Seminars in Oncology
Volume 36 , Pages S69-S76 , August 2009

Statistical and Trial Design Considerations in Central Nervous System Prophylaxis Studies

  • Jennifer A. Smith

      Affiliations

    • Geron Corp, Menlo Park, CA
    • Corresponding Author InformationAddress correspondence to Jennifer A. Smith, PhD, Geron Corp, 230 Constitution Dr, Menlo Park, CA 94025
  • ,
  • Michael Glantz

      Affiliations

    • Department of Neurosurgery, Penn State Hershey Medical Center, Hershey, PA

References 

  1. Boehme V, Zeynalova S, Kloess M, Loeffler M, Kaiser U, Pfeurndschuh M, et al. Incidence and risk factors of central nervous system recurrence in aggressive lymphoma—a survey of 1693 patients treated in protocols of the German High-Grade Non-Hodgkin's Lymphoma Study Group (DSHNHL). Ann Oncol. 2007;18:149–157
  2. Sørlie T, Perou CM, Tibshirani R, Aas T, Geisler S, Johnsen H, et al. Gene expression patterns of breast carcinomas distinguish tumor subclasses with clinical implications. Proc Natl Acad Sci U S A. 2001;98:10869–10874
  3. Haioun C, Besson C, Lepage E, Thieblemont C, Simon D, Rose C, et al. Incidence and risk factors of central nervous system relapse in histologically aggressive non-Hodgkin's lymphoma uniformly treated and receiving intrathecal central nervous system prophylaxis: a GELA study on 974 patients (Groupe d'Etudes des Lymphomes de l'Adulte). Ann Oncol. 2000;11:685–690
  4. Hollender A, Kvaloy S, Nome O, Skovlund E, Lote K, Holte H. Central nervous system involvement following diagnosis of non-Hodgkin's lymphoma: a risk model. Ann Oncol. 2002;13:1099–1107
  5. van Besien K, Ha CS, Murphy S, McLaughlin P, Rodriguez A, Amin K, et al. Risk factors, treatment, and outcome of central nervous system recurrence in adults with intermediate-grade and immunoblastic lymphoma. Blood. 1998;91:1178–1184
  6. Zinzani PL, Magagnoli M, Frezza G, Prologo G, Gherlinzoni F, Bendandi M, et al. Isolated central nervous system relapse in aggressive non-Hodgkin's lymphoma: the Bologna experience. Leuk Lymphoma. 1999;32:571–576
  7. Tomita N, Kodama F, Sakai R, Koharasawa H, Hattori M, Taguchi J, et al. Predictive factors for central nervous system involvement in non-Hodgkin's lymphoma: significance of very high serum LDH concentrations. Leuk Lymphoma. 2000;38:335–343
  8. Herman TS, Hammond N, Jones SE, Butler JJ, Byrne GE, McKelvey EM. Involvement of the central nervous system by non-Hodgkin's lymphoma (The Southwest Oncology Group experience). Cancer. 1979;43:390–397
  9. Liang R, Chiu E, Loke SL. Secondary central nervous system involvement by non-Hodgkin's lymphoma: the risk factors. Hematol Oncol. 1990;8:141–145
  10. Keldsen N, Michalski W, Bentzen SM, Hansen KB, Thorling K. Risk factors for central nervous system involvement in non-Hodgkin's lymphoma—a multivariate analysis. Acta Oncol. 1996;35:703–708
  11. Litam JP, Cabanillas F, Smith TL, Bodey GP, Freireich EJ. Central nervous system relapse in malignant lymphomas: risk factors and implications for prophylaxis. Blood. 1979;54:1249–1257
  12. Ersboll J, Schultz HB, Thomsen BL, Keiding N, Nissen NI. Meningeal involvement in non-Hodgkin's lymphoma: symptoms, incidence, risk factors and treatment. Scand J Haematol. 1985;35:487–496
  13. Kesari S, Batchelor TT. Leptomeningeal metastases. Neurol Clin. 2003;21:25–66
  14. Bromberg JE, Breems DA, Kraan J, Bikker G, van der Holt B, Smitt PS, et al. CSF flow cytometry greatly improves diagnostic accuracy in CNS hematologic malignancies. Neurology. 2007;68:1674–1679
  15. Benesch M, Urban C. Liposomal cytarabine for leukemic and lymphomatous meningitis: recent developments. Expert Opin Pharmacother. 2008;9:301–309
  16. Ceresoli GL, Reni M, Chiesa G, Carretta A, Schipani S, Passoni P, et al. Brain metastases in locally advanced nonsmall cell lung carcinoma after multimodality treatment: risk factors analysis. Cancer. 2002;95:605–612
  17. Evans AJ, James JJ, Cornford EJ, Chan SY, Burrell HC, Pinder SE, et al. Brain metastases from breast cancer: identification of a high-risk group. Clin Oncol (R Coll Radiol). 2004;16:345–349
  18. Melisko ME, Glantz M, Rugo HS. New challenges and opportunities in the management of brain metastases in patients with ErbB2-positive metastatic breast cancer. Nat Clin Pract Oncol. 2009;6:25–33
  19. Mazhar D, Stebbing J, Bower M. Non-Hodgkin's lymphoma and the CNS: prophylaxis and therapy in immunocompetent and HIV-positive individuals. Expert Rev Anticancer Ther. 2006;6:335–341
  20. Thall PF. A review of phase 2-3 clinical trial designs. Lifetime Data Anal. 2008;14:37–53
  21. Mehta MP, Shapiro WR, Glantz MJ, Patchell RA, Weitzner MA, Meyers CA, et al. Lead-in phase to randomized trial of motexafin gadolinium and whole-brain radiation for patients with brain metastases: centralized assessment of magnetic resonance imaging, neurocognitive, and neurologic end points. J Clin Oncol. 2002;20:3445–3453
  22. Meyers CA, Smith JA, Bezjak A, Mehta MP, Liebmann J, Illidge T, et al. Neurocognitive function and progression in patients with brain metastases treated with whole-brain radiation and motexafin gadolinium: results of a randomized phase III trial. J Clin Oncol. 2004;2:157–165
  23. Gelber RD, Cole BF, Gelber S, Goldhirsch A. Comparing treatments using quality-adjusted survival: the Q-TwiST method. Am Stat. 1995;49:161–169
  24. Recht L, Glantz M, Chamberlain M, Hseih CC. Qualitative measurement of quality outcome in malignant glioma patients using an independent living score (ILS). J Neurooncol. 2003;61:127–136
  25. Cole BF, Glantz MJ, Jaeckle K. 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
  26. Glantz M, Conlee J. High grade gliomas. In:  Meyers C,  Perry A editor. Cognition and cancer. Cambridge, UK: Cambridge University Press; 2008;p. 156–169
  27. Benedict RHB, Schretlen D, Groninger L, Brandt J. Hopkins Verbal Learning Test-Revised: normative data and analysis of inter-form and test-retest reliability. Clin Neuropsychol. 1998;12:43–55
  28. Lezak MD, Howleson DB, Loring DW. Neuropsychological assessment. New York: Oxford University Press; 1994;
  29. Lezak MD. Neuropsychological assessment (ed 3). New York: Academic Press; 1995;
  30. Benton AD. Differential behavioral effects in frontal lobe disease. Neuropsychologia. 1968;6:53–60
  31. Benton AL, Hamsher K de S. Multilingual Aphasia Examination. Iowa City, IA: AJA Associates; 1989;
  32. Wechsler D. Wechsler Adult Intelligence Scale–Revisited. San Antonio, TX: The Psychological Corp; 1981;
  33. Folstein MF, Folstein SE, McHugh PR. Mini-mental state”: a practical method for grading the cognitive state of patients for the clinician. J Psychiatr Res. 1975;12:189–198
  34. Smith J, Wefel J. Neurocognitive testing in clinical trials. In:  Meyers C,  Perry A editor. Cognition and cancer. Cambridge, UK: Cambridge University Press; 2008;p. 320–328
  35. Corn BW, Moughan J, Knisely JPS, Fox SW, Chakravarti A, Yung WK, et al. Prospective evaluation of quality of life and neurocognitive effects in patients with multiple brain metastases receiving whole-brain radiotherapy with or without thalidomide on Radiation Therapy Oncology Group (RTOG) trial 0118. Int J Radiat Oncol Biol Phys. 2008;71:71–78
  36. Mehta MP, Rodrigus P, Terhaard CHJ, Rao A, Suh J, Roa W, et al. Survival and neurologic outcomes in a randomized trial of motexafin gadolinium and whole-brain radiation therapy in brain metastases. J Clin Oncol. 2003;21:2529–2536
  37. Melisko ME, Kunwar S, Prados M, Berger MS, Park JW. Brain metastases of breast cancer. Expert Rev Anticancer Ther. 2005;5:253–268
  38. Shah BD, Kesari S, Xu R, Batchelor TT, O'Neill AM, Hochberg FH, et al. Comparison of linear and volumetric criteria in assessing tumor response in adult high-grade gliomas. Neurooncology. 2006;8:38–46
  39. Ananthnarayan S, Bahng J, Roring J, Nghiemphu P, Lai A, Cloughesy T, et al. Time course of imaging changes of GBM during extended bevacizumab treatment. J Neurooncology. 2008;88:339–347
  40. Norden AD, Young GS, Setayesh K, Muzikansky A, Klufas R, Ross GL, et al. Bevacizumab for recurrent malignant gliomas: efficacy, toxicity, and patterns of recurrence. Neurology. 2008;70:779–787
  41. de Groot JF, Yung WK. Bevacizumab and irinotecan in the treatment of recurrent malignant gliomas. Cancer J. 2008;14:279–285
  42. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989;10:1–10
  43. Stupp R, Mason WP, van den Bent MJ, Weller M, Fisher B, Taphoorn MJ, et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352:987–996
  44. Lee JJ, Feng L. Randomized phase II designs in cancer clinical trials: current status and future directions. J Clin Oncol. 2005;23:4450–4457
  45. Simon R, Wittes RE, Ellenberg SS. Randomized phase II clinical trials. Cancer Treat Rep. 1985;69:1375–1381
  46. Aupérin A, Arriagada R, Pignon JP, Le Péchoux C, Gregor A, Stephens RJ, et al. Prophylactic cranial irradiation for patients with small-cell lung cancer in complete remission (Prophylactic Cranial Irradiation Overview Collaborative Group). N Engl J Med. 1999;341:476–484
  47. Lee JJ, Bekele BN, Zhou X, Cantor SB, Komaki R, Lee JS. Decision analysis for prophylactic cranial irradiation for patients with small-cell lung cancer. J Clin Oncol. 2006;24:3597–3603

 STATEMENT OF CONFLICT OF INTEREST: M.G. has received honoraria and research support from Enzon Pharmaceuticals. J.A.S. has nothing to disclose.

PII: S0093-7754(09)00098-0

doi: 10.1053/j.seminoncol.2009.05.008

Seminars in Oncology
Volume 36 , Pages S69-S76 , August 2009