How Cystic Fibrosis Is Treated

Promising new therapies offer hope

While there is no cure for cystic fibrosis (CF), advances in treatment have extended both the life expectancy and quality of life of people living with the disease. Treatment may involve a variety of procedures and medications, including airway clearance techniques, antibiotics, a high-calorie diet, mucus thinners, bronchodilators, pancreatic enzymes, and newer generation drugs known as CFTR modulators.

Severe cases may require a lung transplant.

The cornerstones of a treatment plan include the prevention of respiratory infections, the retention of lung function, and the use of dietary aids to compensate for the malabsorption of nutrients in the intestines. With time, adjustments will undoubtedly be needed.

In the 1980s, people with CF had a median life expectancy of fewer than 20 years. Thanks to newborn screening and advances in the treatment, those living with the disease can expect to live well into their 40s—and perhaps even longer—if treatment is started early and managed consistently.

Self-Care and Lifestyle

While much excitement been centered around the introduction of newer cystic fibrosis drugs, self-care still remains the foundation of CF treatment. This involves airway clearance techniques to remove mucus from the lungs, exercise to maintain lung capacity and strength, and dietary interventions to improve the absorption of fats and nutrients.

Airway Clearance Techniques

Airway clearance techniques (ACTs) commonly used by people with chronic obstructive pulmonary disease (COPD) are just as effective in treating CF lung disease. The techniques aim to dislodge mucus from the lung's air sacs so that you can cough it out. These may be performed several times daily depending on the severity of your condition.

There are a number of commonly used techniques, some of which may be easier for adults than young children:

  • Huff coughing can perform on your own. As opposed to active coughing, which can wear you out, huff coughing involves deep, controlled inhalations so that enough air can get behind the mucus in your lungs to dislodge it. By doing so, you don’t have to exert as much energy to expel it. You inhale deeply, hold your breath, and exhale forcefully to dislodge the mucus.
  • Chest percussion, also known as postural percussion and drainage, is performed with a partner who rhythmically claps your back and chest with cupped hands as you alter positions. Once the mucus is loosened, you can expel it with huff coughing.
  • Chest wall oscillation works similarly to postural drainage but employs a handheld, non-electrical device that vibrates and loosens mucus. Some of the devices can be connected to a nebulizer to combine oscillation with the delivery of inhaled medications.
  • High-frequency chest oscillation involves an inflatable vest attached to an air pulse generator. The machine mechanically vibrates the chest at high frequencies to loosen and release mucus.

Exercise

Exercise is something you can’t afford to avoid if you have CF.

Exercise not only helps maintain lung function, it reduces your risk of CF-related complications such as diabetes, heart disease, and osteoporosis.

Exercise programs need to be individualized based on your age and health status, and ideally designed with your physical therapist or medical care team. Fitness testing may be performed in advance to establish your baseline level of training.

Fitness plans should include stretching exercises (to promote flexibility), aerobic training (to improve endurance and cardiorespiratory health), and resistance training (to build strength and muscle mass). When first starting out, you might initially aim for five- to 10-minute sessions, performed three or more days per week, and gradually build to 20- to 30-minute sessions.

In terms of programs, there is no set “cystic fibrosis workout.” Instead, you and your physical therapist should find the activities (including biking, swimming, walking, or yoga) and exercises (such as resistance bands, weight training, or cross-training) that you can sustain over the long term with the aim of increasing the intensity and duration of your workouts as you get stronger.

A set program for a child is usually not necessary, given how active kids tend to be naturally. That said, if your child has CF, it's wise to speak with your pulmonologist to get a better sense of your child's limitations, which activities may be better than others, and what precautions may need to be taken to prevent infection from other kids and even shared sports equipment.

Diet

Cystic fibrosis affects digestion by clogging the ducts in the pancreas that produce digestive enzymes. Without these enzymes, the intestines are less able to break down and absorb nutrients from food. Coughing and fighting infection can also take their toll, burning calories and leaving you drained and fatigued.

To compensate for this loss and maintain a healthy weight, you need to embark on a high-fat, high-calorie diet. By doing so, you will have the energy reserves to better fight infection and stay healthy.

A doctor will determine what you or your child’s weight should be. The clinical measures may include:

  • Weight-for-length for children under 2
  • Body mass index (BMI) percentiles for people ages 2 to 20 (as height can fluctuate significantly during this period)
  • Numerical BMI for people over 20

Based on that, your age, fitness level, and overall health, a specialist dietitian can help design a diet with the right balance of protein, carbohydrates, and fats.

The Cystic Fibrosis Foundation recommends the following daily caloric intake for women, men, toddlers, children, and teenagers, depending on weight goals:

 Maintain WeightGain Weight
Women2,500 cal/day3,000 cal/day
Men3,000 cal/day3,700 cal/day
Toddlers 1 to 31,300 to 1,900 cal/daySpeak with a specialist
Children 4 to 62,000 to 2,800 cal/daySpeak with a specialist
Children 6 to 12200% of recommended daily caloric intake by ageSpeak with a specialist
Teens3,000 to 5,000 cal/daySpeak with a specialist

Over-the-Counter Remedies

Cystic fibrosis is associated with chronic inflammation due to increased stress placed on the lungs and pancreas by the accumulated mucus. The inflammation causes as much damage to the lungs as recurrent infection and can lead to impairment of the pancreas, kidneys, liver, and other vital organs as well.

Nonsteroidal anti-inflammatory drugs (NSAIDs), such as Advil (ibuprofen) and Aleve (naproxen), are commonly prescribed to reduce inflammation in people with CF. A review of studies from the  Montreal Children’s Hospital concluded that the daily use of Advil can significantly slow the progression of CF lung disease, especially in children. Side effects include nausea, stomach upset, vomiting, and gastric ulcers. Overuse can cause intestinal damage.

Other OTC medications may be used to support a high-calorie diet. To aid in the absorption of nutrients, your doctor may prescribe a pancreatic enzyme supplement. These come in capsule form and are swallowed whole after a meal or snack. While available over the counter, they need to be dose-adjusted by your doctor based on your weight and condition. Side effects include bloating, diarrhea, constipation, headaches, and cramps.

Pancreatic enzymes can also be prescribed for children, when appropriate. The capsule can be broken open, measured, and sprinkled on food if the dose needs to be adjusted or your child is unable to swallow pills.

Your doctor may also recommend vitamin or mineral supplements if blood tests reveal any significant deficiencies. Fat-soluble vitamin supplements, such as vitamins A, D, E, and K, which are essential for growth and fat absorption, are common.

Prescriptions

Prescription drug therapies are used to manage symptoms of the disease and slow the decline of organ damage. The medications can be broadly broken down into four classes:

  • Bronchodilators
  • Mucolytics
  • Antibiotics
  • CFTR Modulators

The drugs may be delivered either orally, by injection, intravenously (into a blood vein), or inhaled with a nebulizer, meter-dosed inhaler (MDI), or a dry powdered inhaler (DPI), depending on the medication.

Bronchodilators

Bronchodilators are drugs that relax constricted airway passages and allow more air into the lungs. They are most commonly delivered with an MDI, which includes an aerosolized canister and a mouthpiece called a spacer. Drugs options include albuterol and Xopenex (levalbuterol).

Bronchodilators are inhaled 15 to 30 minutes before starting airway clearance. They not only increase the amount of mucus you can cough up, they help you inhale other medications, such as mucolytics and antibiotics, deeper into the lungs.

Side effects include nausea, tremors, rapid heartbeat, nervousness, and dizziness.

Mucolytics

Mucolytics, also known as mucus thinners, are inhaled medications that thin the mucus in your lungs so that you can cough them up more easily. There are two types commonly used in CF therapy:

  • Hypertonic saline, a sterile salt solution, can be inhaled with a nebulizer after you’ve taken a bronchodilator. The salt content draws water from surrounding tissue and, by doing so, thins the mucus in the lungs.
  • Pulmozyme (dornase alfa) is a purified enzyme that both thins accumulated mucus and increases the viscosity (slipperiness) in the lungs. Side effects may include a sore throat, watery eyes, runny nose, dizziness, rash, and a temporary change or loss of voice.

Antibiotics

Antibiotics are drugs that kill bacteria. With cystic fibrosis, the accumulation of mucus in the lungs provides bacteria the perfect breeding ground for infection. Because of this, recurrent lungs infections are common in people. The more infections you have, the more damage your lungs will sustain.

Antibiotics can be used to treat acute CF symptoms (called exacerbation) or prescribed prophylactically to prevent infections from occurring. They are delivered either orally or with a nebulizer or DPI. Serious infections may require intravenous treatment.

Among the options:

  • Oral antibiotics can be used to treat milder chronic infections and exacerbations. Zithromax (azithromycin) is a broad-spectrum antibiotic commonly used for this. Serious bacterial infections may require a specific, targeted class of antibiotic drug.
  • Inhaled antibiotics are used prophylactically to prevent bacterial infection but can also be used during acute exacerbations. There are two antibiotics used for this: Cayston (aztreonam) and Tobi (tobramycin). Inhaled antibiotics are only used after you've used a bronchodilator and mucolytic and performed airway clearance.
  • Intravenous antibiotics are reserved for severe cases. The choice of antibiotic would be based on the type of bacterial infection you have. These may include penicillins, cephalosporins, sulphonamides, macrolides, or tetracyclines.

Irrespective of the type you are given, it is important to take the antibiotic drug as prescribed even if you no longer have symptoms. If you don’t and stop early, any bacteria remaining in your system can become resistant to the antibiotic, making it more difficult to treat if the infection returns.

CFTR Modifiers

The cystic fibrosis transmembrane receptor (CTFR) gene produces the CFTR protein, which regulates the movement of water and salt in and out of cells. If the CTFR gene is mutated, as is the case with this disease, the protein it produces will be flawed and cause mucus to thicken abnormally throughout the body.

In recent years, scientists have developed drugs, called CFTR modulator, able to improve CFTR function in people with specific mutations. There are more than 2,000 that can cause CF, and around 80 percent of cases are associated with a specific mutation known as the deltaF508. The drugs do not work for everyone and require you to undergo genetic testing to identify which CFTR mutations you have.

There are three CFTR modulators approved for use by the U.S. Food and Drug Administration (FDA): 

  • Kalydeco (ivacaftor) is a drug that binds to the defective CFTR protein and "holds the gate open" so that water and salt can flow in and out of cells. Kalydeco can be used in adults and children age 2 and over.
  • Orkambi (lumacaftor + ivacaftor) can only be used in people with two copies of the deltaF508 mutation. Having two deltaF508 copies causes the severe deformity of the protein. Orkambi works by correcting the shape of the protein and restoring its intracellular function. Orkambi can be used in adults and children six and over.
  • Symdeko (tezacaftor + ivacaftor) is also a corrector drug designed for people with two deltaF508 mutations. It is used in people who cannot tolerate Orkambi. It may also improve CFTR function associated with 26 other common CFTR mutations. Symdeko can be used in adults and children 12 and over.

The drugs are available in tablet form and taken every 12 hours. A powder formulation of Kalydeco, which can be sprinkled over food, is available for small children. Side effects include a headache, nausea, dizziness, fatigue, diarrhea, and sinus congestion. Cataracts have also been reported in children who use these drugs.

Other CFTR modifiers are currently in development, including two experimental drugs—known as VX-659 and VX-445—that are being studied in combination with Symdeko. Early results from phase 3 clinical trials have shown that using VX-659 or V-445 with Symdeko was superior to using Symdeko alone.

Supportive Therapies

During severe exacerbations or in cases of chronic disease, support measures may be needed to assist with breathing or nutrition. This may involve oxygen therapy and enteral nutrition.

Oxygen Therapy

Oxygen therapy involves the use of a portable oxygen tank with either a mask or nasal prongs to deliver concentrated oxygen to the lungs.

At present, there are no guidelines on the appropriate use of long-term oxygen therapy (LTOT) in people with CF and little evidence as to its benefit one way or the other. With that being said, the current body of research does suggest that oxygen therapy has its place in the short-term treatment of CF lung disease.

People with CF who have significant lung damage will invariably begin to experience hypoxemia (low blood oxygen saturation). It is a condition associated with poor sleep quality, reduced exercise tolerance, and the loss of muscle mass. Supplemental oxygen at night has been shown to improve sleep quality, while low-flow oxygen delivered during exercise can increase the duration and intensity of workouts.

Given the degenerative nature of cystic fibrosis, LTOT may become necessary if the loss of lung function is causing disability and a low quality of life.

Enteral Nutrition

Enteral feeding (tube feeding) involves the placement or surgical implantation of a feeding tube through which liquid food is delivered. You are taught how to perform the feedings at home, usually, with the same liquid supplements. It is meant to supplement eating, not replace it. 

Tube feeding is generally considered if you are losing weight despite eating a high-calorie diet, are unable to tolerate food, or are trying to gain weight prior to a lung transplant. 

For example, if you have a lung infection, the force required to breathe can burn far more energy than you are able to obtain from food. Even if you can eat, the impairment of the pancreas may stifle your ability to gain weight despite your best efforts.

Many are tentative about it when they first start, but most people (including children) learn to adapt. Parents of children with CF often say that tube feeding removes stress at mealtimes, increases their child's weight faster, and reduces concerns about the child's long-term health and development.

Enteral feeding can take several forms. Among them:

  • Nasogastric feeding is the least invasive form of enteral feeding in which an NG tube is placed in your nostril, down your throat, and into your stomach. The tube can be inserted each night and removed in the morning.
  • Gastrostomy is a more permanent option in which a G-tube is inserted into your stomach through an incision in your belly. This allows food to be delivered directly to the stomach. In some cases, the surgeon can place a button at skin level which allows you to open and close the tube when needed (and hides the tube beneath your shirt).
  • Jejunostomy is a procedure in which a J-tube is inserted through the abdomen to a part of the small intestine called the jejunum. This is most often used if you cannot tolerate feeding into the stomach.

Lung Transplantation

No matter how diligent you are with treatment, there will come a day when your lungs will be less able to cope. The damage incurred over a lifetime will take its toll, reducing not only your ability to breath but your very quality of life. At this point, your pulmonologist may recommend a lung transplant that could add years to your life.

Getting on the Waiting List

Getting a lung transplant requires an extensive evaluation to assess your health, your financial eligibility, and your ability to cope and maintain good health practices after undergoing the transplant. The process involves many tests that can take up to a week to perform.

Generally speaking, you would only be considered for a transplant if the result of a pulmonary function test, called the forced expiratory volume in one second (FEV1), dropped below 40 percent. Moreover, your lung functions would need to have decreased to a point where mechanical ventilation is required to perform even the most basic of tasks.

If you are accepted, you are placed on a national lung transplant waiting list. Eligible children are offered lungs on a first-come, first-served basis. Adults, by contrast, are given a Lung Allocation Score (LAS) of 0 to 100 based on the severity of their condition. Those with a higher LAS will be given priority.

While it is impossible to predict how long your wait may be, according to research published in the American Journal of Transplantation, the median wait time for a lung transplant is 3.7 months. Some recipients may get their lungs faster than this, while others may have to wait for years.

How the Surgery Is Performed

Once a donor organ is found and determined to be a match for you, you are immediately scheduled for surgery at a specialist hospital experienced in transplants. In most cases, you would undergo a double lung transplant rather than a single one.

After having an electrocardiogram (ECG) and chest X-ray, you are taken to the operating room and provided an intravenous line in your arm for the anesthesia. Other IV lines are placed in your neck, wrist, collarbone, and groin to monitor your heart rate and blood pressure.

Once the general anesthesia is delivered and you are asleep, the transplant takes six to 12 hours to complete and involves the following steps:

  • The surgeon makes a horizontal incision under your breasts from one side of the chest to the other.
  • You are placed on a heart-lung machine to ensure oxygen and blood is being continuously circulated through your body.
  • One lung is removed, clamping off major blood vessels, and the new lung is inserted in its place.
  • The surgeon then sutures the airway pipes and reconnects the major blood vessels.
  • The second lung is then transplanted in the same manner.
  • Once the transplant is completed, chest tubes are inserted to drain air, fluid, and blood. 
  • Finally, you are removed from the heart-lung machine once your lungs are working. 

What to Expect After the Surgery

Once the surgery is completed, you are placed in an intensive care unit for several days, where you are kept on a respirator and provided nutrition through a feeding tube. The chest tubes are kept in place for several days and removed once you are stabilized.

Once stabilized, you are transferred to a hospital room for anywhere from one to three weeks to begin your recovery. To avoid organ rejection, you are placed on lifelong immunosuppressive drugs.

Complications of lung transplant include infection, bleeding, and sepsis. Exposure to immune suppressive drugs can result in post-transplant lymphoproliferative disorder (PTLP), a form of lymphoma that can cause tumor masses, gastrointestinal inflammation, and bowel obstruction. 

Once home, the average recovery time is around three months and involves extensive gym-based rehabilitation with a physical therapist.

Advances in post-treatment care have increased survival times for lung transplant recipient from 4.2 years in the 1990s to 6.1 years by 2008, according to research published in the Journal of Thoracic Diseases.

Complementary Medicine (CAM)

Complementary therapies are often embraced by people with cystic fibrosis to improve breathing and enhance appetite and nutrition. If you decide to pursue any form of complementary or alternative medicine (CAM), it is important to speak with your doctor to ensure that it neither conflicts with your therapy nor causes harm.

By and large, CAMs are not regulated in the same way as pharmaceutical drugs or medical devices and, as such, cannot be endorsed as effective means of treatment. Still, there are some CAMs that are safer than others and a few that may even be beneficial to people with CF. 

Buteyko Breathing

Buteyko breathing is a breathing technique that involves the conscious control of your breathing rate and/or volume. It is believed by some to improve mucus clearance without the exhaustive process of coughing.

Buteyko breathing incorporates diaphragmatic breathing (known as Adham pranayama in yoga) as well as nasal breathing (Nadi shodhana pranayama). While evidence of its benefits is poorly supported, it is not considered harmful and may help reduce stress, anxiety, and sleep problems.

Ginseng

Ginseng is a cure-all used in traditional Chinese medicine that often promises more than it delivers. With that being said, the oral use of a ginseng solution in mice was shown to disrupt the protective biofilm of the Pseudomonas auruginosa bacteria commonly associated with CF lung infections. (The same result cannot be guaranteed in humans, however.)

Research from the University of Copenhagen suggests the disruption of the biofilm by ginseng may impede bacterial colonization and support antibiotics in controlling infections.

Turmeric

Turmeric contains a powerful anti-inflammatory compound called curcumin that works much in the same way as COX inhibitor medications. It is unclear whether it can reduce the inflammatory effects of CF since it is so poorly absorbed in the intestines and unlikely to reach therapeutic levels, according to research published in the Journal of Genetic Syndromes and Genetic Therapies.

While generally considered safe, turmeric overuse may cause bloating and indigestion.

Marijuana

Medical marijuana, while entirely inappropriate for children and teens, is known to be a powerful appetite stimulant for people with the disease- or treatment-associated anorexia. However, it is unclear what effect smoking marijuana may have on lungs already heavily damaged by CF.

To this end, there is some early evidence that oral drugs containing marijuana's active ingredient, tetrahydrocannabinol (THC), may not only help achieve weight gain but improve FEV1 in people with CF. Research is ongoing.

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