Taking part in a clinical research trial has allowed eight year old Spiderman-fan, George, to make the most of his childhood.
When George was around four years old, we noticed he had a rash. I was worried that it might be meningitis and took him to A&E. After being referred to Royal Manchester Children’s Hospital (RMCH), George was diagnosed with immune thrombocytopenia (ITP), a rare blood disorder that causes those affected to bruise easily and can cause severe bleeding.
It was frightening, but the doctor explained that for many children the condition clears up on its own. But, then at Christmas, George’s nose started bleeding and just wouldn’t stop. We had to rush him to our local A&E, and by infusing immunoglobulin doctors were eventually able to stop the bleeding.
Nose bleeds became a regular occurrence for George and it became clear that he needed some other kind of treatment. We found ourselves visiting A&E two or three times a month with the nose bleeds.
At four years old, George was quite boisterous and I’d find myself chasing after him – trying unsuccessfully to wrap a very little active boy up in cotton wool.
For George, the condition meant that he would miss out on school activities and children’s parties. People were understandably nervous about what would happen if he had a nose bleed whilst in their care.
I remember the first time George rode a bike and he broke his arm. George’s condition meant that his arm swelled up more than a normal breakage. We also couldn’t just take him to the local hospital for surgery, as he needed specialist care.
As a busy mum of three and a full-time nurse, George’s condition was also difficult for me and my husband, Gavin, to manage. George’s nose bleeds always seemed destined to happen on a Friday – out of hours, when getting treatment was difficult.
The condition also interfered with my job. As a nurse I don’t get many weekends off and I’d need to swap shifts at short notice to be able to take George to the hospital.
Dr Grainger and the haematology research nurse team at Royal Manchester Children’s Hospital worked with us to really carefully consider the treatment options available for George.
After talking through the options, we decided that taking part in a research study trialling eltrombopag, a powder which is dissolved in water* and taken orally, and already used in adults with ITP, was the best option. Although at four George was the youngest child in the UK to be part of the study, it was reassuring to know that some older children on the trial were already responding well to the treatment.
The effect of this treatment has been remarkable. Touch wood our trips to A&E are over, as George now rarely experiences nose bleeds and when he does they tend to stop within about five minutes. We have taken a decision to give George his medication at 3am each morning, which can be tough, but it’s something we’ve got used to and means that George’s diet isn’t restricted. You have to leave a gap between taking eltrombopag and eating some foods (e.g. milk), so this way he is able to eat what everyone else is eating and at the same time. Staff at the haematology clinic are monitoring George closely for any side effects to the treatment which is reassuring.
George is happy go lucky. Like most boys his age, he thinks he’s Spiderman and enjoys playing with friends. These days it’s much easier for George to take part in social activities. One of the nurses at RMCH has also provided some training to staff at George’s school. This has resulted in less disruption George’s schooling, as they are now confident in differentiating between symptoms that need medical attention and those that don’t.
George has just celebrated his eighth birthday with friends in style at a trampoline park, something which would have been too risky for him to enjoy previously.
Did you know?
As a result of this study, researchers have demonstrated for the first time the effectiveness of treatment, eltrombopag, in children with persistent or chronic immune thrombocytopenia (ITP). In Manchester the study was conducted by the ITP Centre, in partnership with the NIHR / Wellcome Trust Manchester Clinical Research Facility (CRF).
Being a research nurse working at the cutting edge of improving care for patients is rewarding work. Learn more about Anne and Sharon’s experiences in delivering the eltrombopag trial.
*Eltrombopag is available as a tablet for older children or as a suspension (powder which was then diluted) for younger children unable to take tablets.