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Safety of XimelagatranSafety of Ximelagatran

JAMA. 2005;293(23):2859-2860. doi:10.1001/jama.293.23.2859-a
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AUTHOR INFORMATION

Letters Section Editor: Robert M. Golub, MD, Senior Editor.

SAFETY OF XIMELAGATRAN

To the Editor: In his Editorial about ximelagatran,1 Dr Gurewich discusses the significant limitations of vitamin K antagonists for anticoagulation and the need for a therapeutic alternative, particularly for long-term anticoagulation, and we acknowledge these limitations. Studies of ximelagatran for short-term (7-12 days) thromboprophylaxis in knee replacement surgery patients,2 long-term (18 months) secondary prevention of recurrent venous thromboembolism (VTE) following treatment of VTE,3 and long-term (average 1.4 years) stroke prevention in patients with atrial fibrillation4 5 were conducted, and the commercial marketing application for ximelagatran for these uses was discussed at the US Food and Drug Administration’s Cardiovascular and Renal Drugs Advisory Committee meeting on September 10, 2004.6 We were concerned about the liver and cardiovascular risk findings. Major bleeding rates were not shown to be different between ximelagatran and warfarin. Efficacy for long-term use compared with warfarin, studied solely in the atrial fibrillation trials, showed conflicting results.

In the long-term studies,7 37 (approximately 1 in 200) patients treated with ximelagatran had concurrent elevations of levels of both alanine aminotransferase (ALT) and total bilirubin, connoting severe liver injury,8 including 9 deaths, 3 of them likely related to hepatic dysfunction. The Editorial advances the option that “diligent postmarketing surveillance” could suffice to study risk for delayed hepatotoxicity. However, analysis of data from the population with measurement of long-term end points shows that initial signs of liver injury (ALT levels >3 times the upper limit of normal) were observed during the first month of ximelagatran therapy in 6 of 37 patients who went on to develop severe liver injury (ALT levels >3 times the upper limit of normal and total bilirubin levels >2 times the upper limit of normal).7 The possibility that severe liver injury may occur during the first month of treatment cannot be excluded. We believe that postmarketing surveillance (eg, MedWatch reporting) is most appropriate for identification of new and unexpected events associated with drug therapy, not for formal quantification of a robust and clinically significant risk identified in preapproval studies. Monitoring patients in randomized, controlled studies is the best method for clarifying and quantifying the risk of ximelagatran-associated liver toxicity.

An increased rate of myocardial infarction/coronary artery disease events in patients randomized to ximelagatran was observed by Fiessinger et al in the THRIVE long-term deep vein thrombosis treatment trial9 and in the short-term knee replacement surgery studies, indicating possible cardiovascular toxicity. Randomized, controlled studies would also be needed to study this risk.

References
Gurewich V. Ximelagatran—promises and concerns.  JAMA. 2005;293736-739
PubMed
Francis CW, Berkowitz SD, Comp PC.  et al. EXULT A Study Group.  Comparison of ximelagatran with warfarin for the prevention of venous thromboembolism after total knee replacement.  N Engl J Med. 2003;3491703-1712
PubMed
Schulman S, Wahlander K, Lundstrom T, Clason SB, Eriksson H.THRIVE III Investigators.  Secondary prevention of venous thromboembolism with the oral direct thrombin inhibitor ximelagatran.  N Engl J Med. 2003;3491713-1721
PubMed
Olsson SB.Executive Steering Committee on behalf of the SPORTIF III Investigators.  Stroke prevention with the oral direct thrombin inhibitor ximelagatran compared with warfarin in patients with non-valvular atrial fibrillation (SPORTIF III): randomised controlled trial.  Lancet. 2003;3621691-1698
PubMed
SPORTIF Executive Steering Committee for the SPORTIF V Investigators.  Ximelagatran vs warfarin for stroke prevention in patients with nonvalvular atrial fibrillation: a randomized trial.  JAMA. 2005;293690-698
PubMed
Food and Drug Administration.  Transcript of September 10, 2004 Meeting of FDA Cardiovascular and Renal Drugs Advisory Committee. Available at: http://www.fda.gov/ohrms/dockets/ac/04/transcripts/2004-4069T1.htm. Accessed May 2, 2005
Food and Drug Administration.  Office of Drug Safety Review [of Ximelagatran]. September 10, 2004. Available at: http://www.fda.gov/ohrms/dockets/ac/04/briefing/2004-4069b1.htm. Accessed May 2, 2005
Food and Drug Administration Center for Drug Evaluation and Research (CDER).  Drug-Induced Liver Toxicity: Clinical White Paper. November 2000. Available at: http://www.fda.gov/cder/livertox/default.htm. Accessed May 2, 2005
Fiessinger JN, Huisman MV, Davidson BL.  et al. THRIVE Treatment Study Investigators.  Ximelagatran vs low-molecular-weight heparin and warfarin for the treatment of deep vein thrombosis: a randomized trial.  JAMA. 2005;293681-689
PubMed

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Gurewich V. Ximelagatran—promises and concerns.  JAMA. 2005;293736-739
PubMed
Francis CW, Berkowitz SD, Comp PC.  et al. EXULT A Study Group.  Comparison of ximelagatran with warfarin for the prevention of venous thromboembolism after total knee replacement.  N Engl J Med. 2003;3491703-1712
PubMed
Schulman S, Wahlander K, Lundstrom T, Clason SB, Eriksson H.THRIVE III Investigators.  Secondary prevention of venous thromboembolism with the oral direct thrombin inhibitor ximelagatran.  N Engl J Med. 2003;3491713-1721
PubMed
Olsson SB.Executive Steering Committee on behalf of the SPORTIF III Investigators.  Stroke prevention with the oral direct thrombin inhibitor ximelagatran compared with warfarin in patients with non-valvular atrial fibrillation (SPORTIF III): randomised controlled trial.  Lancet. 2003;3621691-1698
PubMed
SPORTIF Executive Steering Committee for the SPORTIF V Investigators.  Ximelagatran vs warfarin for stroke prevention in patients with nonvalvular atrial fibrillation: a randomized trial.  JAMA. 2005;293690-698
PubMed
Food and Drug Administration.  Transcript of September 10, 2004 Meeting of FDA Cardiovascular and Renal Drugs Advisory Committee. Available at: http://www.fda.gov/ohrms/dockets/ac/04/transcripts/2004-4069T1.htm. Accessed May 2, 2005
Food and Drug Administration.  Office of Drug Safety Review [of Ximelagatran]. September 10, 2004. Available at: http://www.fda.gov/ohrms/dockets/ac/04/briefing/2004-4069b1.htm. Accessed May 2, 2005
Food and Drug Administration Center for Drug Evaluation and Research (CDER).  Drug-Induced Liver Toxicity: Clinical White Paper. November 2000. Available at: http://www.fda.gov/cder/livertox/default.htm. Accessed May 2, 2005
Fiessinger JN, Huisman MV, Davidson BL.  et al. THRIVE Treatment Study Investigators.  Ximelagatran vs low-molecular-weight heparin and warfarin for the treatment of deep vein thrombosis: a randomized trial.  JAMA. 2005;293681-689
PubMed
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