Changing medications can feel like a big step, especially when you’ve followed the same routine for years. For families affected by cystinosis, the move from Cystagon to Procysbi can bring excitement, questions, and sometimes a lot of nerves and uncertainty too.
Every person’s experience will be different, and medication plans should always be discussed with your clinical team. This story shares one family’s experience of their teenager’s transition and is intended as a personal account rather than medical advice.
Taking the First Steps
A 16 year old switched to Procysbi in March. Although it was approved in Scotland several months earlier, in November, it took some time before the medication reached them through the usual prescribing and dispensing process.
To begin with, the family took a gradual approach. For the first week, they continued with Cystagon during the day while switching only the night-time dose to Procysbi.
“We started by just changing the night time dose for one week. This was so that we could test if it was going to be tolerable without disturbing daily activities.”
When that went smoothly, they moved onto a full Procysbi regimen.
Finding a Routine That Works
One of the biggest adjustments was working around the timing requirements for food. Like many families managing cystinosis medications, there was a period of trial and error while working out what fitted best into everyday life.
“We had to experiment with timings due to the food and eating time restrictions that are noted when prescribed.”
After trying different approaches, they settled on a 10am and 10pm routine. The family also had to consider timing around other medications, which added another layer of planning. But as many parents of children with cystinosis know, medication schedules often involve some careful juggling.
“As with all the other rules of medicines, there’s always a way to work it out.”
How Was Tolerability?
One of the main concerns before switching was whether Procysbi would cause additional nausea or sickness. For this teen, that simply wasn’t the case – and we know that’s not the case for everyone.
“Tolerability-wise it was totally fine. No extra nausea or sickness that we were worried about.”
The family’s first white cell cystine (WCC) result after switching came back at 0.4, which was very encouraging.
“We were really chuffed with that first result.”
The Gift of a Full Night’s Sleep
Ask many cystinosis families what they hope for most when considering Procysbi, and sleep often features high on the list. For this family, removing the need for a middle-of-the-night alarm has been transformative.
“So, so worth it for the sleep. I said to my partner last night when going to bed at 10pm, ‘Don’t let me forget how grateful I am not to be setting an alarm for 2am.'”
Sometimes it is the simple things that have the biggest impact on family life.
Growing Independence
The timing of the switch has also helped as this young person becomes more independent. Shortly after changing medications, they were preparing to head away on a week-long trip with their Guiding group. For the parents, there was huge reassurance in knowing that medication support would only be needed during waking hours.
“It feels so much better to just ask the leaders to cover off the medicine during the waking day.”
As teenagers take on new adventures, school trips and residential experiences, finding medication routines that fit more naturally into everyday life can make a real difference.
A Practical Benefit
One practical advantage families may appreciate is that Procysbi does not need to be kept refrigerated once it has been dispensed by the pharmacy. For many families, this can make day-to-day management and travelling a little easier.
Every Journey Is Different
While this family’s transition to Procysbi has been positive, every person with cystinosis is different. Experiences of side effects, timings, medication schedules and practical arrangements can vary significantly, particularly for individuals who receive medication via a G-tube or have other complex medication needs.
What this story does show is that, with support from clinical teams and some flexibility while finding the right routine, a transition can open up new possibilities for sleep, independence and everyday family life.
And sometimes, not having to set that 2am alarm can feel like a very big win indeed.