Design and endpoints of clinical trials in hepatocellular carcinoma

Journal of the National Cancer Institute

Volumn 100, Issue 10, 2008, Pages 698-711

  Llovet, Josep M ^a,b   Di Bisceglie, Adrian M ^c   Bruix, Jordi ^b   Kramer, Barnett S ^d   Lencioni, Riccardo ^e   Zhu, Andrew X ^f   Sherman, Morris ^g   Schwartz, Myron ^a   Lotze, Michael ^h   Talwalkar, Jayant ⁱ   Gores, Gregory J ⁱ  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b HOSPITAL CLÍNIC   (Spain)

^c SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d NATIONAL INSTITUTES OF HEALTH   (United States)

^e UNIVERSITY OF PISA   (Italy)

^f HARVARD MEDICAL SCHOOL   (United States)

^g UNIVERSITY OF TORONTO   (Canada)

^h UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

ⁱ MAYO CLINIC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALCOHOL; BEVACIZUMAB; ERLOTINIB; INTERFERON; SORAFENIB; TAMOXIFEN; TEMSIROLIMUS; ANTINEOPLASTIC AGENT; TUMOR MARKER;

ARTIFICIAL EMBOLISM; CANCER GROWTH; CANCER RECURRENCE; CANCER STAGING; CANCER SURVIVAL; CLINICAL RESEARCH; COMORBIDITY; COST BENEFIT ANALYSIS; DISEASE COURSE; DISEASE FREE SURVIVAL; EVIDENCE BASED MEDICINE; EXPERIMENTAL DESIGN; HUMAN; LIVER CELL CARCINOMA; LIVER DISEASE; LIVER FUNCTION; LIVER RESECTION; LIVER TRANSPLANTATION; MOLECULAR IMAGING; NOTE; PATIENT SELECTION; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROGNOSIS; RADIOFREQUENCY ABLATION; BIOASSAY; BLOOD; CHEMOEMBOLIZATION; CLINICAL TRIAL; LIVER FUNCTION TEST; LIVER TUMOR; METHODOLOGY; PATHOLOGY; PATHOPHYSIOLOGY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; RANDOMIZED CONTROLLED TRIAL; REVIEW; SURVIVAL; TREATMENT OUTCOME; TUMOR RECURRENCE;

ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CARCINOMA, HEPATOCELLULAR; CHEMOEMBOLIZATION, THERAPEUTIC; CLINICAL TRIALS AS TOPIC; CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; DISEASE PROGRESSION; DISEASE-FREE SURVIVAL; ENDPOINT DETERMINATION; EVIDENCE-BASED MEDICINE; HUMANS; LIVER FUNCTION TESTS; LIVER NEOPLASMS; NEOPLASM RECURRENCE, LOCAL; NEOPLASM STAGING; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SURVIVAL ANALYSIS; TREATMENT OUTCOME; TUMOR MARKERS, BIOLOGICAL;

EID: 44449085884     PISSN: 00278874     EISSN: 14602105     Source Type: Journal    
DOI: 10.1093/jnci/djn134     Document Type: Note

References (61)

1. Parkin DM, Bray F, Ferlay J, Pisani P. Global cancer statistics 2002. CA Cancer J Clin. 2005;55(2):74-108.
2. Sangiovanni A, Del Ninno E, Fasani P, et al. Increased survival of cirrhotic patients with a hepatocellular carcinoma detected during surveillance. Gastroenterology. 2004;126(4):1005-1014.
3. El Serag HB, Mason AC. Rising incidence of hepatocellular carcinoma in the United States. N Engl J Med. 1999;340(10):745-750.
4. Llovet JM, Burroughs A, Bruix J. Hepatocellular carcinoma. Lancet. 2003;362(9399):1907-1917.
5. Llovet JM, Bruix J. Systematic review of randomized trials for unresectable hepatocellular carcinoma: chemoembolization improves survival. Hepatology. 2003;37(2):429-442.
6. López PM, Villanueva A, Llovet JM. Systematic review: evidence-based management of hepatocellular carcinoma - an updated analysis of randomized controlled trials. Aliment Pharmacol Ther. 2006;23(11):1535-1547.
7. Bruix J, Sherman M. Management of hepatocellular carcinoma. Hepatology. 2005;42(5):1208-1236.
8. Bruix J, Sherman M, Llovet JM, et al. Clinical management of hepatocellular carcinoma. Conclusions of the Barcelona-2000 EASL Conference. J Hepatol. 2001;35(3):421-430.
9. Llovet JM, Bru C, Bruix J. Prognosis of hepatocellular carcinoma: the BCLC staging classification. Semin Liver Dis. 1999;19(3):329-338.
10. Marrero JA, Fontana RJ, Barrat A, et al. Prognosis of hepatocellular carcinoma: comparison of 7 staging systems in an American cohort. Hepatology. 2005;41(4):707-716.
11. Cillo U, Vitale A, Grigoletto F, et al. Prospective validation of the Barcelona Clinic Liver Cancer staging system. J Hepatol. 2006;44(4 ):723-731.
12. Vauthey J, Lauwers G, Esnaola N, et al. Simplified staging for hepatocellular carcinoma. J Clin Oncol. 2002;20(6):1527-1536.
13. Llovet JM, Schwartz M, Mazzaferro V. Resection and liver transplantation for hepatocellular carcinoma. Semin Liver Dis. 2005;25(2):181-200.
14. Villa E, Moles A, Ferretti I, et al. Natural history of inoperable hepatocellular carcinoma: estrogen receptors' status in the tumor is the strongest prognostic factor for survival. Hepatology. 2000;32(2):233-238.
15. Mazzaferro V, Regalia E, Doci R, et al. Liver transplantation for the treatment of small hepatocellular carcinomas in patients with cirrhosis. N Engl J Med. 1996;334(11):693-699.
16. Lo CM, Ngan H, Tso WK, et al. Randomized controlled trial of transarterial lipiodol chemoembolization for unresectable hepatocellular carcinoma . Hepatology. 2002;35(5):1164-1171.
17. Llovet JM, Real MI, Montana X, et al. Arterial embolisation or chemoembolisation versus symptomatic treatment in patients with unresectable hepatocellular carcinoma: a randomised controlled trial. Lancet. 2002;359(9319):1734-1739.
18. Llovet J, Ricci S, Mazzaferro V, et al.; SHARP Investigators. Sorafenib improves survival in advanced Hepatocellular Carcinoma (HCC): results of a phase III randomized placebo-controlled trial. J Clin Oncol. 2007;LBA1.
19. Chien JY, Friedrich S, Heathman MA, de Alwis DP, Sinha V. Pharmacokinetics, pharmacodynamics, and the stages of drug development: role of modeling and simulation. AAPS J. 2005;7(3):E544-E559.
20. National Cancer Institute Web site. US National Institute of Health. http://www.cancer.gov. [Last accessed: April 17, 2008.]
21. Whitehead J. Stopping clinical trials by design. Nat Rev Drug Discov. 2004;3(11):973-977.
22. Pocock SJ. Clinical Trials: A Practical Approach. Chichester, UK: Wiley; 1983.
23. Jadad A, Moore A, Carroll D, et al. Assessing the quality of reports of randomized clinical trials: is blinding necessary?. Control Clin Trials. 1996;17(1):1-12.
24. Moher D, Schulz KF, Altman DG: CONSORT Group. The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomised trials. Lancet. 2001;357(9263):1191-1194.
25. Wright JC, Weinstein MC. Gains in life expectancy from medical interventions-standardizing data on outcomes. N Engl J Med. 1998;339(6):380-386.
26. Laupacis A, Feeny D, Detsky AS, et al. How attractive does a new technology have to be to warrant adoption and utilization? Tentative guidelines for using clinical and economic evaluations. CMAJ. 1992;146(4):473-481.
27. Spruance SL, Reid JE, Grace M, Samore M. Hazard ratio in clinical trials. Antimicrob Agents Chemother. 2004;48(8):2787-2792.
28. Rothenberg ML, Carbone DP, Johnson DH. Improving the evaluation of new cancer treatments: challenges and opportunities. Nat Rev Cancer. 2003;3(4):303-309.
29. Lee JJ, Feng L. Randomized phase II designs in cancer clinical trials: current status and future directions. J Clin Oncol. 2005;23(19):4450- 4457.
30. Johnson JR, Williams G, Pazdur R. End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol. 2003;21(7):1404-1411.
31. Putter H, Ficco M, Geskus RB. Tutorial in biostatistics: competing risks and multi-state models. Stat Med. 2007;26(11):2389-2430.
32. Kim WR, Therneau TM, Benson JT, et al. Deaths on the liver transplant waiting list: an analysis of competing risks. Hepatology. 2006;43(2):345-351.
33. Imamura H, Matsuyama Y, Tanaka E, et al. Risk factors contributing to early and late phase intrahepatic recurrence of hepatocellular carcinoma after hepatectomy. J Hepatol. 2003;38(2):200-207.
34. Cheung ST, Chen X, Guan XY, et al. Identify metastasis-associated genes in hepatocellular carcinoma through clonality delineation for multinodular tumor. Cancer Res. 2002;62(16):4711-4721.
35. Chen Y, Yeh SH, Chen JT, et al. Chromosomal changes and clonality relationship between primary and recurrent hepatocellular carcinoma. Gastroenterology. 2000;119(2):431-440.
36. Heffernan N, Cella D, Webster K, et al. Measuring health-related quality of life in patients with hepatobiliary cancers: the functional assessment of cancer therapy-hepatobiliary questionnaire. J Clin Oncol. 2002;20(9):2229-2239.
37. Blazeby JM, Currie E, Zee BC, Chie WC, Poon RT, Garden OJ EORTC Quality of Life Group. Development of a questionnaire module to supplement the EORTC QLQ-C30 to assess quality of life in patients with hepatocellular carcinoma, the EORTC QLQ-HCC18. Eur J Cancer. 2004;40(16):2439-2444.
38. Gennari A, Amadori D, De Lena M, et al. Lack of benefit of maintenance paclitaxel in first-line chemotherapy in metastatic breast cancer. J Clin Oncol. 2006;24(24):3912-3918.
39. Miller AB, Hoogstraten B, Staquet M, Winkler A. Reporting results of cancer treatment. Cancer. 1981;47(1):207-214.
40. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. J Natl Cancer Inst. 2000;92(3):205-216.
41. Shepherd FA, Rodrigues Pereira J, et al.; National Cancer Institute of Canada Clinical Trials Group. Erlotinib in previously treated non-small-cell lung cancer. N Engl J Med. 2005;353(2):123-132.
42. Hudes G, Carducci M, Tomczak P, et al.;Global ARCC Trial. Temsirolimus, interferon alfa, or both for advanced renal-cell carcinoma. N Engl J Med. 2007;356(22):2271-2281.
43. Hurwitz H, Fehrenbacher L, Novotny W, et al. Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer. N Engl J Med. 2004;350(23):2335-2342.
44. Millar AW, Lynch KP. Rethinking clinical trials for cytostatic drugs. Nat Rev Cancer. 2003;3(7):540-545.
45. Kelloff GJ, Krohn KA, Larson SM, et al. The progress and promise of molecular imaging probes in oncologic drug development. Clin Cancer Res. 2005;11(22):7967-7985.
46. Hylton N. Dynamic contrast-enhanced magnetic resonance imaging as an imaging biomarker. J Clin Oncol. 2006;24(20):3293-3298.
47. Gharib AM, Thomasson D, Li KC. Molecular imaging of hepatocellular carcinoma. Gastroenterology. 2004;127(5 suppl 1):S153-S158.
48. Rebmann V, Schütt P, Brandhorst D, et al. Soluble MICA as an independent prognostic factor for the overall survival and progression-free survival of multiple myeloma patients. Clin Immunol. 2007;123(1):114-120.
49. Colli A, Fraquelli M, Casazza G, et al. Accuracy of ultrasonography, spiral CT, magnetic resonance, and alpha-fetoprotein in diagnosing hepatocellular carcinoma: a systematic review. Am J Gastroenterol. 2006;101(3):513-523.
50. Forner A, Vilana R, Ayuso C, et al. Diagnosis of hepatic nodules < 20 mm in cirrhosis. Prospective validation of the AASLD guidelines for HCC. Hepatology. 2008;47(1):97-104.
51. Llovet JM, Chen Y, Wurmbach W, et al. A molecular signature to discriminate dysplastic nodules and early hepatocellular carcinoma in HCV-cirrhosis. Gastroenterology. 2006;131(6):1758-1767.
52. D'Amico G, Garcia-Tsao G, Pagliaro L. Natural history and prognostic indicators of survival in cirrhosis: a systematic review of 118 studies. J Hepatol. 2006;44(1):217-231.
53. Prospective validation of the CLIP score: a new prognostic system for patients with cirrhosis and hepatocellular carcinoma. The Cancer of the Liver Italian Program (CLIP) Investigators. Hepatology. 2000;31(4):840-845.
54. Leung TW, Tang AM, Zee B, et al. Construction of the Chinese University Prognostic Index for hepatocellular carcinoma and comparison with the TNM staging system, the Okuda staging system, and the Cancer of the Liver Italian Program staging system: a study based on 926 patients. Cancer. 2002;94(6):1760-1769.
55. Sala M, Llovet JM, Vilana R, et al. Initial response to percutaneous ablation predicts survival in patients with hepatocellular carcinoma. Hepatology. 2004;40(6):1352-1360.
56. Livraghi T, Giorgio A, Marin G, et al. Hepatocellular carcinoma and cirrhosis in 746 patients: long-term results of percutaneous ethanol injection. Radiology. 1995;197(1):101-108.
57. Kamath PS, Wiesner RH, Malinchoc M, et al. A model to predict survival in patients with end-stage liver disease. Hepatology. 2001;33(2):464-470.
58. Simon R, Thall PF, Ellenberg SS. New designs for the selection of treatments to be tested in randomized clinical trials. Stat Med. 1994;13(5-7):417-429.
59. Slamon DJ, Leyland-Jones B, Shak S, et al. Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med. 2001;344(11):783-792.
60. Kobayashi S, Boggon TJ, Dayaram T, et al. EGFR mutation and resistance of non-small-cell lung cancer to gefitinib. N Engl J Med. 2005;352(8):786-792.
61. Lee JS, Heo J, Libbrecht L, et al. A novel prognostic subtype of human hepatocellular carcinoma derived from hepatic progenitor cells. Nat Med. 2006;12(4):410-416.