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Why Aspiration Is a Silent, Hidden Danger for Cystic Fibrosis Patients

Dr. Gwen A. Huitt is an infectious disease doctor at National Jewish Health with a special interest in mycobacteria, bronchiectasis, and cystic fibrosis. Here, she talks to us about the hidden dangers of a major medical issue she feels doesn’t receive the attention it needs in the CF community — aspiration.

Q: What is aspiration? What is silent aspiration?

A: Aspiration is defined as any liquid, substance, or foreign body that gains access (below the vocal cords) to the airways. Many times when we have an overt aspiration, a cough is triggered. Think, “something went down the wrong pipe.” This may occur when folks drink fluids too quickly, toss their head back to take pills, etc. A small amount of liquid trickles down the windpipe, irritating it and causing a cough. Additionally, overt aspiration may occur in some folks with neurologic disorders that impair the ability to swallow appropriately (think stroke, Parkinson’s disease, etc.).

Silent aspiration may also occur in many neuromuscular disorders as well in “normal” hosts. This is where my patient population lives for the most part. There are two distinct situations that may occur. The first would be that when we take a drink, some small amounts of liquid “pools” in a recess around the vocal cords and then little amounts can trickle over the vocal cords down into the airway, but it does not trigger a cough or any sensation that something has just gained access to the airway. The second scenario is when we silently or overtly reflux up liquids from the stomach or esophagus and they reach high enough in the esophagus that they then trickle into the airway.

Q: What contributes most to aspiration?

A: For our patient population, we believe that overdistending the stomach with too much liquid, bending forward or lying too flat on your back, stomach, or on your right side contributes to most of our silent reflux episodes.

Q: What are the dangers of aspiration for a CF patient?

A: The dangers of aspiration for CF or non-CF patients are that you are sending not only germs such as pseudomonas or non-tuberculosis mycobacteria (NTM) into the airway that contribute to infection, but also that digestive enzymes and acids cause significant inflammation in the airways. This situation worsens inflammation and infection in the vulnerable airway.

MORE: Three travel considerations if you have a lung disease

Q: What are telltale signs of aspiration damage in the lungs?

A: We know that aspiration can lead to bronchiectasis. Additionally, by looking at microbiology of the sputum, we may find many organisms that are predominantly only supposed to be found in the digestive tract. When we see certain organisms such as citrobacter or E. coli we know for sure that these organisms were translocated from the digestive tract to the airway via aspiration. In all likelihood, other organisms such as pseudomonas, NTM, and Klebsiella are also primarily acquired in the airway via this mechanism. Much more research needs to be done in this area though.

Q: What is something about aspiration you think people would be surprised to learn?

A: That so much of aspiration is silent and we currently don’t have any good test to assess for intermittent reflux that may lead to aspiration. Also, there is no medication that stops reflux (which then leads to aspiration). Medications such as PPI (i.e., Nexium) or H2 blocker (i.e., Zantac) medications suppress acid production, which certainly can help with heartburn or cough, but they do not stop the physical action of reflux.

Q: Should reflux medication be a last resort or is it enough of a danger that it should be used as soon as a patient begins exhibiting reflux/aspiration symptoms?

A: As I said earlier, we currently have no medication to stop the action of reflux. In many ways, taking these medications may actually make reflux worse because you don’t feel heartburn symptoms but most certainly are still refluxing. Also, part of what PPIs and H2 blockers do is lower acid. Part of the action of acid in digestive juices is to kill some proportion of germs that we swallow. If you are still refluxing (while taking PPIs) and you then aspirate some of this digestive “soup,” you are actually aspirating more germs per aliquot of gastric contents. [But] you should definitely take a medication to help with heartburn symptoms or if you have been seen by a [gastro doctor] and they have diagnosed ulcer disease or Barrett’s esophagus.

Q: Do you believe aspiration is taken as seriously in the CF health care setting as it should be?

A: No, I do not think that aspiration is taken seriously at all in the CF community. Nor is it taken as seriously in the non-CF world.

Original article found at: https://cysticfibrosisnewstoday.com/2017/12/14/aspiration-risks/?utm_source=Cystic+Fibrosis&utm_campaign=a772c5a83f-RSS_THURSDAY_EMAIL_CAMPAIGN&utm_medium=email&utm_term=0_b075749015-a772c5a83f-71418393

Congratulations to Our Scholarship Winners!

The US Adult CF Association (USACFA) is excited to announce our recipients of the Lauren Melissa Kelly Scholarship for the Spring of 2018.

In our evaluation, we look for students who demonstrate tremendous academic achievement, community involvement and a powerful understanding of how having CF matched with these achievements places them in a unique situation to gain leadership roles within the community. Our scholarship is open to all pursuing any degree, from associates to Ph.Ds. We believe that any higher education is a strong foundation for advocacy and involvement in CF.

We are pleased to announce Hannah Buck and Mary Grace Bernard as the recipients of this semesters’ scholarship. Congratulations to them! They will be awarded $2500 each.

Both of our recipients demonstrated the leadership, intelligence, and drive of Lauren Melissa Kelly. We at USACFA look forward to seeing them further develop their leadership and advocacy in the cystic fibrosis community.

We are also pleased to award runner-up scholarships in the amount of $250 to five deserving applicants: Elizabeth Shea, Chloe Creager, Rebekah Weigner, Holly Beasley and Christopher Davis, Jr.

We are excited to announce more scholarship opportunities coming soon! Please stay tuned for more information. For questions, please contact us at scholarship@usacfa.org.

Tips for Surviving a Holiday Admission

Guest Blog By: Lauren Jones Hunsaker

Most of us have had to suffer through a holiday admission at one point.  It’s a reality of our disease, which, for most of us, never made a habit of consulting our social calendars before burdening us with an exacerbation.  I’ve spent several Christmases, birthdays, wedding anniversaries and many-a-Thanksgiving admitted.  As far back as middle school, most of my holidays were spent in the hospital simply because it was an advantageous time for an admission.

It’s not an easy thing to endure for kids or adults.  On a good day, being in the hospital is boring, uncomfortable and tedious.  The holidays magnify our misery by adding self-pity and a serious case of F.O.M.O. to the litany of grievances.  However, there are a few easy things you can do to weather the emotional storm of a holiday admission:

  1. Make a To Do List. While a day of nothing but channel-changing can seem never ending, a daily to-do list can help compartmentalize your day and alleviate the monotony of an admission.  Reading, exercising, playing games or journaling at specific times can be helpful distractions and keep you from staring wistfully out of your window.
  2. Enjoy Holiday T.V. Ordinarily, I don’t watch a lot of television.  But during the holidays there are so many classic movies, parades and specials on, allow yourself to indulge in the holiday deluge.  If nothing else, it helps pass the time and connects you to the outside world (“Hey, did you catch It’s a Wonderful Life for the sixteenth time on NBC?”  “I sure did!” “I don’t really understand why ‘Hee Haw!’ is funny.” “Me either, but Clarence is my favorite.”).
  3. Schedule Holiday Events Post-Discharge. The holidays are the holidays because of family.  The pilgrims will not cast a pox on your house if you host Thanksgiving the following weekend so ask family members if they are willing to attend a second family gathering after your discharge.  This gives you something to look forward to and helps temper loneliness while your family memorializes turkey carving on Facebook Live.
  4. Try a Little Empathy. No one wants to be in a hospital on Christmas, including doctors, nurses and nurse’s assistants.  I know what you’re thinking – “they’re getting paid to be there.”    And so as to preempt your next argument, yes, some are getting paid a higher wage for working on a holiday.  But remember that many don’t have a choice in their work schedules, just as we don’t have a choice as to when we’re admitted.  Staff are away from their families and missing holiday gatherings so they can take care of patients.  Take a moment to tell a favorite nurse thank you for working on the holiday.  Sometimes making someone else feel better makes you feel better too.
  5. Order in a Special Meal. Diet restrictions permitting, indulge in a holiday craving (i.e., turkey with stuffing, Chinese takeout, multiple desserts).  Your doctors will love that you’re packing in some extra calories and it’ll give you a break from repetitious hospital food.
  6. Take Advantage of Tech. Twenty years ago, when we would emerge from an admission (shielding our eyes from the blazing sun, unaccustomed to human life), we had no idea what had occurred during our fourteen days of solitude.  Gone are the days.  With social media and constant connectivity, we rarely experience the same isolation we once did.  Use today’s technology to cyber-attend family events from afar—FaceTime into family dinner or Skype into religious celebrations.  Social media can sometimes (and ironically) exacerbate loneliness, but use its advantages to stay connected during the holidays.  Feeling included can boost morale and help you power through an admission.

New Promising Results from Phase 3 of Combination Therapy

Findings from a phase 3 trial evaluating the efficacy and safety of tezacaftor in combination with ivacaftor in patients with cystic fibrosis (CF) who were homozygous for the Phe508del mutation were published in the New England Journal of Medicine.

The Phe508del mutation has been known to result in greatly reduced conductance regulator (CFTR) protein activity and a loss of chloride secretion, which can lead to impaction of mucus in the airways, gastrointestinal tract, and exocrine organs, with the potential for severe clinical consequences including gradual loss of lung function, nutritional deficits, pulmonary exacerbations, and respiratory failure. It is the most prevalent CFTR mutation worldwide, and affects approximately 46% of American CF patients.

Previous data has shown Ivacaftor’s association with a rate of progressive decline in lung function that is lower than that in untreated patients. In a phase 2 clinical trial involving patients who were homozygous for the Phe508del mutation or heterozygous for the Phe508del and G551D mutations, when combined with the investigational CFTR corrector tezacaftor, it has exhibited enhanced CFTR function and improved lung function.

In August, just one month removed from Vertex’s announcement of positive datafrom Phase 1 and Phase 2 studies, Rare Disease Report covered the acceptance of applications for the use of the tezacaftor/ivacaftor combination treatment in this patient population by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).

The phase 3 trial enrolled a total of 510 patients 12 years and older with CF who were homozygous for the Phe508del CFTR mutation at 91 sites in the U.S., Canada, and Europe from January 30, 2015 to January 20, 2017. Patients were randomly assigned to be administered either tezacaftor and ivacaftor (administered as a fixed-dose combination tablet containing 100 mg of tezacaftor and 150 mg of ivacaftor in the morning and a tablet containing 150 mg of ivacaftor in the evening) combination therapy or placebo for 24 weeks.

In total, 475 patients completed the full 24 weeks of the trial, with 93.6% (n=235) in the tezacaftor-ivacaftor group and 93% (n=240) in the placebo group. While no significant difference in the body mass index (BMI) was experienced between the groups at week 24, the use of the combination therapy led to a significantly greater absolute change from baseline in the predicted forced expiratory volume in 1 second (FEV1) than placebo. Despite advances in standard-of-care therapy, patients with CF continue to lose lung function at a rate of an estimated 1% to 3% per year. This trial exhibited a significant effect of the combination therapy compared to the placebo, as the mean absolute change from baseline in FEV1 through week 24 was 3.4 percentage points in the former, compared to 0.6 in the latter.

The most common adverse events (AEs) among the enrolled patients included infective pulmonary exacerbation, cough, headache, nasopharyngitis, increased sputum production, pyrecia, hemoptysis, oropharyngeal pain, and fatigue. The incidence of AEs was similar in both the group for combination therapy and the placebo group, however, those treated with lumacaftor-ivacaftor in the phase 3 did not experience an increased incidence of respiratory events (33 patients [13.1%] vs. 41 patients [15.9%]).

This improved safety profile of the tezacaftor-ivacaftor combination supports its use in a broad range of patients with CF, and, if approved, the therapy will be the third of Vertex’s drugs approved for CF patients, and the second intended specifically to treat patients with F508del mutations (Orkami [lumacaftor/ivacaftor]).

For original article please visit: http://www.raredr.com/news/phase-3-combination-therapy-cystic-fibrosis?t=physicians

For the published study please visit: http://www.nejm.org/doi/full/10.1056/NEJMoa1709846?query=genetics#t=articleDiscussion

Apply for the chance of a $10,000 scholarship!

The Sacks for CF Scholarship is related to quarterback sacks made during the NFL season. The undergraduate and graduate award is made annually to 30 people who strive for therapy adherence and academic success.

Continue reading Apply for the chance of a $10,000 scholarship!

Study Links PPI Use to Treat Gastroesophageal Reflux with More Frequent Hospitalizations

Doctors should frequently re-evaluate the use of protein pump inhibitors (PPIs) for cystic fibrosis (CF) patients, urges a University of Florida study which warns that long-term PPI use leads to a higher risk of hospitalization for pulmonary exacerbations.

Identifying risk factors associated with pulmonary exacerbations is critical since they cause a decline in pulmonary function and survival rates among CF patients.

PPI use, in particular, is believed to cause community-acquired pneumonia (CAP). Even though most CF patients use PPIs to control gastroesophageal reflux (GER), scientists still don’t fully understand the link between PPIs and pulmonary exacerbations in CF.

In the study, “Proton Pump Inhibitor Use Is Associated With an Increased Frequency of Hospitalization in Patients With Cystic Fibrosis,” which appeared in the journal Gastroenterology Research, researchers investigated that link and the risks it entails.

The study involved 114 adults who had been seen at UF’s Adult Cystic Fibrosis Center in Gainesville, Florida, between January and December 2016. Researchers collected data on PPI use and hospitalization during a one-year follow-up.

Results showed that 59 of the 114 patients (51.7 percent) used PPI for six or more months, and that exactly the same proportion (51.7 percent) had been hospitalized at least once during the one-year follow-up period. Among those who were hospitalized, PPI use was closely linked with the number of hospitalizations for pulmonary exacerbation, though researchers observed no link between frequency of hospitalization and PPI dosage.

No significant difference was found in GER between hospitalized and non-hospitalized patients.

The UF study is limited, in that it’s retrospective and therefore doesn’t establish a cause-effect relationship between PPIs and pulmonary exacerbation. Researchers say there’s still a possibility that GER itself — rather than the subsequent use of PPIs — causes increased pulmonary exacerbations. Yet they point out that the prevalence of GER was similar among hospitalized and non-hospitalized patients, supporting a causative link between PPI and pulmonary exacerbations.

Based on their findings, the UF team suggests that “prescribers of PPI therapy should exercise pharmacovigilance; frequently re-evaluating indications and appropriateness of therapy and in the setting of GER considering alternate management modalities such as anti-reflux surgery where appropriate.”

For original article please visit: https://cysticfibrosisnewstoday.com/2017/12/07/proton-pump-inhibitor-use-is-associated-with-an-increased-frequency-of-hospitalization-in-patients-with-cystic-fibrosis/

Deadline to Enroll in a 2018 Marketplace Plan Ends Friday December 15th!!

The deadline to enroll in a 2018 Marketplace health insurance plan is Friday, December 15, 2017.  A person can visit www.HealthCare.gov to create an account and review options for coverage.  Then the person can apply for coverage for  2018.  All plans on the government Marketplace offer essential benefits, such as emergency room visits and prescription drug coverage, as well as preventive care including shots and screenings.  The plans offered are run by private insurance companies.  Many states have a variety of plans available.
Some people in the CF community think that the Affordable Care Act has been repealed.  This NOT true.  The Affordable Care Act is still the law of the land.
This means that some people with CF may be eligible for premium help when purchasing a policy on the Marketplace.  The household income cut off is much higher than the cut off for Medicaid which means many people with CF may be eligible for help paying their premiums if their plan is purchased through the Marketplace.
 Some states run their own Marketplaces.  When a person goes to www.Healthcare.gov the site will link them to their state marketplace if the state has their own marketplace.
In some cases the premium help that is available is higher than the help that has been available in prior years.
The only way to enroll in a health plan through the Marketplace after December 15, 2017 is if you qualify for a Special Enrollment Period.  A person qualifies for a Special Enrollment Period if the person has certain life events occur such as losing health coverage, moving, having a change in income, getting married or having a change in family size.
If a person with CF is without health insurance coverage or wants to see if a better more affordable option is available then he or she should go to www.Healthcare.gov to see what is available before time runs out.

Trial to Possibly Treat Nonesense Mutations Begins

Sevion Therapeutics and Eloxx Pharmaceuticals announced that a first healthy subject has been dosed in a Phase 1b clinical trial assessing the safety, tolerability and drug properties of ELX-02 as a potential treatment of several genetic diseases caused by nonsense mutations, including cystic fibrosis (CF).

Continue reading Trial to Possibly Treat Nonesense Mutations Begins

Only I Can Look Into My Mirror

Guest Blog By: Brennen Reeves

If I were to count all of the scars on my body, the ones visible to a bystander at the beach, I’d need your fingers and toes, plus mine. And someone to mark the tallies. Thirty-eight. I’ve counted. From my chest, to my stomach, up to my neck, and down to my ankles the grooves are endless it seems. I don’t think I have any on my back but then again I cannot see my back and I still have trouble with sensation from the bilateral lung transplant I received in 2011, so I wouldn’t be able to feel them either. Continue reading Only I Can Look Into My Mirror