Cystic fibrosis (CF) is a disease that makes it hard for the body to digest food and absorb nutrients. A new combination medicine called elexacaftor/tezacaftor/ivacaftor helps fix the underlying problem in CF. Researchers looked at what happened to people’s nutrition and weight over two years after they started taking this medicine. The study found that people gained weight, had better nutrition levels, and felt healthier overall. This is exciting because weight gain and good nutrition are really important for people with CF to stay strong and healthy.

The Quick Take

  • What they studied: Whether a new three-part medicine for cystic fibrosis helps people gain weight and get better nutrition over two years
  • Who participated: People with cystic fibrosis who started taking the medicine elexacaftor/tezacaftor/ivacaftor (specific number of participants not provided in available information)
  • Key finding: People taking this medicine showed improvements in weight gain and nutrition levels over the 24-month study period, suggesting the medicine helps the body absorb nutrients better
  • What it means for you: If you or a loved one has cystic fibrosis, this medicine may help you maintain a healthier weight and get better nutrition from food. However, results vary by person, and you should discuss this with your CF care team to see if it’s right for you.

The Research Details

This was a real-world study, which means researchers followed people with cystic fibrosis in their everyday lives as they took the new medicine, rather than in a controlled lab setting. They tracked what happened to these patients over 24 months (two years) by looking at their weight, nutrition levels, and overall health. Real-world studies are valuable because they show how medicines actually work when people use them at home, not just in perfect laboratory conditions.

The researchers collected information about participants’ nutrition status, body weight, and other health markers at different time points during the two-year period. This type of study design helps doctors understand the long-term benefits of new treatments and how they affect people’s daily lives.

Understanding how this new medicine affects nutrition is really important for people with CF. People with cystic fibrosis struggle to digest food properly because their pancreas doesn’t work well, making it hard to gain and maintain weight. A medicine that helps fix the root cause of CF could make a big difference in their health and quality of life. This real-world study shows what actually happens to real patients, which is more useful than lab studies alone.

This study tracked real patients over a long period (24 months), which is a strength because it shows lasting effects. However, the specific number of participants wasn’t provided in the available information, which makes it harder to judge how reliable the results are. Real-world studies are generally considered good quality because they reflect actual patient experiences, but they don’t always have the same strict controls as other types of research.

What the Results Show

The main finding is that people with cystic fibrosis who took this three-part medicine showed improvements in their nutrition and weight over the two-year study period. This is significant because people with CF typically struggle with weight gain and nutrition absorption due to how the disease affects their digestive system.

The medicine appears to work by fixing the faulty protein that causes cystic fibrosis, which allows the pancreas to work better and helps the body absorb nutrients from food more effectively. When the body can absorb nutrients better, people naturally gain weight and have better overall nutrition levels.

These improvements in nutrition and weight are important because they help people with CF stay stronger, have more energy, and potentially avoid some of the serious complications that can happen when CF patients don’t get enough nutrition.

Beyond weight and nutrition, the study likely tracked other health markers that improved alongside better nutrition. People with better nutrition often experience improvements in energy levels, immune function, and overall quality of life. The two-year timeframe allowed researchers to see if these benefits lasted over time, which is important for understanding whether this medicine provides long-term help.

Before this medicine was available, people with CF had to rely on pancreatic enzyme supplements and high-calorie diets to try to maintain nutrition and weight. This new medicine works differently—it actually fixes the underlying problem in the cells rather than just treating the symptoms. Previous research showed that when CF patients could absorb nutrients better, their health improved significantly, so these results fit with what scientists expected.

The study didn’t provide the specific number of people who participated, which makes it harder to know how reliable the results are. We don’t know if all types of CF patients benefited equally, or if some groups had better results than others. The study only lasted two years, so we don’t know if the benefits continue beyond that time. Additionally, real-world studies can’t control for all the different factors in people’s lives that might affect nutrition, like diet quality or other medicines they’re taking.

The Bottom Line

If you have cystic fibrosis, talk to your CF care team about whether this medicine might help you. The evidence suggests it can improve nutrition and weight gain, which are important for your health. This medicine appears to be most helpful for people whose CF is caused by specific genetic mutations, so your doctor will need to test you to see if it’s right for you. Start with your CF specialist’s guidance on dosing and monitoring.

This research is most relevant for people with cystic fibrosis and their families. It’s especially important for CF patients who struggle with weight gain and nutrition absorption. Healthcare providers who treat CF patients should be aware of these benefits. People without CF don’t need to worry about this medicine, as it’s specifically designed for CF.

Based on this two-year study, improvements in weight and nutrition appear to develop gradually over months. You shouldn’t expect overnight changes, but over several months of taking the medicine, you should notice improvements in how you feel, your energy level, and your weight. The benefits seem to continue over the full two-year period studied, suggesting this is a long-term treatment.

Want to Apply This Research?

  • Track your weight weekly and record it in the app. Also track how you feel energy-wise (1-10 scale) and any changes in appetite or digestion. Take photos of meals to monitor nutrition intake over time.
  • Work with a CF nutritionist to set realistic weight gain goals. Use the app to log meals and snacks, especially high-calorie foods that help with nutrition. Set reminders to take your medicine at the same time each day to stay consistent.
  • Create a monthly check-in in the app to review weight trends, energy levels, and nutrition markers. Share this data with your CF care team at regular appointments. Track any side effects or changes in digestion to discuss with your doctor.

This research describes outcomes for people with cystic fibrosis taking a specific medicine. This information is for educational purposes and should not replace medical advice from your healthcare provider. If you have cystic fibrosis or a family member does, consult with your CF care team before making any changes to treatment. Individual results may vary based on genetics, overall health, and other factors. Always work with your doctor to determine the best treatment plan for your specific situation.