European pharmacogenomics approach to coumarin therapy

EU-PACT

Grant Agreement number:HEALTH-F2-2009-223062

EC contribution:€2.5 million

Duration in months: 48

Project type:Medium sized collaborative project

Starting date:1 October 2008

Project web-site:

Background:The narrow therapeutic range of coumarins and an unpredictable response due to large intra- and inter-patient variability makes careful patient monitoring necessary. The principal covariates (together accounting for 35-50% of the variability) are polymorphisms in genes encoding for CYP2C9 and VKORC1.It is not known whether genotype-guided dosing during induction and maintenance therapy of coumarins will increase the efficacy and safety of the treatment.

Objectives:The aim of the EU-PACT study is to determine whether a clinically applicable dosing algorithm including genetic information of the patient, clinical data and (when available) last INR increases the time in range (TIR) of the INR during anticoagulation with acenocoumarol, phenprocoumon and warfarin compared to a dosing algorithm without these genotypes.

Approach and methodology: Two dosing algorithms will be investigated in the EU-PACT trial. The trial will be performed at 13 centers in 7 European countries. The study will be a two-armed, single-blinded, randomized controlled trial in which genotype-guided dosing is compared to a standard clinical dosing algorithm with a maximum follow-up duration of 3 months. Patients receiving genotype-guided dosing will be genotyped with a point of care test before the first dose. In order to demonstrate that genotype-guided dosing improves TIR of the INR by 5% compared to the control regimen and assuming a 10% dropout after study entry, a minimum of 985 patients per drug should be recruited (80% power, 5% significance level and a mean %TIR of the INR of 47 to 63.4).

Expected results: It is expected that patients randomized to the genotype-guided dosing arm will spent a lager percentage of the time within the target range. Since TIR is closely related to bleeding and recurrent thrombosis risks, it is also expected that patients dosed according to the genotype-guided dosing algorithm will experience less bleedings and less recurrent thrombosis.

Potential impact:Bleedings and recurrent thrombosis are common in patients on coumarin therapy. If the genotype-guided dosing regimen shows an improvement inTIR, and therefore an improvement in the efficacy and safety of coumarin treatment, then it will help to reduce the frequency of one of Europe’s most common causes of drug-related hospital admissions and death.

Keywords: EU-PACT,pharmacogenomics, coumarin therapy, warfarin, CYP2C9, VKORC1

Co-ordinator (Name, Full address, Tel, Fax, e-mail (and web-site of organisation, if applicable)

A.H. Maitland-van der Zee

UtrechtUniversity, Faculty of Science

Utrecht Institute for Pharmaceutical Sciences

Division of Pharmacoepidemiology and Pharmacotherapy

Sorbonnelaan 16

3584CA Utrecht, The Netherlands

PO Box 80082

3508 TB Utrecht, The Netherlands

Tel +31-302534042

Fax +31-302539166

E-mail:

Website: and

Partners (Name, Full address)

A. de Boer

UtrechtUniversity, Faculty of Science

Utrecht Institute for Pharmaceutical Sciences

Division of Pharmacoepidemiology and Pharmacotherapy

PO Box 80082

3508 TB Utrecht, The Netherlands

F.R. Rosendaal

Leiden University Medical Center

Clinical Epidemiology

Albinusdreef 2

2333ZA Leiden, The Netherlands

W.K. Redekop

Erasmus Universitair Medisch Centrum Rotterdam Erasmus MC

Gravendijkwal 230

3015 CE Rotterdam, The Netherlands

J. Kirchheiner

Universitaet Ulm

Institute of Natural Medicine and Clinical Pharmacology

Helmholtzstrasse 16

89081 Ulm, Germany

A.K. Daly

University of Newcastle upon Tyne

6 Kensington Terrace

NE1 7RU Newcastle Upon Tyne, United Kingdom

F. Kamali

University of Newcastle upon Tyne

6 Kensington Terrace

NE1 7RU Newcastle Upon Tyne, United Kingdom

M. Pirmohamed

The University of Liverpool

Brownlow Hill, FoundationBuilding 765

L69 7ZX Liverpool, United Kingdom

R. Barallon

LGC Limited

Queens Road

TW11 OLY, TeddingtonMiddlesex, United Kingdom

L. Bequemont

Université Paris-Sud XI

Rue Georges Clemenceau 15

91405 Orsay, France

M. Wadelius

UppsalaUniversity

St Olofsgatan 10B

75105 Uppsala, Sweden

V.G. Manolopoulos

DemocritusUniversity of Thrace

Panepistimioupoli

69100 Komotini, Greece

E. Haschke-Becher

KH ElisabethinenLinz

Fadingerstrasse 1

4010 Linz, Austria

M. Briz