Regorafenib: An evidence-based review of its potential in patients with advanced liver cancer

Core Evidence

Volumn 9, Issue , 2014, Pages 81-87

  Ravi, Sujan ^a   Singal, Ashwani K ^b  

^a University of Alabama at Birmingham   (United States)

^b University of Alabama at Birmingham   (United States)

Author keywords

Advanced HCC; HCC; Hepatocellular carcinoma; Multikinase inhibitors; Regorafenib

Indexed keywords

BILIRUBIN; BRIVANIB; CREATININE; ERLOTINIB; LINIFANIB; REGORAFENIB; SORAFENIB; SUNITINIB;

ACIDOSIS; ADVANCED CANCER; BILIRUBIN BLOOD LEVEL; CANCER PATIENT; CREATININE BLOOD LEVEL; DIARRHEA; DRUG BIOAVAILABILITY; DRUG EFFICACY; DRUG HALF LIFE; DRUG SAFETY; DRUG STRUCTURE; EASTERN COOPERATIVE ONCOLOGY GROUP; EVIDENCE BASED PRACTICE; FATIGUE; FUNCTIONAL STATUS; HUMAN; HYPERTENSION; HYPOALBUMINEMIA; HYPOPHOSPHATEMIA; INTRACELLULAR SIGNALING; LIVER CANCER; LIVER CARCINOGENESIS; LIVER CELL CARCINOMA; MODEL FOR END STAGE LIVER DISEASE SCORE; NAMED INVENTORIES, QUESTIONNAIRES AND RATING SCALES; OVERALL SURVIVAL; PHARMACODYNAMICS; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROTEINURIA; REVIEW; SKIN MANIFESTATION;

EID: 84904582152     PISSN: 15551741     EISSN: 1555175X     Source Type: Journal    
DOI: 10.2147/CE.S48626     Document Type: Review

References (44)

1. Ferlay J, Soerjomataram I, Ervik M, et al. Globocan 2012: Estimated Cancer Incidence and Mortality Worldwide. Geneva: WHO; 2012.
2. European Association for the Study of the Liver, European Organisation for Research and Treatment of Cancer. EASL-EORTC clinical practice guidelines: management of hepatocellular carcinoma. J Hepatol. 2012;56(4):908-943.
3. Llovet JM, Bruix J. Novel advancements in the management of hepatocellular carcinoma in 2008. J Hepatol. 2008;48 Suppl 1:S20-S37.
4. Eastern Cooperative Oncology Group. ECOG performance status. 2006. Available from: http://ecog.dfci.harvard.edu/general/perf-stat.html. Accessed April 8, 2014.
5. Bruix J, Sherman M. Management of hepatocellular carcinoma: an update. Hepatology. 2011;53(3):1020-1022.
6. Llovet JM, Di Bisceglie AM, Bruix J, et al. Design and endpoints of clinical trials in hepatocellular carcinoma. J Natl Cancer Inst. 2008;100(10):698-711.
7. Huo TI, Lin HC, Hsia CY, et al. The model for end-stage liver disease based cancer staging systems are better prognostic models for hepatocellular carcinoma: a prospective sequential survey. Am J Gastroenterol. 2007;102(9):1920-1930.
8. Vauthey JN, Lauwers GY, Esnaola NF, et al. Simplified staging for hepatocellular carcinoma. J Clin Oncol. 2002;20(6):1527-1536. (Pubitemid 34260532)
9. Okuda K, Ohtsuki T, Obata H, et al. Natural history of hepatocellular carcinoma and prognosis in relation to treatment. Study of 850 patients. Cancer. 1985;56(4):918-928. (Pubitemid 15228292)
10. Cabibbo G, Enea M, Attanasio M, Bruix J, Craxi A, Camma C. A meta-analysis of survival rates of untreated patients in randomized clinical trials of hepatocellular carcinoma. Hepatology. 2010;51(4):1274-1283.
11. Semela D, Dufour JF. Angiogenesis and hepatocellular carcinoma. J Hepatol. 2004;41(5):864-880. (Pubitemid 39434320)
12. Chiang DY, Villanueva A, Hoshida Y, et al. Focal gains of VEGFA and molecular classification of hepatocellular carcinoma. Cancer Res. 2008;68(16):6779-6788.
13. Ito Y, Takeda T, Sakon M, et al. Expression and clinical significance of erb-B receptor family in hepatocellular carcinoma. Br J Cancer. 2001;84(10):1377-1383. (Pubitemid 32522107)
14. Breuhahn K, Longerich T, Schirmacher P. Dysregulation of growth factor signaling in human hepatocellular carcinoma. Oncogene. 2006;25(27):3787-3800. (Pubitemid 43980474)
15. Tovar V, Alsinet C, Villanueva A, et al. IGF activation in a molecular subclass of hepatocellular carcinoma and pre-clinical efficacy of IGF-1R blockage. J Hepatol. 2010;52(4):550-559.
16. Villanueva A, Newell P, Chiang DY, Friedman SL, Llovet JM. Genomics and signaling pathways in hepatocellular carcinoma. Semin Liver Dis. 2007;27(1):55-76.
17. Robinson DR, Wu YM, Lin SF. The protein tyrosine kinase family of the human genome. Oncogene. 2000;19(49):5548-5557.
18. Farazi PA, DePinho RA. Hepatocellular carcinoma pathogenesis: from genes to environment. Nat Rev Cancer. 2006;6(9):674-687. (Pubitemid 44286000)
19. Roberts LR, Gores GJ. Hepatocellular carcinoma: molecular pathways and new therapeutic targets. Semin Liver Dis. 2005;25(2):212-225. (Pubitemid 40781073)
20. Llovet JM, Ricci S, Mazzaferro V, et al. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med. 2008;359(4):378-390.
21. Cheng AL, Kang YK, Lin DY, et al. Sunitinib versus sorafenib in advanced hepatocellular cancer: results of a randomized phase III trial. J Clin Oncol. 2013;31(32):4067-4075.
22. Cainap C, Qin S, Huang WT. Phase III trial of linifanib versus sorafenib in patients with advanced hepatocellular carcinoma (HCC). J Clin Oncol. 2013;30 Suppl 34:249.
23. Johnson PJ, Qin S, Park JW, et al. Brivanib versus sorafenib as first-line therapy in patients with unresectable, advanced hepatocellular carcinoma: results from the randomized phase III BRISK-FL study. J Clin Oncol. 2013;31(28):3517-3524.
24. Zhu AX, Rosmorduc O, Evans J, et al. SEARCH: a phase III, randomized, double-blind, placebo-controlled trial of sorafenib plus erlotinib in patients with advanced hepatocellular carcinoma (HCC). Eur J Cancer. 2012;48 Suppl:2.
25. El-Serag HB, Rudolph KL. Hepatocellular carcinoma: epidemiology and molecular carcinogenesis. Gastroenterology. 2007;132(7):2557-2576. (Pubitemid 46890874)
26. Llovet JM, Decaens T, Raoul JL, et al. Brivanib in patients with advanced hepatocellular carcinoma who were intolerant to sorafenib or for whom sorafenib failed: results from the randomized phase III BRISK-PS study. J Clin Oncol. 2013;31(28):3509-3516.
27. Wilhelm SM, Carter C, Tang L, et al. BAY 43-9006 exhibits broad spectrum oral antitumor activity and targets the RAF/MEK/ERK pathway and receptor tyrosine kinases involved in tumor progression and angiogenesis. Cancer Res. 2004;64(19):7099-7109. (Pubitemid 39331023)
28. Fabian MA, Biggs WH 3rd, Treiber DK, et al. A small molecule-kinase interaction map for clinical kinase inhibitors. Nat Biotechnol. 2005;23(3):329-336. (Pubitemid 41094421)
29. Wilhelm S, Carter C, Lynch M, et al. Discovery and development of sorafenib: a multikinase inhibitor for treating cancer. Nat Rev Drug Discov. 2006;5(10):835-844. (Pubitemid 44480537)
30. Strumberg D, Schultheis B. Regorafenib for cancer. Expert Opin Investig Drugs. 2012;21(6):879-889.
31. Wilhelm SM, Dumas J, Adnane L, et al. Regorafenib (BAY 73-4506): a new oral multikinase inhibitor of angiogenic, stromal and oncogenic receptor tyrosine kinases with potent preclinical antitumor activity. Int J Cancer. 2011;129(1):245-255.
32. Solimando DA Jr, Waddell JA. Drug monographs: bosutinib and Regorafenib. Hosp Pharm. 2013;48(3):190-194.
33. Mross K, Frost A, Steinbild S, et al. A phase I dose-escalation study of Regorafenib (BAY 73-4506), an inhibitor of oncogenic, angiogenic, and stromal kinases, in patients with advanced solid tumors. Clin Cancer Res. 2012;18(9):2658-2667.
34. Bruix J, Tak WY, Gasbarrini A, et al. Regorafenib as second-line therapy for intermediate or advanced hepatocellular carcinoma: multicentre, open-label, phase II safety study. Eur J Cancer. 2013;49(16):3412-3419.
35. George S, Wang Q, Heinrich MC, et al. Efficacy and safety of Regorafenib in patients with metastatic and/or unresectable GI stromal tumor after failure of imatinib and sunitinib: a multicenter phase II trial. J Clin Oncol. 2012;30(19):2401-2407.
36. Strumberg D, Scheulen ME, Schultheis B, et al. Regorafenib (BAY 73-4506) in advanced colorectal cancer: a phase I study. Br J Cancer. 2012;106(11):1722-1727.
37. Eisen T, Joensuu H, Nathan PD, et al. Regorafenib for patients with previously untreated metastatic or unresectable renal-cell carcinoma: a single-group phase 2 trial. Lancet Oncol. 2012;13(10):1055-1062.
38. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst. 2000;92(3):205-216.
39. Faivre S, Raymond E, Boucher E, et al. Safety and efficacy of sunitinib in patients with advanced hepatocellular carcinoma: an open-label, multicentre, phase II study. Lancet Oncol. 2009;10(8):794-800.
40. de Lope CR, Tremosini S, Forner A, Reig M, Bruix J. Management of HCC. J Hepatol. 2012;56 Suppl 1:S75-S87.
41. Pawlik TM, Reyes DK, Cosgrove D, Kamel IR, Bhagat N, Geschwind JF. Phase II trial of sorafenib combined with concurrent transarterial chemoembolization with drug-eluting beads for hepatocellular carcinoma. J Clin Oncol. 2011;29(30):3960-3967.
42. Park JW, Koh YH, Kim HB, et al. Phase II study of concurrent transarterial chemoembolization and sorafenib in patients with unresectable hepatocellular carcinoma. J Hepatoly. 2012;56(6):1336-1342.
43. Kudo M, Imanaka K, Chida N, et al. Phase III study of sorafenib after transarterial chemoembolisation in Japanese and Korean patients with unresectable hepatocellular carcinoma. Eur J Cancer. 2011;47(14):2117-2127.
44. Abdel-Rahman O, Elsayed ZA. Combination trans arterial chemoembolization (TACE) plus sorafenib for the management of unresectable hepatocellular carcinoma: a systematic review of the literature. Dig Dis Sci. 2013;58(12):3389-3396.