Zane's Clinical Trial Experience

By - Gemma, mum to Zane

My 8 year old son Zane has been on a clinical trial for an extended half life Factor VIII product now for over 5 years. Zane has severe haemophilia A and was diagnosed at a year old due to large, lumpy bruising that was appearing all over his body from about 8 months, when he started rolling and moving around. From about 22 months, during a year stay in Australia, Zane suffered his first few spontaneous joint bleeds, which is when he started on prophylaxis and we learnt to infuse, culminating in him having Factor VIII every 48 hours from the age of 2.

On returning to England in the winter of 2013, our lovely nurse at the time called us from the Oxford Haemophilia Centre, telling us that a new long lasting Factor VIII trial was about to start and that Zane would only have to be injected with Factor VIII twice weekly, which was significantly less than the every 48 hours we were currently doing. We trusted our nurse when she told us that Zane was suitable for this trial and that it was a good one.

I remember that we didn't have very long to make this decision and we had so much to weigh up. Zane hadn't had any bleeds or problems since he'd been on prophylaxis and part of me felt that if things weren't broken then there's no need to fix it. So much was going through my head and although there were no major obvious risks involved we were going into the unknown. Were we putting Zane at risk of inhibitors by changing his product and would the Factor VIII give him enough coverage with such a big gap? So many unanswered questions!

Thank goodness for my hero of a husband, Jon, who is so forward thinking and very positive and always had been about Zane and his condition! If this product worked it would change Zane's life and also help towards improving the lives of others with haemophilia, which would be amazing. One of the biggest issues with haemophilia is vein access, especially in small children and how wonderful it would be if intravenous injections only had to be done twice a week instead of every other day. We have always been lucky with Zane, as even from as young as 18 months he has always had good veins so we didn't even have to consider a port. The other thing to consider is the reduced stress on the parents life of only having to infuse twice weekly instead of waking up with that dread every other day. It gives us parents a break too! Think also of the reduced quantity of boxes we would have to take on holiday too. Lots of benefits.

So as you can probably guess, we went for it and 5 years on we've never looked back. Zane has his Factor on a Monday morning and a Thursday evening. We have recently changed this, as we were previously doing a Monday morning and Friday morning but Zane does lots of sport and the long stretch mid-week was leading to a few niggles. As you all know, a mere bruise can turn into something quite nasty if there is little Factor in the blood, so we were finding that we were having to administer early doses on Thursday which we've now stuck with through recommendations from our wonderful Consultant and nurses at Oxford.

I can happily say that the disadvantages of the trial are barely noticeable - three monthly blood test, bulky packaging, a little paperwork and the fact that you have to pull exact tiny ml's through into the syringe to administer (it changes with Zane's weight) each dose are all things that you get used to very quickly and are a very small price to pay for Zane's fantastic quality of life on the product. We also get to have free lunch at Pret when we visit too which is a real treat as well as the gold star health service that being on a trial brings.

Most importantly, Zane is a happy, healthy and strong 8 year old boy. He's always the one that you find at the top of every tree and I honestly believe that he will never let Haemophilia get in the way of doing anything !!! He is my hero and never complains about the problems he faces. He has an amazing big sister who supports and loves him so much and we make sure that we make the most of and enjoy every day and try and let the little worries pass us by. This week has been very momentous as Zane has been learning to self infuse – something that took us months to master, seems to have taken Zane about 10 minutes to learn!