Pharmacokinetics Study

It is an exciting time for haemophilia care in Ireland. Improving patient care is to the forefront of our research focus in the National Coagulation Centre (NCC). Our aim is to personalise and individualise treatment for people with haemophilia. The first phase of our state of the art research focuses on adults with mild, moderate or severe haemophilia A.

Tailoring treatment to your body’s needs:
If you have haemophilia A and require surgery or have a bleed we use medications to boost your factor VIII level. These are dosed according to your weight and your factor VIII level. Your body gradually breaks down the medication and your factor VIII returns to your normal level. No two people are the same and this also applies to the rate at which your body breaks down factor VIII. Some people will do this very slowly and therefore should need less medication while others will break down factor VIII very quickly and need higher doses to prevent bleeding.

We know these differences exist – how can we use this information to improve your care?
Pharmacokinetics (PK) involves a series of blood tests through which we can find out the rate that your body breaks down factor VIII. Once we know your PKs it will become part of your patient record in the NCC. We hope that we will be able to use this information in the future to develop a treatment plan specifically for you. There are very few other areas in hospital medicine that are using this state of the art approach to patient care. We are delighted to offer this opportunity to our patients.

We started PK studies in the NCC in July 2013 and invited people with haemophilia A to attend. We were extremely pleased to receive such an enthusiastic response to this project and would like to thank everyone who has already responded and attended.

As this is a national research study we hope to involve as many people as possible to get a true reflection of people with haemophilia A in Ireland. The Cork Comprehensive Coagulation Centre is working with us to offer PK studies in Cork University Hospital. For participants travelling from other parts of Ireland to St. James’s Hospital for PK studies, the Irish Haemophilia Society has kindly offered the use of the patient apartment if required.

Taking this concept further: PERSONAL Clinical trial
The second phase of our research is to actually put PK dosing into practice. We will be offering all people with severe Haemophilia A on regular prophylaxis (at least 5 times per fortnight) the opportunity to use their PK results to individualise their prophylaxis. We will calculate out what the exact dose of factor VIII required by your body to ensure your factor VIII levels are always optimal. We hope to show that individualising prophylaxis is at least as good, if not better, than current prophylactic regimens.

It is important to note that this is not a trial of a new drug, we will be using your current medication but matching the dose according to your individual requirements. This trial is called the PERSONAL trial (PERSONALising Factor VIII prophylaxis regimens: Efficacy of standard versus pharmacokinetically based regimens in adult patients with severe haemophilia A).

This innovative trial is extremely exciting. Not only is this the first trial of its kind but also we will collaborate with the world expert in PK studies and haemophilia, Professor Peter Collins. We aim to start this clinical trial in December 2013. People who choose to participate in the trial will be involved for 18 months in total. For the first 6 months they continue on their standard prophylaxis and for the subsequent 12 months change to individualised prophylaxis.

From his trial, we will study a number of outcomes. Firstly, how your body responds to the new personalised prophylaxis. Secondly, we will monitor peoples’ activity levels and feelings about how prophylaxis affects their quality of life. We hope that by including this information, we can build up a more holistic view of how people with severe haemophilia manage prophylaxis.

Finally, we will also offer regular MRIs and X-rays of elbows, ankles and knees to all participants on the PERSONAL trial. This will be combined with regular physiotherapy assessment. Through these joint health checks we hope to detect even subtle bleeds that may go otherwise unnoticed. This will help gain more information overall about joint disease in people with haemophilia A.

Our national research opportunity:
This research offers Ireland the opportunity to move to the forefront of haemophilia research. Not only do we hope that this research will be of benefit to patients in Ireland, but we aim that the results obtained may be used to help people with haemophilia internationally.

No research can possibly be successful without the support of our patients. Involvement in any research is completely voluntary and choosing not to participate will in no way affect the care you receive. If however, you are interested in participating, we will be more than happy to provide you with more information or arrange to meet you.

If you are considering participating or have further queries we have a dedicated email address pkstudy@stjames.ie or call me on 085 830 1599.

Dr. Michelle Lavin
National Coagulation Centre

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