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Azithromycin for People with CF:
Is It Right for You?
 

Azithromycin is a commonly used antibiotic. You may know it as Zithromax®. Many people have taken this drug to treat pneumonia, sore throats or ear infections.

In a clinical trial, it was shown that azithromycin helped people with CF who had Pseudomonas aeruginosa (Pseudomonas) in their lungs. Their lung function improved, they had fewer pulmonary exacerbations (lung infections) and they were able to gain more weight.

Another clinical trial was done to see if azithromycin helped kids with CF who did not have Pseudomonas. The result of this 6-month clinical trial was published in the Journal of the American Medical Association (JAMA) and another study was reported in Pediatric Pulmonology.

Also, a study published in the Journal of Clinical Investigation thought there might be a link between the use of azithromycin and an increased risk of infection with nontuberculous mycobacteria (NTM). In early 2013, the U.S. Food and Drug Administration (FDA) sent out information stating that azithromycin may cause irregular heartbeats. 

You may be asking: What does this mean for me or my child? Here is some information that can help you talk to your CF health care provider to see if you or your child should be taking azithromycin regularly.


What were the results of the first trial, for those people with Pseudomonas?

During the first clinical trial, lung function (FEV1), weight, use of antibiotics and days spent in the hospital to treat lung infections were watched for changes. The people with CF who were involved in the study were split into two groups. One group took a placebo — an inactive medication or sugar pill. The other group took azithromycin.

During this trial, neither the patients nor the CF researchers knew which patients took azithromycin or placebo pills. Over six months, the group that took the azithromycin had about a 6 percent improvement in their lung function, had fewer pulmonary exacerbations, took fewer antibiotics (other than azithromycin) and had an increase in their weight. They also spent 47 percent fewer days in the hospital for the treatment of a lung infection.

This clinical trial was done with the support of the CF Foundation.

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What were the results of the second clinical trial for kids without Pseudomonas?

During the second clinical trial, lung function (FEV1), weight and lung infections were watched for changes. The people with CF who were involved in the study were split into two groups. One group took a placebo — an inactive medication or sugar pill.

The other group took azithromycin. During this trial, neither the patient nor the CF researchers knew who took which pill. Over six months, the group that took azithromycin had fewer exacerbations, took fewer oral antibiotics and had an increase in their weight.

Lung function was about the same in those who took placebo and those who took azithromycin. However, the people in the azithromycin group reported less coughing than the group taking the placebo.

This clinical trial was done with the support of the CF Foundation.

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Were there any side effects?

The side effects found in both studies were mild. In the study of people with Pseudomonas, those on azithromycin had more nausea, diarrhea and wheezing.
In the study of kids without Pseudomonas, those on azithromycin did NOT have more side effects. A few people had side effects that made the researchers reduce the amount of the drug that they took.

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What does the study in the Journal of Clinical Investigation mean for people with CF?

The article in the Journal of Clinical Investigation suggested that long-term use of the antibiotic azithromycin by people with CF might increase the risk of an infection with NTM. NTM can cause lung problems in people who have CF.

However, this study was done in the laboratory and did not involve testing in people, including those with CF. Therefore, what it means to someone with CF is not yet known. The CF Foundation is working with the world’s experts in this area to determine if there is a potential risk for people with CF and what should be done.

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What does the FDA's message about irregular heartbeats mean for people with CF?

In early 2013, the U.S. Food and Drug Administration sent out a communication stating that azithromycin may cause irregular heartbeats.

People at risk for having an irregular heart beat while taking azithromycin are those with known risk factors. These are people who have heart rhythm problems, low levels of potassium or magnesium in the blood, a slower-than-normal heart rate, and people using certain drugs to treat irregular heartbeats or arrhythmias. However, in a recently published study in the 2013 May New England Journal of Medicine, azithromycin use was not linked with an increased risk of death from cardiovascular (heart) causes in a general population of young and middle-aged adults.

People with CF and their families should talk to their CF care providers about the benefits and the potential risks of taking azithromycin and decide if it’s the right medication. Talk to your CF doctor BEFORE you stop any medication that has been prescribed.

Click here to read the FDA’s drug safety communication related to azithromycin.

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Is azithromycin right for me or my child?

Your CF health care team can help answer this question. Knowing who was chosen for the clinical trials might help you decide if azithromycin is right for you or your child.

In the first study, the people with CF who took part in the study had a positive culture for Pseudomonas for at least one year, were 6 years of age or older, weighed at least 55 pounds and had mild to moderate lung disease. If a patient had a sputum culture that contained NTM or Burkholderia cepacia complex (B. cepacia), or had liver disease or kidney disease, they were not included in the trial.

In the second study, the kids with CF who took part in the study did not have Pseudomonas for at least one year, were 6 to 18 years of age and had mild to moderate lung disease, although most had mild lung disease. If a patient had a sputum culture that contained NTM or B. cepacia, or had liver disease or kidney disease, they were not included in the trial.

Your CF health care team can provide you with more information. If you are thinking about starting azithromycin, you or your child should have sputum cultures done to see if there is NTM in the sputum. Blood tests should also be done to see if there are signs of liver or kidney disease. These tests are important to help your CF health care team know if azithromycin is right for you or your child.

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Who should NOT take azithromycin?

Anyone who is allergic to azithromycin, erythromycin or any macrolide-related antibiotic should not take azithromycin. People with liver disease, pregnant women and those with a positive culture for NTM need to talk with their CF health care team before starting azithromycin.

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What are nontuberculous mycobacteria (NTM)?

Nontuberculous mycobacteria are a type of bacteria found in the environment and sometimes cause lung infections in people with CF. Lung infections from this type of bacteria can be passed between people with CF. Also, they can be treated with the proper medications. However, if you have NTM in your sputum, it could affect your doctor’s decision about whether to start you on azithromycin. Ask your CF health care team for more information or if you have concerns. You can learn more about NTM by watching the CF Education webcast Nontuberculosis Mycobacteria (NTM) in Cystic Fibrosis.

Talk to your CF doctor BEFORE you stop any medication that has been prescribed for you or your child.

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What about other medications?

If you take antacids (such as Mylanta®, Maalox®), take them two hours before or four hours after the azithromycin is taken. As with any medication, you should tell your CF health care team what over-the-counter vitamins, herbals and other CF drugs you or your child are taking.

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How is azithromycin taken?

Azithromycin is a tablet that can be taken with food. In clinical trials, people with CF took one or two azithromycin pills on Monday, Wednesday and Friday. How many pills a person takes is based on how much the person weighs.

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Should people with CF who are taking azithromycin stop taking other CF drugs?

Azithromycin is just one more “tool” that can be used in CF care. It may be used as a part of your or your child’s regular CF treatment. It is not meant to replace other proven CF drugs. Do not stop any therapy before you talk with your CF health care team. It is always a good idea to talk about all of your therapies with your CF health care team to make sure that you or your child are getting the proper treatment.

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What happened to the germs or bacteria in the lungs of the people in the first trial?

In the first study in people with Pseudomonas, there was no change in the germs. More studies are planned to figure out exactly how azithromycin works in CF.

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What about antibiotic resistance?

People can — and do — become resistant to antibiotics that are taken over a long time. This did not seem to happen in the azithromycin clinical trials. Your CF health care team will watch for signs of resistance to azithromycin and any other antibiotics.

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Was the CF Foundation involved in the trials?

The CF Foundation worked with the principal investigators to conceive, design and initiate both clinical trials to bring azithromycin to people with CF. In addition to providing the funds to do the studies, the CF Foundation supported the Therapeutics Development Network involving CF Foundation-accredited care centers that participated in these clinical trials.

To find a cure for this disease, the CF Foundation follows the approach of pursuing several different strategies. It continues to invest millions of dollars in science that uses the latest technology to discover new CF-specific drugs.

At the same time, the azithromycin studies are a part of a “low-hanging fruit” strategy to study the potential of drugs already on the market for other diseases that may have a benefit for people with CF.

Your participation is essential in the process of developing new drugs and making them available sooner for everyone with CF. The CF Foundation thanks all of the people with CF who learn, ask and join CF clinical trials and acknowledges the efforts of the physicians, research coordinators, study leaders and Pfizer who made this clinical trial a success. 

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Learn more about chronic medications for CF lung health

You can learn more about chronic medications for CF lung health, including azithromycin on the CF Foundation’s website:

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Updated 5/22/2013

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