. Gururangan S, Chi SN, Young Poussaint T, et al. Lack of efficacy of bevacizumab plus irinotecan in children with recurrent malignant glioma and diffuse brainstem glioma: A Pediatric Brain Tumor Consortium study. J Clin Oncol 2010; 28: 3069-3075.
18. McGuire CS, Sainani KL, Fisher PG. Incidence patterns for ependymoma: A surveillance, epidemiology, and end results study. J Neurosurg 2009; 110: 725-729.
19. Merchant TE, Li C, Xiong X, et al. Conformal radiotherapy after surgery for paediatric ependymoma: A prospective study. Lancet Oncol 2009; 10: 258-266.
20. Tihan T, Zhou T, Holmes E, et al. The prognostic value of histological grading of posterior fossa ependymomas in children: A Children's Oncology Group study and a review of prognostic factors. Mod Pathol 2008; 21: 165-177.
21. Korshunov A, Witt H, Hielscher T, et al. Molecular staging of intracranial ependymoma in children and adults. J Clin Oncol 2010; 28: 3182-3190.
22. Godfraind C, Kaczmarska JM, Kocak M, et al. Distinct disease-risk groups in pediatric supratentorial and posterior fossa ependymomas. Acta Neuropathol 2012; 124: 247-257.
23. Witt H, Mack SC, Ryzhova M, et al. Delineation of two clinically and molecularly distinct subgroups of posterior fossa ependymoma. Cancer Cell 2011; 20: 143-157.
24. Archer TC, Pomeroy SL. Posterior fossa ependymomas: A tale of two subtypes. Cancer Cell 2011; 20: 133-134.
25. Boop FA. Repeat surgery for residual ependymoma. J Neurosurg Pediatr 2011; 8: 244-245; discussion 245.
26. Massimino M, Solero CL, Garre ML, et al. Second-look surgery for ependymoma: The Italian experience. J Neurosurg Pediatr 2011; 8: 246-250.
27. Zacharoulis S, Ji L, Pollack IF, et al. Metastatic ependymoma: A multi-institutional retrospective analysis of prognostic factors. Pediatr Blood Cancer 2008; 50: 231-235.
28. Kilday JP, Mitra B, Domerg C, et al. Copy number gain of 1q25 predicts poor progression-free survival for pediatric intracranial ependymomas and enables patient risk stratification: A prospective European clinical trial cohort analysis on behalf of the Children's Cancer Leukaemia Group (CCLG), Societe Francaise d'Oncologie Pediatrique (SFOP), and International Society for Pediatric Oncology (SIOP). Clin Cancer Res 2012; 18: 2001-2011.
29. van Veelen-Vincent ML, Pierre-Kahn A, Kalifa C, et al. Ependymoma in childhood: Prognostic factors, extent of surgery, and adjuvant therapy. J Neurosurg 2002; 97: 827-835.
30. Grundy RG, Wilne SA, Weston CL, et al. Primary postoperative chemotherapy without radiotherapy for intracranial ependymoma in children: The UKCCSG/SIOP prospective study. Lancet Oncol 2007; 8: 696-705.
31. Grill J, Le Deley MC, Gambarelli D, et al. Postoperative chemotherapy without irradiation for ependymoma in children under 5 years of age: A multicenter trial of the French Society of Pediatric Oncology. J Clin Oncol 2001; 19: 1288-1296.
32. Zacharoulis S, Ashley S, Moreno L, et al. Treatment and outcome of children with relapsed ependymoma: A multi-institutional retrospective analysis. Childs Nerv Syst 2010; 26: 905-911.
33. Messahel B, Ashley S, Saran F, et al. Relapsed intracranial ependymoma in children in the UK: Patterns of relapse, survival and therapeutic outcome. Eur J Cancer 2009; 45: 1815-1823.
34. Merchant TE, Boop FA, Kun LE, et al. A retrospective study of surgery and reirradiation for recurrent ependymoma. Int J Radiat Oncol Biol Phys 2008; 71: 87-97.
35. Goodrich LV, Milenkovic L, Higgins KM, et al. Altered neural cell fates and medulloblastoma in mouse patched mutants. Science 1997; 277: 1109-1113.
36. Chen JK, Taipale J, Young KE, et al. Small molecule modulation of Smoothened activity. Proc Natl Acad Sci U S A 2002; 99: 14071-14076.
37. Lipinski RJ, Hutson PR, Hannam PW, et al. Dose- and route-dependent teratogenicity, toxicity, and pharmacokinetic profiles of the hedgehog signaling antagonist cyclopamine in the mouse. Toxicol Sci 2008; 104: 189-197.
38. Romer JT, Kimura H, Magdaleno S, et al. Suppression of the Shh pathway using a small molecule inhibitor eliminates medulloblastoma in Ptc1(+/-) p53(-/-) mice. Cancer Cell 2004; 6: 229-240.
39. Rubin LL, de Sauvage FJ. Targeting the Hedgehog pathway in cancer. Nat Rev Drug Discov 2006; 5: 1026-1033.
40. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. N Engl J Med 2009; 361: 1164-1172.
41. Rudin CM, Hann CL, Laterra J, et al. Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-044. N Engl J Med 2009; 361: 1173-1178.
42. Jones DT, Jager N, Kool M, et al. Dissecting the genomic complexity underlying medulloblastoma. Nature 2012; 488: 100-105.
43. Pugh TJ, Weeraratne SD, Archer TC, et al. Medulloblastoma exome sequencing uncovers subtype-specific somatic mutations. Nature 2012; 488: 106-110.
44. Robinson G, Parker M, Kranenburg TA, et al. Novel mutations target distinct subgroups of medulloblastoma. Nature 2012; 488: 43-48.
45. Northcott PA, Shih DJ, Peacock J, et al. Subgroup-specific structural variation across 1, 000 medulloblastoma genomes. Nature 2012; 488: 49-56.
46. Kawauchi D, Robinson G, Uziel T, et al. A mouse model of the most aggressive subgroup of human medulloblastoma. Cancer Cell 2012; 21: 168-180.
47. Pei Y, Moore CE, Wang J, et al. An animal model of MYC-driven medulloblastoma. Cancer Cell 2012; 21: 155-167.
48. Eberhart CG. Three down and one to go: Modeling medulloblastoma subgroups. Cancer Cell 2012; 21: 137-138.
49. Gibson P, Tong Y, Robinson G, et al. Subtypes of medulloblastoma have distinct developmental origins. Nature 2010; 468: 1095-1099.
50. Schwartzentruber J, Korshunov A, Liu XY, et al. Driver mutations in histone H3.3 and chromatin remodelling genes in paediatric glioblastoma. Nature 2012; 482: 226-231.
51. Wu G, Broniscer A, McEachron TA, et al. Somatic histone H3 alterations in pediatric diffuse intrinsic pontine gliomas and non-brainstem glioblastomas. Nat Genet 2012; 44: 251-253.
52. 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.
53. Hawkins CE, Bartels U, Bouffet E. Molecular genetic approaches and potential new therapeutic strategies for pediatric diffuse intrinsic pontine glioma. J Clin Oncol 2011; 29: 3956-3957.
54. Johnson RA, Wright KD, Poppleton H, et al. Cross-species genomics matches driver mutations and cell compartments to model ependymoma. Nature 2010; 466: 632-636.
55. Lannering B, Rutkowski S, Doz F, et al. Hyperfractionated versus conventional radiotherapy followed by chemotherapy in standard-risk medulloblastoma: Results from the randomized multicenter HIT-SIOP PNET 4 trial. J Clin Oncol 2012; 30: 3187-3193.
56. Gajjar A, Chintagumpala M, Ashley D, et al. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): Long-term results from a prospective, multicentre trial. Lancet Oncol 2006; 7: 813-820.
57. Gandola L, Massimino M, Cefalo G, et al. Hyperfractionated accelerated radiotherapy in the Milan strategy for metastatic medulloblastoma. J Clin Oncol 2009; 27: 566-571.
58. Dufour C, Grill J, Lellouch-Tubiana A, et al. High-grade glioma in children under 5 years of age: A chemotherapy only approach with the BBSFOP protocol. Eur J Cancer 2006; 42: 2939-2945.
59. Fangusaro J. Pediatric high grade glioma: A review and update on tumor clinical characteristics and biology. Front Oncol 2012; 2: 105.
60. Stupp R, Hegi ME, Gilbert MR, et al. Chemoradiotherapy in malignant glioma: Standard of care and future directions. J Clin Oncol 2007; 25: 4127-4136.
61. Bradley KA, Zhou T, McNall-Knapp RY, et al. Motexafin-gadolinium and involved field radiation therapy for intrinsic pontine glioma of childhood: A Children's Oncology Group Phase 2 Study. Int J Radiat Oncol Biol Phys 2012. DOI: http://dx.doi.org/10.1016/j.ijrobp.2012.09.004
62. Ellison DW, Onilude OE, Lindsey JC, et al. beta-Catenin status predicts a favorable outcome in childhood medulloblastoma: The United Kingdom Children's Cancer Study Group Brain Tumour Committee. J Clin Oncol 2005; 23: 7951-7957.
63. 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: 3061-3068.
64. Atkinson JM, Shelat AA, Carcaboso AM, et al. An integrated in vitro and in vivo high-throughput screen identifies treatment leads for ependymoma. Cancer Cell 2011; 20: 384-399.
65. Fouladi M, Stewart CF, Blaney SM, et al. Phase I trial of lapatinib in children with refractory CNS malignancies: A Pediatric Brain Tumor Consortium study. J Clin Oncol 28: 4221-4227.
66. Tabori U, Wong V, Ma J, et al. Telomere maintenance and dysfunction predict recurrence in paediatric ependymoma. Br J Cancer 2008; 99: 1129-1135.