Clinical trials in neuro-oncology

Handbook of Clinical Neurology

Volumn 104, Issue , 2012, Pages 417-434

  Junck, Larry ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84855541112     PISSN: 00729752     EISSN: None     Source Type: Book Series    
DOI: 10.1016/B978-0-444-52138-5.00028-1     Document Type: Chapter

References (75)

1. Aaronson N.K., Ahmedzai S., Bergman B., et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst 1993, 85:365-376.
2. Abrantes-Pais F.de N., Friedman J.K., Lovallo W.R., et al. Psychological or physiological: Why are tetraplegic patients content?. Neurology 2007, 69:261-267.
3. Altman D.G., Schulz K.F., Moher D., et al. The revised CONSORT statement for reporting randomized trials: explanation and elaboration. Ann Intern Med 2001, 134:663-694.
4. Andrews D.W., Scott C.B., Sperduto P.W., et al. Whole brain radiation therapy with or without stereotactic radiosurgery boost for patients with one to three brain metastases: phase III results of the RTOG 9508 randomised trial. Lancet 2004, 363:1665-1672.
5. Aoyama H., Shirato H., Tago M., et al. Stereotactic radiosurgery plus whole-brain radiation therapy vs stereotactic radiosurgery alone for treatment of brain metastases. A randomized controlled trial. JAMA 2006, 295:2483-2491.
6. Ballman K.V., Buckner J.C., Brown P.D., et al. The relationship between six-month progression-free survival and 12-month overall survival end points for phase II trials in patients with glioblastoma multiforme. Neuro-oncol 2007, 9:29-38.
7. Cairncross J.G., Ueki K., Zlatescu M.C., et al. Specific genetic predictors of chemotherapeutic response and survival in patients with anaplastic oligodendrogliomas. J Natl Cancer Inst 1998, 90:1473-1479.
8. Cairncross G., Swinnen L., Bayer R., et al. Myeloablative chemotherapy for recurrent aggressive oligodendroglioma. Neuro-oncol 2000, 2:114-119.
9. Cairncross G., Berkey B., Shaw E., et al. Phase III trial of chemotherapy plus radiotherapy compared with radiotherapy alone for pure and mixed anaplastic oligodendroglioma: Intergroup Radiation Therapy Oncology Group Trial 9402. J Clin Oncol 2006, 24:2707-2714.
10. Cato A., Sutton L., Cato A. Clinical Drug Trials and Tribulations 2002, Marcel Dekker, New York. (2nd edn).
11. Chamberlain M.C., Glantz M.J., Chalmers L., et al. Early necrosis following concurrent Temodar and radiotherapy in patients with glioblastoma. J Neurooncol 2007, 82:81-83.
12. Chang S.M., Reynolds S.L., Butowski N., et al. GNOSIS: guidelines for neuro-oncology: standards for investigational studies - reporting of phase 1 and phase 2 clinical trials. Neuro-oncol 2005, 7:425-434.
13. Cheng L., Bao S., Rich J.N. Potential therapeutic implications of cancer stem cells in glioblastoma. Biochem Pharmacol 2010, 80:654-665.
14. Cleophas T.J., Zwinderman A.H., Cleophas T.F. Statistics Applied to Clinical Trials 2006, Springer, Dordrecht. (3rd edn).
15. Cole B.F., Glantz M.J., Jaeckle K.A., et al. Quality-of-life-adjusted survival comparison of sustained-release cytosine arabinoside versus intrathecal methotrexate for treatment of solid tumor neoplastic meningitis. Cancer 2003, 97:3053-3060.
16. Curran W.J., Scott C.B., Horton J., et al. Recursive partitioning analysis of prognostic factors in three Radiation Therapy Oncology Group malignant glioma trials. J Natl Cancer Inst 1993, 85:704-710.
17. Denysenko T., Gennero L., Roos M.A., et al. Glioblastoma cancer stem cells: heterogeneity, microenvironment and related therapeutic strategies. Cell Biochem Funct 2010, 28:343-351.
18. de Wit M.C.Y., de Bruin H.G., Eijkenboom W., et al. Immediate post-radiotherapy changes in malignant glioma can mimic tumor progression. Neurology 2004, 63:535-537.
19. Friedman H.S., Petros W.P., Friedman A.H., et al. Irinotecan therapy in adults with recurrent or progressive malignant glioma. J Clin Oncol 1999, 17:1516-1525.
20. Friedman H.S., Prados M.D., Wen P.Y., et al. Bevacizumab alone and in combination with irinotecan in recurrent glioblastoma. J Clin Oncol 2009, 27:4733-4740.
21. Galanis E., Buckner J.C., Maurer M.J., et al. Validation of neuroradiologic response assessment in gliomas: measurement by RECIST, two-dimensional, computer-assisted tumor area, and computer-assisted tumor volume methods. Neuro-oncol 2006, 8:156-165.
22. Galicich J.H., French L.A., Melby J.C. Use of dexamethasone in treatment of cerebral edema associated with brain tumors. J Lancet 1961, 81:46-53.
23. Gaspar L., Scott C., Rotman M., et al. Recursive partitioning analysis (RPA) of prognostic factors in three radiation therapy oncology group (RTOG) brain metastases trials. Int J Radiat Oncol Biol Phys 1997, 37:745-751.
24. Geller N.L. Advances in Clinical Trial Biostatistics 2004, Marcel Dekker, New York.
25. Gilbert M.R., Gonzalez J., Hunter K., et al. A phase I study for a factorial design of dose-dense temozolomide (TMZ) alone and in combination with permutations of thalidomide (THAL), isotretinoin (CRA) and/or celecoxib (CEL) as post-chemoradiation adjuvant therapy for newly diagnosed glioblastoma (GBM). Neuro-oncol 2006, 8:442-443.
26. Glantz M.J., Jaeckle K.A., Chamberlain M.C., et al. A randomized controlled trial comparing intrathecal sustained-release cytarabine (DepoCyt) to intrathecal methotrexate in patients with neoplastic meningitis from solid tumors. Clin Cancer Res 1999, 5:3394-3402.
27. Glantz M.J., LaFollette S., Jaeckle K.A., et al. Randomized trial of a slow-release versus a standard formulation of cytarabine for the intrathecal treatment of lymphomatous meningitis. J Clin Oncol 1999, 17:3110-3116.
28. Goodman S., Zahurak M., Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Stat Med 1995, 14:1149-1161.
29. Good P.I. A Manager's Guide to the Design and Conduct of Clinical Trials 2006, John Wiley & Sons, Hoboken. (2nd edn).
30. Grant R., Liang B.C., Slattery J., et al. Chemotherapy response criteria in malignant glioma. Neurology 1997, 48:1336-1340.
31. Green S., Benedetti J., Crowley J. Clinical Trials in Oncology: Interdisciplinary Statistics 1997, Chapman & Hall, London.
32. Hegi M.E., Diserens A.-C., Gorlia T., et al. MGMT gene silencing and benefit from temozolomide in glioblastoma. N Engl J Med 2005, 352:997-1003.
33. Hess K.R., Wong E.T., Jaeckle K.A., et al. Response and progression in recurrent malignant glioma. Neuro-oncol 1999, 1:282-288.
34. Jaeckle K.A., Batchelor T., O'Day S.J., et al. An open label trial of sustained-release cytarabine (DepoCyt) for the intrathecal treatment of solid tumor neoplastic meningitis. J Neurooncol 2002, 5:231-239.
35. Kaplan E.L., Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc 1958, 53:457-481.
36. Karim A.B.M.F., Maat B., Hatlevoll R., et al. A randomized trial on dose-response in radiation therapy of low-grade cerebral glioma: European Organization for Research and Treatment of Cancer (EORTC) Study 22844. Int J Radiat Oncol Biol Phys 1996, 36:549-556.
37. Karnofsky D.A., Burchenal J.H. The clinical evaluation of chemotherapeutic agents in cancer. evaluation of chemotherapeutic agents 1949, 191-205. Columbia University Press, New York. C.M. MacLeod (Ed.).
38. Keime-Guibert F., Chinot O., Taillandier L., et al. Radiotherapy for glioblastoma in the elderly. N Engl J Med 2007, 356:1527-1535.
39. Kocher M., Soffietti R., Abacioglu U., et al. Adjuvant whole-brain radiotherapy versus observation after radiosurgery or surgical resection of one to three cerebral metastases: results of the EORTC 22952-26001 study. J Clin Oncol 2011, 29:134-141.
40. Lamborn K.R., Yung W.K.A., Chang S.M., et al. Progression-free survival: an important endpoint in evaluating therapy for recurrent high-grade gliomas. Neuro-oncol 2008, 10:162-170.
41. Levin V.A., Wara W.M., Davis R.L., et al. Phase III comparison of BCNU and the combination of procarbazine, CCNU, and vincristine administered after radiotherapy with hydroxyurea for malignant gliomas. J Neurosurg 1985, 63:218-223.
42. Levin V.A., Ictech S., Hess K.R. Impact of phase II trials with progression-free survival as end-points on survival-based phase III studies in patients with anaplastic gliomas. BMC Cancer 2007, 7:106.
43. Macdonald D.R., Cascino T.L., Schold S.C., et al. Response criteria for phase II studies of supratentorial malignant glioma. J Clin Oncol 1990, 8:1277-1280.
44. Macdonald D.R., Kiebert G., Prados M., et al. Benefit of temozolomide compared to procarbazine in treatment of glioblastoma multiforme at first relapse: effect on neurological functioning, performance status, and health related quality of life. Cancer Invest 2005, 23:138-144.
45. Mellinghoff I.K., Wang M.Y., Vivanco I., et al. Molecular determinants of the response of glioblastomas to EGFR kinase inhibitors. N Engl J Med 2005, 353:2012-2024.
46. Newlands E.S., Blackledge G.R.P., Slack J.A., et al. Phase I trial of temozolomide (CCRG 81045: M&B 39831: NSC362856). Br J Cancer 1992, 65:287-291.
47. O'Quigley J., Pepe M., Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990, 46:33-48.
48. Osoba D., Aaronson N.K., Muller M., et al. The development and psychometric validation of a brain cancer quality-of-life questionnaire for use in combination with general cancer-specific questionnaires. Qual Life Res 1996, 5:139-150.
49. Patchell R.A., Tibbs P.A., Walsh J.W., et al. A randomized trial of surgery in the treatment of single metastases to the brain. N Engl J Med 1990, 322:494-500.
50. Patchell R.A., Tibbs P.A., Regine W.F., et al. Postoperative radiotherapy in the treatment of single metastases to the brain: a randomized trial. JAMA 1998, 280:1485-1489.
51. Patchell R.A., Tibbs P.A., Regine W.F., et al. Direct decompressive surgical resection in the treatment of spinal cord compression caused by metastatic cancer: a randomised trial. Lancet 2005, 366:643-648.
52. Piantadosi S., Fisher J.D., Grossman S. Practical implementation of a modified continual reassessment method for dose-finding trials. Cancer Chemother Pharmacol 1998, 41:429-436.
53. Prados M.D., Yung W.K., Jaeckle K.A., et al. Phase-1 trial of irinotecan (CPT-11) in patients with recurrent malignant glioma: a North American Brain Tumor Consortium study. Neuro-oncol 2004, 6:44-54.
54. Sanai N., Alvarez-Buylla A., Berger M.S. Neural stem cells and the origin of gliomas. N Engl J Med 2005, 353:811-822.
55. Shah G.D., Kesari S., Xu R., et al. Comparison of linear and volumetric criteria in assessing tumor response in adult high-grade gliomas. Neuro-oncol 2006, 8:38-46.
56. Shapiro W.R., Mechtler L., Cher H., et al. A randomized, double-blind study comparing corticorelin acetate with dexamethasone in patients with primary malignant glioma who require increased dexamethasone doses to control symptoms of peritumoral brain edema. J Clin Oncol 2009, 27:15s. (abstract).
57. Shaw E., Arusell R., Scheithauer B., et al. Prospective randomized trial of low- versus high-dose radiation therapy in adults with supratentorial low-grade glioma: initial report of a North Central Cancer Treatment Group/Radiation Therapy Oncology Group/Eastern Cooperative Oncology Group study. J Clin Oncol 2002, 20:2267-2276.
58. Slotman B., Faivre-Finn C., Kramer G., et al. EORTC Radiation Oncology Group and Lung Cancer Group. Prophylactic cranial irradiation in extensive small-cell lung cancer. N Engl J Med 2007, 357:664-672.
59. Stupp R., Dietrich P.-Y., Ostermann Kraljevic S., et al. Promising survival for patients with newly diagnosed glioblastoma multiforme treated with concomitant radiation plus temozolomide followed by adjuvant temozolomide. J Clin Oncol 2002, 20:1375-1382.
60. Stupp R., Mason W.P., van den Bent M.J., et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med 2005, 352:987-996.
61. Taphoorn M.J., Stupp R., Coens C., et al. Health-related quality of life in patients with glioblastoma: a randomised controlled trial. Lancet Oncol 2005, 6:937-944.
62. US Food and Drug Administration Center for Drug Evaluation and Research Common Toxicity Criteria for Adverse Events (CTCAE) v 3.0. Online Available:, (accessed 10 July 2008). http://www.fda.gov/cder/cancer/toxicityframe.htm.
63. van den Bent M.J., Afra D., de Witte O., et al. Long-term efficacy of early versus delayed radiotherapy for low-grade astrocytoma and oligodendroglioma in adults: the EORTC 22845 randomised trial. Lancet 2005, 366:985-990.
64. van den Bent M.J., Carpentier A.F., Brandes A.A., et al. Adjuvant procarbazine, lomustine, and vincristine improves progression-free survival but not overall survival in newly diagnosed anaplastic oligodendrogliomas and oligoastrocytomas: a randomized European Organisation for Research and Treatment of Cancer phase III trial. J Clin Oncol 2006, 24:2715-2722.
65. van den Bent M.J., Taphoorn M.J.B., Wefel J.S., et al. Response evaluation in neuro-oncology: assessment of outcome in trials on diffuse low grade gliomas. A report of the RANO group. Lancet Oncol 2011, (in press).
66. Vecht C.J., Wagner G.L., Wilms E.B. Treating seizures in patients with brain tumors: drug interactions between antiepileptic and chemotherapeutic agents. Semin Oncol 2003, 30:49-52.
67. Vredenburgh J.J., Desjardins A., Herndon J.E., et al. Bevacizumab plus irinotecan in recurrent glioblastoma multiforme. J Clin Oncol 2007, 25:4722-4729.
68. Walker M.D., Alexander E., Hunt W.E., et al. Evaluation of BCNU and/or radiotherapy in the treatment of anaplastic gliomas. J Neurosurg 1978, 49:333-343.
69. Weitzner M.A., Meyers C.A., Gelke C.K., et al. The Functional Assessment of Cancer Therapy (FACT) scale. Development of a brain subscale and revalidation of the general version (FACT-G) in patients with primary brain tumors. Cancer 1995, 75:1151-1161.
70. Wen P.Y., Macdonald D.R., Reardon D.A., et al. Updated response assessment criteria for high-grade gliomas: Response Assessment in Neuro-oncology Working Group. J Clin Oncol 2010, 28:1963-1972.
71. Westphal M., Hilt D.C., Bortey E., et al. A phase 3 trial of local chemotherapy with biodegradable carmustine (BCNU) wafers (Gliadel wafers) in patients with primary malignant glioma. Neuro-oncol 2003, 5:79-88.
72. Wong E.T., Hess K.R., Gleason M.J., et al. Outcomes and prognostic factors in recurrent glioma patients enrolled onto phase II clinical trials. J Clin Oncol 1999, 17:2572-2578.
73. World Medical Organization Declaration of Helsinki (1964). Br Med J 1996, 313:1448-1449.
74. Yung W.K.A., Prados M.D., Yaya-Tur R., et al. Multicenter phase II trial of temozolomide in patients with anaplastic astrocytoma or anaplastic oligoastrocytoma at first relapse. J Clin Oncol 1999, 17:2762-2771.
75. Yung W.K.A., Albright R.E., Olson J., et al. A phase II study of temozolomide vs. procarbazine in patients with glioblastoma multiforme at first relapse. Br J Cancer 2000, 83:588-593.