Expert Guidance for Overcoming Common Challenges In Management of Cystic Fibrosis

Course Director

Stanley B. Fiel, MD, FACP, FCCP

Stanley B. Fiel, MD, FACP, FCCP
The Icahn School of Medicine at
Mount Sinai
Atlantic Health System
Morristown Medical Center
Morristown, New Jersey

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Part 1 of a 2-part series

Dr. Fiel provides expert feedback to the questions submitted by your peers during a recent survey on this topic.


Cystic fibrosis (CF) is a complex, chronic, multisystem disease for which there is currently no cure. Nonetheless, advances in management have led to dramatic improvements in patient survival. With this development, new issues have arisen for CF patients and their care providers, including an increased symptom burden and adherence problems due to complex treatment regimens. In this activity, Dr. Fiel addresses questions related to common patient care challenges in management of CF.


This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.
Additional support provided by Penn State College of Medicine and Answers in CME.

Course Director
Stanley B. Fiel, MD, FACP, FCCP, has a financial interest/relationship or affiliation in the form of:
Consultant for Bayer Corporation; Boehringer Ingelheim Pharmaceuticals, Inc.; Novartis Corporation; and Pfizer Inc.
Grant/Research Support from Cystic Fibrosis Foundation; Gilead; Novartis Corporation; and Vertex Pharmaceuticals Incorporated.
Speakers Bureau participant with Dey Pharma; Genentech, Inc.; Gilead; GlaxoSmithKline; Novartis Corporation; and Pfizer Inc.
Medical Director
Kadrin Wilfong, MD
Answers in CME, Inc.
Kadrin Wilfong, MD, has no financial interests/relationships or affiliations in relation to this activity.

Answers in CME staff who may potentially review content for this activity have disclosed no relevant financial relationships.

Penn State College of Medicine staff and faculty involved in the development and review of this activity have disclosed no relevant financial relationships.

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How can we help young patients with CF better manage the burden of administering the various medications in the context of a busy life and demanding school/work schedules?

Dr. Fiel: These questions are extremely important, and part of discussions that we as a care team have with our patients when they come for their visits. We realize that adherence is a major problem in CF care because of the complexity of all the treatment regimens, and it probably takes anywhere from close to an hour to two-plus hours to really do all of their appropriate treatments. And looking at the medical literature on adherence, [it has been reported] that patients adhere about 50% to maybe 60% at most in their care if you look at all of their treatment regimens.1

Narrator: Different factors impact adherence—ranging from patient’s age, mental health, quality of family relationships to access to care, work life, to peer support, among others. Furthermore, adherence is impacted by the fact that currently approved lung-directed therapies for CF are for long-term maintenance; thus, any perceived benefits do not become apparent immediately, but only after years of chronic use.

As key steps in managing adherence, clinicians should assess adherence during each visit based on pharmacy records and patient reports, make an effort to accurately identify patients who are nonadherent, and explore ways to make interventions more practical for patients, as well as to families and care teams. Education alone may not be enough, and multicomponent interventions have been found to be best. They may include education, reminders, self-monitoring, tailoring the care regimens to the needs of each patient, feedback about health and behavior, problem-solving, contingencies and rewards, direct observation of therapy administration, social support, cognitive behavioral therapy, motivational interviewing, and parent/family training and therapy.2,3 New technologies, including health apps, may offer additional opportunities for tracking and monitoring of treatments to help improve adherence.

Dr. Fiel: Basically, when our team meets with patients, we talk about negotiating care and trying to understand the obstacles for patients as to why they may or may not want to continue with their therapies. Cystic fibrosis is a disease where patients frequently don't feel remarkably better after taking their medications. It's not like asthma, where they will get a very significantly improvement in their air flow or their breathing, and these patients will frequently have cough and be congested most of the day, even with good pulmonary toilet. So we try to come up with approaches for them that would include exercise plans, keeping day planners to sort out which types of therapies work best, work around their school or jobs, try and have them discuss with their workplace or spouses or roommates ways to improve their adherence and maximize the efficiency of the kinds of things that they do. These discussions take place every time we meet with our patients, to see if there are different obstacles for them, so that they can have a very realistic goal in treatment.

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How do you decide which inhaled antibiotic to choose when a patient with CF is chronically infected with Pseudomonas aeruginosa? What do you do when the patient develops intolerance or resistance to the antibiotics?

Dr. Fiel: We have the two approved therapies at the moment: tobramycin4-7 and aztreonam.8-11 Since both are approved in individuals with moderate lung disease—approximately 25% to 75% FEV1 and >6 or 7 years of age—we use these agents when patients first colonize with P aeruginosa. As to which should be used first? Tobramycin has been around the longest, with aztreonam only being approved for the last few years. The choice is frequently left up to patients, since both drugs are effective and both have pros and cons. Tobramycin is used twice a day, aztreonam is used three times a day and is delivered by an ultrasonic nebulizer via the e-flow system that is delivered quickly, in just a couple of minutes, whereas tobramycin solution may take 15 to 20 minutes.12,13

Tobramycin inhalation powder, which was just recently approved by the FDA, is a portable, dry powder inhalation that shows noninferiority to the liquid form of tobramycin, and that can be delivered in just a couple of minutes. So that is a useful addition to our armamentarium in relation to adherence for these patients.14

Narrator: The tobramycin inhalation powder was studied in a phase 3 clinical program in which two trials evaluated its efficacy compared with placebo, and a third evaluated its safety compared with the tobramycin inhalation solution. This non-nebulized formulation is indicated for CF patients six years of age and older, and reduces the time required for treatment by about 70% compared with the aerosolized form—saving about 13 hours per treatment cycle.15 The 112 mg twice-daily dose is delivered by inhaling the contents of four capsules via a specific apparatus.16

It should be noted that the inhaled antibiotics are only indicated for treatment of P aeruginosa infections, which affect approximately 70% to 80% of CF patients with lung disease. For the reminder of the patients, antimicrobial maintenance therapies are lacking.

Dr. Fiel: There have been no established resistance patterns that make patients not respond to treatment. Basically, the resistance that we see, using the classic Kirby-Bauer microbiologic resistance patterns, don't hold for the aerosolization technique, since the levels in the airway are way above any of the resistance levels that we may see. And, in fact, when patients were looked at in the clinical trials, the level of resistance of Pseudomonas and the response to therapy did not correlate. So patients with high resistance patterns responded just as well as those with low resistance patterns.17,18

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What is the optimal way to sequence the different therapies for CF, including antibiotics, mucolytics, bronchodilators, and anti-inflammatory agents?

Dr Fiel: Usually, therapy always begins with bronchodilation; followed by mucolytics or hydration with hypertonic saline; and then finally an aerosolized antibiotic.

The aerosolized antibiotic should come after chest physiotherapy. There are some patients who feel that to save time, they may be able to substitute liquification with hypertonic saline and mucolytics. In fact, the two agents don't work exactly similarly and one should not necessarily replace the other.

The problem, of course, relates to patients’ time and what they can feasibly do in a given day, but the mucolytics such as rhDNase have a more specific action in removing the extracellular DNA and have data that show improvement in early changes in lung disease that has not been seen with hypertonic saline.

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  1. Riekert KA et al. 26th Annual North American Cystic Fibrosis Conference (NACFC 2012). Abstract 464.
  2. Haynes RB et al. Cochrane Database Syst Rev. 2008;(2):CD000011. doi: 10.1002/14651858.CD000011.pub3.
  3. Kahana S et al. J Pediatr Psychol. 2008;33:590-611.
  4. Ramsey BW et al. N Engl J Med. 1999;340:23-30.
  5. Moss RB. Chest. 2002;121:55-63.
  6. Burns JL et al. J Infect Dis. 1999;179:1190-1196.
  7. Bowman CM. J Cyst Fibros. 2002;1:194-198.
  8. McCoy KS et al. Am J Respir Crit Care Med. 2008;178:921-928.
  9. Retsch-Bogart GZ et al. Chest. 2009;135:1223-1232.
  10. Oermann CM et al. Pediatr Pulmonol. 2010;45:1121-1134.
  11. Wainwright CE et al. J Cyst Fibros. 2011;10:234-242.
  12. TOBI (tobramycin inhaled solution) prescribing information. Accessed January 30, 2014.
  13. CAYSTON (aztreonam for inhalation solution) prescribing information.
    Accessed January 30, 2014.
  14. TOBI PODHALER (tobramycin inhalation powder) prescribing information.
    Accessed January 30, 2014.
  15. Konstan MW et al. J Cyst Fibros. 2011;10:54-61.
  16. Parkins MD, Elborn JS. Expert Rev Respir Med. 2011;5:609-622.
  17. Saiman L et al. Clin Infect Dis. 1996;23:532-537.
  18. McCoy KS et al. Pediatr Pulmonol. 2008;31 Suppl:351.

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This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.
Additional support provided by Penn State College of Medicine and Answers in CME.

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