Re-evaluating GI Medication Regimens in People With Cystic FibrosisRe-evaluating GI Medication Regimens in People With Cystic Fibrosis https://pediatricsnationwide.org/wp-content/uploads/2023/02/AdobeStock_63552580-1024x681.png 1024 681 Emily Siebenmorgen Emily Siebenmorgen https://pediatricsnationwide.org/wp-content/uploads/2023/05/Emily.Siebenmorgen-scaled-e1684876333147.jpg
- February 28, 2023
- Emily Siebenmorgen
After widespread adoption of highly effective modulator therapy (HEMT), a new study evaluated the GI medication regimens of people with cystic fibrosis (CF). The study, published in Pediatric Pulmonolgy, found patient-initiated decreases in dosing and withdrawal from common GI medications is already occurring, yet there is no evidence to support this practice.
Highly effective modulator therapy consists of either ivacaftor (Kalydeco) or Trikafta (elexacaftor/tezcaftor/ivacaftor) to address folding defects of the cystic fibrosis transmembrane receptor protein in people with CF.
“HEMT is now the primary therapy for around 85% of CF patients whose genotypes are eligible for these medications,” says A. Jay Freeman, MD, MS, medical director of Pancreas Care at Nationwide Children’s Hospital. “The drugs are known to improve lung function, improve body mass index and significantly advance the care for patients with CF.”
HEMT has become an effective addition to many patients’ medication regimens, but it’s not a one-stop-shop treatment for CF.
“What everyone in the field is looking at is: How does care for a patient with CF change now that they’ve got this highly effective therapy? Now we want to know if these patients are going to be able to get off of other medications in their regimen, such as GI medications and breathing treatments,” Dr. Freeman says.
Recent literature took a standardized approach to examining the removal of breathing treatments for CF patients using HEMT. Dr. Freeman explains their group’s study aims to show the CF community’s interest in re-evaluating its approach to GI mediations, as well – namely, acid suppressive medications (ASM) and pancreatic enzyme therapy (PERT).
“For pancreatic enzymes, the obstacle for patients with CF is the pill burden,” Dr. Freeman says. “Some of these patients are taking 20 pills or more a day. If they’re eating three meals and two snacks a day, that means they’re taking pills for enzymes five times a day – and some teenagers eat way more than that.”
And PERT is often just one part of a CF patient’s pill regimen. People with CF are often on several GI medications, such as ASM from the start of their CF diagnosis.
“From an acid suppression standpoint, a lot of patients don’t even know why they’re on the medicine,” Dr. Freeman says. “It’s just something that’s been prescribed at some point by somebody – maybe because it improves acid suppression or maybe it improves how you absorb your enzymes.”
Dr. Freeman says they were surprised to find that not only were a huge number of patients on acid suppression medications, but nearly 80% had been on them had been on them for five years or longer.
“When we use those medications in GI, it’s usually a short-term thing,” Dr. Freeman says. “ASM is being prescribed for people with CF in a way that’s not normal for a GI doctor, and there’s no approach to get these patients off these medications.”
In addition, the study found that patients’ symptoms generally didn’t improve after they decided to stop taking their acid suppression medications themselves.
“Over half of our participants said their symptoms were the same, better, or indistinguishable after taking themselves off of acid suppression medications,” Dr. Freeman says. “This finding is a lightning rod for re-evaluating if these patients really need to be on these medications. Many of us in the GI field find ourselves taking our patients off of these medications over time, but I don’t think that’s necessarily what happens across the board in the CF world.”
Dr. Freeman explains patients self-regulating their other medications after success on HEMT is probably fine in some circumstances – particularly with ASM. The concern, he says, is for patients who decide to stop their PERT.
“We’re finding some patients report feeling better after they stop taking their enzymes,” Dr. Freeman says, “but you see their weight isn’t so good, and their fat-soluble vitamins are dropping. They’re treating constipation with malabsorption. This can make their stool look normal, even if they haven’t hit the right balance.”
Overall, Dr. Freeman explains this study highlights the importance of reviewing a patient’s medications, and to not continue medications indefinitely without a good reason.
“Every now and then you need to ‘test the system,’ if you will, and stop or decrease a medication to see if it actually has the benefit that you think it does. As treatment regimens change, providers have to adapt and change as well.”
Sathe M, Moshiree B, Aliaj E, Lee M, Hudson J, Gifford A, Attel S, Gamel B, Freedman, Schwarzenberg SJ, Freeman AJ. Need to study simplification of gastrointestinal medication regimen in cystic fibrosis in the era of highly effective modulators. Pediatric Pulmonology. 30 Nov. 2022;1-8. https://doi.org/10.1002/ppul.26257
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