Hereditary Angioedema (HAE) is a very rare genetic condition. It affects around 1 in 50,000 people*. We interviewed Chloe to find out about her experience of taking part in a drug study.
My body does not recognise when to stop swelling
Living with a condition such as HAE means that I have spontaneous painful swelling attacks across various parts of my body. For example, if my hand swelled up it would just keep going and going. Severe attacks can leave me hospitalised and are extremely disruptive to everyday life.
North Bristol NHS Trust run the ISIS 721744-CS5 study to test the effectiveness of a drug designed to lower the amount of a certain protein produced by the liver. When this protein enters the blood stream it can lead to swelling attacks in patients with HAE. To test the effectiveness of the treatment, researchers monitored the patient’s symptoms and number of attacks. The study was randomised and placebo-controlled, meaning participants will not know if they are receiving the test drug or a placebo.
I took part in the study because it reduced the number of injections
Since the age of 15, I have received intravenous injections every four to six days. Now, aged 32, my veins were feeling tired, and it was having a big impact on my life. After hearing about a study that meant having significantly less injections, it felt like a good option. I'd also exhausted all options available on my treatment plan.
I got to understand how every part of my body is working
One of the benefits of taking part in research was having lots of different tests done, it's like having a regular overall health check. I attend the hospital every two to three weeks to receive an injection, talk to my Consultant and ask questions to the Research Team about anything that I'm worried about. I then receive a weekly call to check on my symptoms and to discuss whether I'm still happy to participate in the study. I feel like I've had a choice all the way through whether to participate or withdraw.
Since starting the study, I have had no attacks.
I've gone from having one every four to six days to none in over a year – that has dramatically changed my life. Although I do not know whether I am taking the test drug or a placebo, I am definitely feeling less fatigued and have now taken up running. I'm more active and generally less fearful about ordinary life. Life feels more normal again.
It has been so life changing for me, I have enrolled my daughter onto a similar study for her age group
With this being a hereditary condition, my daughter also has HAE. After my positive experience, I was thrilled to discover she could take part in a similar research study suited to her age group.
For anyone thinking about taking part in research
I would encourage you to talk through all your concerns and fears with the Research Team and make sure that every anxiety is attended to. They can provide you with information to reassure you to make a well-informed decision. The team were so supportive and have been very friendly. My opinion about going on drug studies has completely changed. I encourage you to give it a go if it feels like it might help. You can end at it any time.
Further Information
- Discover more about our Current Research
- Read/watch more Patient Stories
Video Transcript
My name's Chloe. I have a condition called HEA, which is Hereditary Angioedema and I basically don't have enough enzymes to tell my body to stop swelling.
I took part in the trial because I was having to take injections intravenously every 4 to 6 days to manage my health. And after hearing about a trial, that meant I would have to take an injection significantly less, it felt like a really good option. And also, I'd exhausted my options with what was available for my treatment plan.
From my experience, it’s been life changing and very, very positive to the point that I've put my daughter, who has the same hereditary condition on a trial for her age.
So I have had no attacks. I've gone from having one every 4 to 6 days to none, which has been for over a year now. And that has dramatically changed my life. And the simple things in life is not being as fatigued I've taken up running.
I'm more active generally and less fearful about ordinary life. Life feels more normal again.
My opinion about going on trials has completely changed through doing one. So I encourage you to give it a go, if it feels like it might help. And to really know that you can end it any time.