Researchers studied a new medicine called elexacaftor/tezacaftor/ivacaftor (ETI) in young children with cystic fibrosis (CF) to see if it could help their pancreases work better. The pancreas is an organ that helps digest food. In this study, about 28% of children whose pancreases weren’t working well at the start actually improved after taking the medicine for 6 months. However, some children’s pancreases stopped working well again by 12 months. The study also found that the medicine helped increase vitamin D levels. These findings suggest the medicine can help, but doctors need to watch children carefully and adjust their digestive enzyme medicines slowly.

The Quick Take

  • What they studied: Whether a new three-part medicine (ETI) could help young children with cystic fibrosis whose pancreases weren’t working properly, and whether their pancreases could start working better on their own.
  • Who participated: 51 children with cystic fibrosis between ages 2 and 6 years old from a large children’s hospital in the UK who started taking the new medicine. The children were tracked before starting the medicine and for up to 12 months after starting it.
  • Key finding: About 28% of children whose pancreases weren’t working well (12 out of 43 kids) showed improvement 6 months after starting the medicine. However, 3 of these children’s pancreases stopped working well again by 12 months, showing that improvement may not always last.
  • What it means for you: If your child has CF and their pancreas isn’t working well, this medicine may help their pancreas function improve. However, doctors need to monitor your child carefully because the improvement might not be permanent. Don’t stop or change digestive enzyme medicines without talking to your doctor first.

The Research Details

This study looked at real-world results from children taking a new CF medicine at a major children’s hospital in the UK. Researchers collected information about the children’s health before they started the medicine (looking back at old medical records) and then tracked them carefully after they started taking it (collecting new information going forward). They measured three main things: how well the pancreas was working (using a test called faecal elastase), vitamin levels in the blood, and sweat chloride levels (a marker of CF severity).

The study included 51 children out of 68 who were eligible. These children were between 2 and 6 years old when they started the medicine. The researchers looked at what happened at 6 months and 12 months after starting treatment. This type of study is valuable because it shows what actually happens in real hospitals with real patients, rather than just in controlled research settings.

Understanding how this medicine works in real-world situations is important because clinical trials (controlled research studies) don’t always show exactly what happens when doctors use medicines in everyday practice. This study helps doctors know what to expect and how to monitor children properly. It also helps families understand whether their child’s pancreas function might improve and what signs to watch for.

This study has several strengths: it tracked children over time at a specialized CF center with experienced doctors, it included a reasonable number of children (51), and it collected both old and new medical information. However, the study is limited because it only looked at children from one hospital in the UK, so results might be different in other places. The study also didn’t have a comparison group of children not taking the medicine, so we can’t be completely sure the medicine caused the improvements. Some children’s information was collected from old records, which might not be as detailed as newly collected information.

What the Results Show

The main finding was that some children’s pancreases started working better after taking the new medicine. Specifically, 12 out of 43 children whose pancreases weren’t working well (28%) showed improvement 6 months after starting the medicine. This is important because CF often damages the pancreas, and when it stops working, children need to take digestive enzymes with every meal.

However, the improvement wasn’t permanent for everyone. By 12 months, 3 of the 12 children who improved had their pancreases stop working well again. One additional child whose pancreas wasn’t working well at 6 months actually improved by 12 months. This means the situation is complicated—some children improve, some stay improved, and some go back to having pancreases that don’t work well.

Because some children’s pancreases started working better, their doctors were able to reduce or stop their digestive enzyme medicines. This is good news because it means fewer pills to take with meals. However, doctors had to be very careful about making these changes and continued to monitor the children closely.

The study also looked at vitamin levels because children with CF often have trouble absorbing vitamins. The good news was that vitamin D levels increased significantly after starting the medicine. This is helpful because vitamin D is important for bone health and immune function. However, vitamin A and vitamin E levels didn’t change much, and the researchers didn’t see the high vitamin A levels that were seen in older children taking the same medicine. This suggests that younger children might respond differently to the medicine than older children.

Earlier research showed that this medicine helped improve pancreas function in older children with CF (ages 5-15). This new study confirms that the medicine can also help younger children (ages 2-6), which is important because the medicine was only recently approved for use in children as young as 2 years old. The study also found a connection between how much the pancreas improved and how much the sweat chloride decreased—when sweat chloride went down more, the pancreas tended to improve more. This supports what doctors already believed about how the medicine works.

This study has several important limitations to keep in mind. First, it only included children from one hospital in the UK, so the results might be different in other countries or hospitals. Second, there was no comparison group of children not taking the medicine, so we can’t be 100% certain the medicine caused the improvements (though it’s very likely). Third, some of the information came from old medical records that might not be as complete as information collected specifically for research. Fourth, the study was relatively small (51 children), so the results might change if a larger study was done. Finally, the study only followed children for up to 12 months, so we don’t know what happens over longer periods.

The Bottom Line

If your child has CF and their pancreas isn’t working well, talk to your CF doctor about whether this medicine might help. The evidence suggests it may improve pancreas function in some young children (moderate confidence level). If your child starts taking this medicine and their pancreas improves, work with your doctor to slowly adjust digestive enzyme medicines rather than stopping them suddenly. Continue regular monitoring of your child’s pancreas function and symptoms, even if it seems to be working well, because improvement may not always last (high confidence level).

This research is most relevant for families with young children (ages 2-6) who have CF and pancreatic insufficiency (when the pancreas doesn’t work well). It’s also important for CF doctors and specialists who treat young children. Parents of older children with CF might find it interesting to understand how the medicine works in younger age groups. This research is less relevant for people with CF who don’t have pancreatic problems or for adults with CF, though some principles might apply.

If this medicine helps your child, you might see improvements in pancreas function within 6 months. However, it’s important to understand that improvement may not happen for everyone, and even if it does happen, it might not last. Some children’s pancreases stop working well again within a year. Therefore, you should expect to continue monitoring your child’s health regularly for at least 12 months and beyond, rather than expecting a quick fix.

Want to Apply This Research?

  • Track your child’s digestive symptoms weekly (such as greasy stools, stomach pain, or bloating) and note any changes in enzyme doses. Record these observations in a simple log with dates to share with your CF doctor at appointments.
  • Work with your CF care team to create a medication adjustment plan if pancreas function improves. Use the app to set reminders for enzyme doses and to track when doses change, helping you remember the current correct dose at each meal.
  • Set up monthly check-ins in the app to review your child’s symptoms and enzyme use patterns. Create alerts for scheduled lab tests (faecal elastase and vitamin levels) to ensure you don’t miss important monitoring appointments that help track pancreas function over time.

This research describes what happened in one hospital with a specific group of children. Results may vary for individual children based on their age, CF type, and overall health. This information is not a substitute for medical advice from your child’s CF care team. Do not make any changes to your child’s medicines, enzymes, or treatment plan without first talking to your CF doctor. Some children’s pancreases improved on this medicine while others did not, and some improvements were not permanent. Your child’s response may be different. Always work with your CF specialist to monitor your child’s health and adjust treatment as needed.