How to avoid being surprised by hepatotoxicity at the final stages of drug development and approval

Clinics in Liver Disease

Volumn 17, Issue 4, 2013, Pages 749-767

  Regev, Arie ^a  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Causality assessment; Drug development; Drug induced liver injury; Hy's Law

Indexed keywords

ACETYLSALICYLIC ACID; ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; ASPARTATE AMINOTRANSFERASE; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; BILIRUBIN; BILIRUBIN GLUCURONIDE; BROMFENAC; DELEVALOL; GLUTAMATE DEHYDROGENASE; HEPARIN; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; IDITOL DEHYDROGENASE; ISONIAZID; MICRORNA 122; NEW DRUG; PLACEBO; TACRINE; TIENILIC ACID; TROGLITAZONE; UNCLASSIFIED DRUG; XIMELAGATRAN;

ABDOMINAL DISCOMFORT; ABDOMINAL TENDERNESS; ACUTE LIVER FAILURE; ADAPTATION; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALCOHOL CONSUMPTION; ALKALINE PHOSPHATASE BLOOD LEVEL; ASCITES; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BILIRUBIN BLOOD LEVEL; CHOLESTASIS; CLINICAL PRACTICE; DEVELOPMENTAL STAGE; DRUG ANALYSIS; DRUG APPROVAL; DRUG DEVELOPMENT; DRUG FATALITY; DRUG INDUCED DISEASE; DRUG INDUSTRY; DRUG MARKETING; DRUG SAFETY; DRUG WITHDRAWAL; EOSINOPHILIA; FATIGUE; FEVER; FOOD AND DRUG ADMINISTRATION; HEPATIC ENCEPHALOPATHY; HUMAN; HYPERSENSITIVITY; HYPERTENSION; INTERNATIONAL NORMALIZED RATIO; JAUNDICE; LIVER CELL DAMAGE; LIVER DYSFUNCTION; LIVER FAILURE; LIVER INJURY; LIVER TOXICITY; LIVER TRANSPLANTATION; LYMPHADENOPATHY; MALAISE; NAUSEA; PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTIVE VALUE; PROTHROMBIN TIME; RASH; REVIEW; RISK FACTOR; SIDE EFFECT; TOXIC HEPATITIS; UPPER ABDOMINAL PAIN; VOMITING;

CAUSALITY ASSESSMENT; DRUG DEVELOPMENT; DRUG-INDUCED LIVER INJURY; HY'S LAW;

BIOMARKERS, PHARMACOLOGICAL; DRUG EVALUATION, PRECLINICAL; DRUG-INDUCED LIVER INJURY; HUMANS;

EID: 84884922742     PISSN: 10893261     EISSN: 15578224     Source Type: Journal    
DOI: 10.1016/j.cld.2013.07.014     Document Type: Review

References (56)

1. Navarro V.J., Senior J.R. Drug-related hepatotoxicity. NEngl J Med 2006, 354(7):731-739.
2. FDA, Guidance for industry. Drug-induced livery injury: premarketing clinical evaluation. July 2009 [online]. Available at:. Accessed December 17, 2009. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM174090.pdf.
3. Goodman Z. Drug hepatotoxicity. Clin Liver Dis 2002, 6(2):381-389.
4. Lewis J.H., Ahmed M., Shobassy A., et al. Drug-induced liver disease. Curr Opin Gastroenterol 2006, 22(3):223-233.
5. Manier J.W., Chang W.W., Kirchner J.P., et al. Hepatotoxicity associated with ticrynafen-a uricosuric diuretic. Am J Gastroenterol 1982, 77(6):401-404.
6. Zimmerman H.J., Lewis J.H., Ishak K.G., et al. Ticrynafen-associated hepatic injury: analysis of 340 cases. Hepatology 1984, 4(2):315-323.
7. Moses P.L., Schroeder B., Alkhatib O.N., et al. Severe hepatotoxicity associated with bromfenac sodium. Am J Gastroenterol 1999, 94(5):1393-1396.
8. Hunter E.B., Johnston P.E., Tanner G., et al. Bromfenac (Duract)-associated hepatic failure requiring liver transplant. Am J Gastroenterol 1999, 94(8):2299-2301.
9. Rabkin J.M., Smith M.J., Orloff S.L., et al. Fatal fulminant hepatitis associated with bromfenac use. Ann Pharmacother 1999, 33(9):945-947.
10. Fontana R.J., McCashland T.M., Benner K.G., et al. Acute liver failure associated with prolonged use of bromfenac leading to liver transplantation. Liver Transpl Surg 1999, 5(6):480-484.
11. Gitlin N., Julie N.L., Spurr C.L., et al. Two cases of severe clinical and histologic hepatotoxicity associated with troglitazone. Ann Intern Med 1998, 129(1):36-38.
12. Vella A., deGroen P.C., Dinneen S.F. Fatal hepatotoxicity associated with troglitazone. Ann Intern Med 1998, 129(12):1080.
13. Herrine S.K., Choudary C. Severe hepatotoxicity associated with troglitazone. Ann Intern Med 1999, 130(2):163-164.
14. Watkins P.B., Whitcomb R.W. Hepatic dysfunction associated with troglitazone. NEngl J Med 1998, 338:916-917.
15. Maria V.A., Victorino R.M. Hypersensitivity immune reaction as a mechanism for dilevalol-associated hepatitis. Ann Pharmacother 1992, 26(7):924-926.
16. He R. Clinical review of exanta (ximelagatran) tablets, FDA Cardiovascular and Renal Drugs Advisory Committee Briefing Information. 2004. Available at:. Accessed January 20, 2013. http://www.fda.gov/ohrms/dockets/ac/04/briefing/2004-4069B1_04_FDA-Backgrounder-MOR-180.pdf.
17. Lee W.M., Larrey D., Olsson R., et al. Hepatic findings in long-term clinical trials of ximelagatran. Drug Saf 2005, 28(4):351-370.
18. Senior John. Lessons from isoniazid. Would it be approved today? March, 2008. Available at:. Accessed December 12, 2012. http://www.fda.gov/downloads/Drugs/ScienceResearch/.../ucm076755.pdf.
19. Mitchell J.R., Long M.W., Thorgeirsson U.P., et al. Acetylation rates and monthly liver function tests during one year of isoniazid preventative therapy. Chest 1975, 68(2):181-190.
20. Nolan C.M., Goldberg S.V., Buskin S.E. Hepatotoxicity associated with isoniazid preventative therapy: a 7-year survey from a public health tuberculosis clinic. JAMA 1999, 281(11):1014-1018.
21. Harrill A.H., Roach J., Fier I., et al. The effects of heparins on the liver: application of mechanistic serum biomarkers in a randomized study in healthy volunteers. Clin Pharmacol Ther 2012, 92(2):214-220.
22. Olsson R., Korsan-Bengtsen B.M., Korsan-Bengtsen K., et al. Serum aminotransferases after low-dose heparin treatment. Acta Med Scand 1978, 204(3):229-230.
23. Dukes G.E., Sanders S.W., Russo J., et al. Transaminase elevations in patients receiving bovine or porcine heparin. Ann Intern Med 1984, 100(5):646-650.
24. Carlson M.K., Gleason P.P., Sen S. Elevation of hepatic transaminases after enoxaparin use: case report and review of unfractionated and low-molecular weight heparin induced hepatotoxicity. Pharmacotherapy 2001, 21(1):108-113.
25. Watkins P.B., Zimmerman H.J., Knapp M.J., et al. Hepatotoxic effects of tacrine administration in patients with Alzheimer's disease. JAMA 1994, 271:992-998.
26. NIDDK, LiverTox, Clinical and research information on drug-induced liver injury. Available at:. Accessed December 20, 2012. http://livertox.nlm.nih.gov/Isoniazid.htm.
27. Lucena M.I., Molokhia M., Shen Y., et al. Susceptibility to amoxicillin-clavulanate-induced liver injury is influenced by multiple HLA class I and II alleles. Gastroenterology 2011, 141(1):338-347.
28. Kleiner D.E., Gaffey M.J., Sallie R., et al. Histopathologic changes associated with fialuridine hepatotoxicity. Mod Pathol 1997, 10(3):192-199.
29. Berends M.A., Snoek J., De Jong J., et al. Liver injury in long-term methotrexate treatment in psoriasis is relatively infrequent. Aliment Pharmacol Ther 2006, 24:805-811.
30. Temple R. Hy's law: predicting serious hepatotoxicity. Pharmacoepidemiol Drug Saf 2006, 15:241-243.
31. Zimmerman H.J. Drug-induced liver disease. Hepatotoxicity, the adverse effects of drugs and other chemicals on the liver 1999, 428-433. Lippincott Williams & Wilkins, Philadelphia. 2nd edition.
32. Senior J.R. Regulatory perspectives. Drug-induced liver disease 2003, 739-754. Marcel Dekker, New York. N. Kaplowitz, L.D. DeLeve (Eds.).
33. Björnsson E., Olsson R. Outcome and prognostic markers in severe drug-induced liver disease. Hepatology 2005, 42:481-489.
34. Andrade R.J., Lucena M.I., Fernandez M.C., et al. Drug-induced liver injury: an analysis of 461 incidences submitted to the Spanish registry over a 10-year period. Gastroenterology 2005, 129:512-521.
35. Guo T, Gelperin K, Senior J. A tool to help you decide (detect potentially serious liver injury). March 2008 [online]. Available at:. Accessed December 17, 2009. http://www.fda.gov/downloads/Drugs/ScienceResearch/ResearchAreas/ucm076777.pdf.
36. Watkins P.B., Desai M., Berkowitz S.D., et al. Evaluation of drug-induced serious hepatotoxicity (eDISH) application of this data organization approach to phaseIII clinical trials of rivaroxaban after total hip or knee replacement surgery. Drug Saf 2011, 34(3):243-252.
37. Chitturi S., Farrell G.C. Drug induced liver disease. Schiff's diseases of the liver 2012, 703-782. Wiley-Blackwell, Oxford, UK. 11th edition. E.R. Schiff, W.C. Maddrey, M.F. Sorrell (Eds.).
38. Danan G., Benichou C. Causality assessment of adverse reactions to drugs: I. A novel method based on the conclusions of international consensus meetings: applications to drug induced liver injury. JClin Epidemiol 1993, 46:1323-1330.
39. Maria V.A., Victorino A.J. Development and validation of a clinical scale for the diagnosis of drug-induced hepatitis. Hepatology 1997, 26:664-669.
40. Rochon J., Protiva P., Seef L.B., et al. Reliability of the Roussel Uclaf causality assessment method for assessing causality in drug-induced liver injury. Hepatology 2008, 48:1175-1183.
41. Papay J.I., Clines D., Rafi R., et al. Drug-induced liver injury following positive drug rechallenge. Regul Toxicol Pharmacol 2009, 54(1):84-90.
42. Brown G., Copeland T., Willward M., et al. Monadic testing of new products-an old problem and some partial solutions. JMark Res Soc 1973, 15:112-131.
43. Friedman H.H., Amoo T. Rating the rating scales. JMarket Manag 1999, 9:114-123.
44. Aithal G.P., Watkins P.B., Andrade R.J., et al. Case definition and phenotype standardization in drug-induced liver injury. Clin Pharmacol Ther 2011, 89(6):806-815.
45. Kowdley K.V., Wang C.C., Welch S., et al. Prevalence of chronic hepatitis B among foreign-born persons living in the United States by country of origin. Hepatology 2012, 56(2):422-433.
46. Kunihom M.H., Purcell R.H., McQuillan G.M., et al. Epidemiology of hepatitis E virus in the United States: results from the third National Health and National Health and Nutritional Examination survey, 1988-1994. JInfect Dis 2009, 200:48-56.
47. Laurent-Puig P., Dussaix E., de Paillette L., et al. Prevalence of hepatitis C RNA in suspected drug induced liver injury. JHepatol 1993, 19:487-489. [letter].
48. Davern T.J., Chalasani N., Fontana R.J., et al. Acute hepatitis E infection accounts for some cases of suspected drug-induced liver injury. Gastroenterology 2011, 141:1665-1672.
49. Desmet V.J., Gerber M., Hoofnagle J.H., et al. Classification of chronic hepatitis: diagnosis, grading and staging. Hepatology 1994, 19:1513-1520.
50. Czaja A.J. Acute and acute severe (fulminant) autoimmune hepatitis. Dig Dis Sci 2013, 58(4):897-914.
51. Whitehead M.W., Hawkes N.D., Hainsworth I., et al. Aprospective study of the causes of notably raised aspartate aminotransferase of liver origin. Gut 1999, 45(1):129.
52. Bernal W., Ma Y., Smith H.M., et al. The significance of autoantibodies and immunoglobulins in acute liver failure: a cohort study. JHepatol 2007, 47(5):664-670.
53. Marudanayagam R., Shanmugam V., Gunson B., et al. Aetiology and outcome of acute liver failure. HPB (Oxford) 2009, 11(5):429-434.
54. Mindikoglu A.L., Regev A., Schiff E.R. Hepatitis B virus reactivation after cytotoxic chemotherapy: the disease and its prevention. Clin Gastroenterol Hepatol 2006, 4:1076-1081.
55. Rosenberg P., Urwitz H., Johannesson A., et al. Psoriasis patients with diabetes type 2 are at high risk of developing liver fibrosis during methotrexate treatment. JHepatol 2007, 46:1111-1118.
56. Chalasani N., Bjornsson E. Risk factors for idiosyncratic drug-induced liver injury. Gastroenterology 2010, 138:2246-2259.