Luca and Susan’s story
The nurses have taught me how to inject myself… I love being able to take part in sports again at school.
Luca, 13, was invited to take part in a clinical research study at Royal Manchester Children’s Hospital (RMCH) after being diagnosed with immune thrombocytopenia (ITP).
ITP is an autoimmune disease affecting platelets. Around 400 children in the UK develop ITP each year, although the majority get better spontaneously.
Platelets are minuscule components in the blood which help to clot it when the body is injured. In ITP, the body mistakenly creates antibodies to fight against platelets, meaning that they do not work so well. A normal number of platelets is between 150 and 400 x 109 per litre. If someone has too many platelets, their blood will clot too easily, and if someone does not have enough platelets, they may bruise and bleed more easily.
Many people have no symptoms for ITP, but some, as in Luca’s case, suffer with severe bleeding, bruising and develop a rash, called a purpura.
The three year study, led by Dr John Grainger, Consultant Paediatric Haematologist at RMCH aims to find out if experimental drug, romiplostim is safe in children with ITP and whether it causes any side effects. It will also look to see if the medication can raise the platelet counts in children with ITP and to evaluate changes in bone marrow structure after long-term exposure.
Romiplostim is still experimental in children with ITP, but is approved by regulatory health agencies for adults with ITP.
200 young people are taking part in the study, at centres across the world including in Australia, Canada, Europe, Israel, South Africa, South America and the United States.
Here, Luca, from Congleton in Cheshire, explains more about his clinical research journey…
I was 11-years-old when I suffered from an asthma attack and was admitted to hospital. Doctors put sticky plasters on my chest to monitor my heart rate and when they were removed, I noticed a pinprick rash and bruising where they had been. I also kept bruising very easily, and suffered from blood blisters on my lips. I was referred to Royal Manchester Children’s Hospital by doctors at my local hospital near home, and I met Dr Grainger, who diagnosed ITP.
My condition seemed to get worse and I suffered with bad nose bleeds and more bruising. I was given medication and steroids but experienced a bad allergic reaction with sickness and fatigue.
I was not allowed to take part in sports– my favourite hobby – including skateboarding, football and athletics, in case I was injured and suffered with bleeding and bruising. My mum had to come and collect me from school during my sports lessons. That was a sad time for me and I wanted my condition to go away.
Instead of school sports, I started taking tennis lessons including hitting the ball very gently and playing easy-going matches with my mum – but even if the ball accidentally knocked me, I would bruise easily!
I was then invited to take part in the clinical research study; I was the second child in the UK to sign up. The medication is injected under my skin to the abdominal area, thigh or upper arm once every week. At first, I needed to attend regular study visits where I had a number of procedures, including disease assessments, general and safety assessments, urine and blood tests.
After eight weeks, the nurses taught my mum and I how to mix the romiplostim and how to give the injection on our own, so that we were able to do it at home and only had to come into hospital once every month. We are now responsible for storing the romiplostim in the fridge, recording the date and time that the injection was given in the diary and returning the empty containers and record to hospital.
Since taking part in the study, I have been on a skiing holiday with school and on holiday to Portugal with my best friend during half term, where I administered my own injections. It is a long process but after 60 weeks on the research study, I can say that it has changed my life for the better!
Luca’s mum, Susan, says she had no hesitations about allowing her son to take part in the study…
Luca suffered from ITP when he was younger, but as with most cases of the condition in children, it went away on its own, without any treatment. When he was 11 though, and we noticed the onset of bruising, rashes, nose bleeds and fatigue, I knew that the ITP has returned.
For me, as Luca’s mother, seeing him so disappointed that he could not take part in his favourite sports lessons at school was very disheartening. He did not really open up about his condition and was very quiet. That’s why I wanted to keep him busy with other activities at home.
When we were first approached about the possibility of Luca taking part in a research study, I had absolutely no hesitations; we wanted to do it, and to play our part in helping someone else in the future.
Taking part in the study has been a long and demanding journey. We have visited the hospital weekly for over a year now. At first, I thought the medication would just zap the condition away, but of course that is not the case and we have experienced peaks of jubilation and troughs of disappointment throughout the process.
ITP is a hot topic now when I chat to other mums and parents. I want to raise awareness of the condition and tell people about the symptoms to look out for, explain what a platelet count is and what it means for their children. I never thought in my life I’d be so obsessed by numbers! One week Luca’s count soared up to 700, but at the moment we’re at a steady and satisfactory platelet count of 278.
I would say to other parents of children taking part in research studies: don’t presume the condition is just going to go away; take each week as it comes and keep a diary, to retain a record of good and bad weeks. I have kept a diary for two years now to monitor Luca’s progress.
The staff at Royal Manchester Children’s Hospital have been absolutely brilliant! They make you feel so at ease and we have got to know the team very well over the last two years.
The nurses explain everything so thoroughly so that you understand exactly what is happening. They make sure you are happy and feel confident in administering the study drug on your own, which makes such a difference to travelling to hospital every week to receive the drug, as now we’re able to do it safely at home.
I am so pleased with Luca’s progress; the fact that he’s now well enough to partake in sports again at school, and go on two holidays, just shows the improvement in his condition and I am so thankful to the research staff here in Manchester for making it all possible for him.