Enzyme replacement therapy for paediatric patients
Pharmaxo Healthcare provides an enzyme replacement therapy service for paediatric patients with rare disorders. The service, managed by Pharmaxo Healthcare’s regional nurse manager and paediatric lead nurse Leanne Brant, allows children and young people to receive their treatment at home or at school.
How did the service begin and how did you get involved?
NHS England and the paediatric referral centres requested Pharmaxo Healthcare to consider extending our existing enzyme replacement therapy (ERT) service for adults to paediatric patients.
This service would be for lysosomal storage disorders such as mucopolysaccharidosis type I and II, also known as Hurler syndrome and Hunter syndrome, and Gaucher disease.
I saw the advertisement for a paediatric lead nurse to set up a new service and I thought it looked really interesting. My background is in ERT, and I was looking for a new challenge.
I started in April 2022. The first paediatric patient was referred to us from Birmingham Children’s Hospital (BCH) in May 2023. This patient was new to treatment and to homecare.
Why provide home- and school-based care?
Treatments can take anything from 40 minutes to five hours, and adding in travel, waiting and delays means that hospital-based appointments can take all day.
There are also the additional costs as parents or carers may have to take time off work, and may have to pay for parking, meals and drinks as well. Children and young people can end up missing school, college and after-school events.
Carrying out infusions while children are at school or at home takes the pressure off parents and carers, and means that children and young people don’t need to miss out on school or social life.
How do the treatments work?
The nurses carry all the equipment they need to administer treatments safely. The nurses are all registered paediatric nurses and competent to manage all clinical situations. The shorter infusions can be carried out at home, or in a quiet classroom or school nurse’s office. The child or young person wears an infusion pump in a backpack for the duration of the treatment, and this means that they can move around the house, or go to lessons, lunch or the playground.
When treatments are at school, parents and carers can sit in on the venous access procedure, when the needle is inserted and the infusion is set up. They can also hand over the child’s drugs to the nurse at school if they prefer.
Some children and young people enjoy the attention at school – one patient introduces his nurse to his friends – but others don’t want to be seen as different. For these, we can arrange treatment at home around their school times. We can also arrange treatments before and after holidays, allowing families to go away together without having to arrange local care while they are away.
One parent told me that the home-based treatment after school had changed their lives. Overall, home- and school-based care gives everyone in the family more flexibility.
We also train the parents and carers, and the children and young people themselves (once they are old enough to understand), to carry out the infusions if they want to be more independent and have the freedom to step away from specific timeslots.
Every six months we carry out a review with the independent patients to make sure that everything is going well, and I ring them regularly to offer extra support, or even respite infusions when they want to go back to just being a parent or carer for a while.
As the patients get older, we start a conversation about the transition to the adult service, which happens between the ages of 16 and 18. Because Pharmaxo Healthcare also runs an adult service, they can stay with us, and they might even see some familiar faces as some of our paediatric team also support adult patients.
Were there any challenges to setting up the service?
I was the first paediatric nurse to work at Pharmaxo Healthcare, so I had to educate our staff on the needs of paediatric patients – an example is an ‘All About Me’ booklet that includes information about the child or young person and their preferences, such as wanting treatment on a certain day, or needing a particular teddy bear. We are also creating information packs and frequently asked questions for parents and older patients.
The biggest challenge was recruiting paediatric nurses. We needed trained paediatric nurses, as adult nurses are not trained to administer drugs to children, and they needed to be able to access ports and insert cannulas to administer the treatments. We now have a team of four paediatric nurses, and we are looking to expand the team.
Was there anything you didn’t expect?
I didn’t expect the service to grow this quickly! It’s been fantastic to get the service off the ground at last. We have an excellent relationship with the clinical team at BCH and this has been pivotal to the growth of the service.
What do the nurses feel about being part of the service?
They enjoy the opportunity to get to know the patients and their families. We rotate the teams every few weeks to make sure that all faces remain familiar. This makes it easier when nurses take their well-deserved holidays, or when new nurses join the team. They also enjoy the challenge of doing something different.
And how about you?
I love what I do. We’ve had some great feedback from Birmingham Children’s Hospital and from NHS England. I can’t wait to expand the service, when we start to work with patients from Manchester Children’s Hospital and Great Ormond Street Hospital in London. I’m also looking forward to developing the services we provide to paediatric patients.