Dr. Michael S. Schechter, MD, MPH
Director of the Emory Cystic Fibrosis Clinic, Associate Chief of the Division of Pulmonary, Allergy/Immunology, Cystic Fibrosis and Sleep, and Associate Professor of Pediatrics and Environmental Health at Emory University

The reason why the doctors all seem to have different views on this is that nobody knows for sure.  This is because almost all the research on safety and effectiveness of Pulmozyme (or alfa dornase) has been done on patients over the age of 5 years. There was one study 10 years ago that looked at the effect of Pulmozyme in children under 5, and it suggested some benefit, but only 12 children took part in the study.

The situation is the same for just about all of the other CF therapies; in fact, it is true for most medications that are used in children with or without CF.  It is much more difficult to do research on young children because of ethical considerations, and it is often harder to measure the changes that suggest benefit and safety.  Certainly, it is more expensive for the pharmaceutical companies to do this kind of research.

Pulmozyme works by loosening secretions that are made thick by the presence of white blood cells in the mucus that are there as a result of chronic infection.  While this suggests that it would work best in people with advanced lung disease, we know from research in children over 5 that it is effective even in those with mild lung disease.  Thus, it is quite possible that early therapy with Pulmozyme, before lung disease is established, will provide significant long term benefits for younger children with CF. On the other hand, it is also possible that Pulmozyme wouldn’t help, which at best might mean a waste of time and money, and at worst it might cause side effects that we don’t know about.

The bottom line: although we know that Pulmozyme is effective in children over 5, even those with mild lung problems, nobody really knows for sure the best age that Pulmozyme should be started and under what circumstances.  Therefore, it is a personal decision between you and your physician.  Parents may have different opinions, just as doctors might, so the decision that is right for you will not be the same as the decision that is right for someone else.

Rebecca Hefele, R.Ph

This is a tricky question, there really is no information to advocate for or against the use of Pulmozyme in infants. Micromedex specifically states that there is limited information in children < 5 years, therefore the risk/benefits would have to be carefully considered. That being said, there is an ongoing trial investigating the use of Pulmozyme in infants (1 month-30 months), but there are no results yet. It is scheduled to end December 2011. The Pulmozyme prescribing information does include information for use with baby nebulizers and also states that there seems to be an increase in cough and rash with children < 5 years old. Additionally, Cystic Fibrosis Pulmonary Guidelines do state that Pulmozyme is recommended for mild, moderate, and severe disease in children  > 6 years. We think that it probably isn’t appropriate as a preventative treatment, especially if there are no symptoms to treat; BUT, not knowing really anything about the baby’s condition we couldn’t really say yes or no either way.
Hope this is helpful;

Rebecca L. Hefele, R.Ph.
Anna Raymer – pharmacy candidate 2012
 

I was personally so honored to be involved in this video series.

Now online! Living with cystic fibrosis means making time to fit your treatments into your busy schedule. Now on http://bit.ly/qCFQGQ, you can check out a Video Short in which CF patients, experts and caregivers discuss the importance of establishing a routine and understanding how your daily treatments work to help keep you healthy. The video features Lisa Greene, certified parent coach, educator, and co-author of Parenting Children with Health Issues; Dr. Foster Cline, child psychiatrist, co-founder of the Love & Logic® Institute and co-author of Parenting Children with Health Issues; America’s Got Talent finalists, Ali and Christina Christensen, and their parents; Kat Quinn East, CF parent and founder of the Blooming Rose Foundation; and Somer Love, an adult with CF and author of the blog, Love to Breathe. The stories in this second installment of the CF Community Video Short series may help you to find new ways to motivate your kids to adhere to their treatments and instill the power of choice.

Dr. Jerimiah Lysinger, MD
Director of the Billings Clinic Cystic Fibrosis Center

This is an excellent question.  Azithromycin has traditionally been used in patients with CF who are older than 6 and chronically infected withPseudomonas aeruginosa.  However, this has been changing recently as this medication is now being used in patients without Pseudomonasand many providers will start it earlier than 6.  In studies involving patients with CF who are chronically infected with Pseudomonas, the studies have shown an improvement in FEV1 (pulmonary function tests), a reduction in the number of pulmonary exacerbations, a reduction in the number of days or courses of antibiotics needed (other than azithromycin), and an increase in weight.  In studies with patients with CF who do not have P. aeruginosa, patients had no difference in pulmonary function tests, but did report less cough.  Most of the side effects of azithromycin are related to the stomach and can include nausea and diarrhea.  The Cystic Fibrosis Foundation has a wonderful discussion of azithromycin at  http://www.cff.org/treatments/Therapies/Respiratory/Azithromycin/ that I would recommend.

Terri A. Laguna, MD, MSCS, FCCP, FAAP

Assistant Professor of Pediatrics, Pediatric Pulmonary Medicine
Associate Director of the Minnesota CF Center

The question of what to do in the setting of a throat culture positive for MRSA is a dilemma in the CF world.  In the situation described, with a 4 year old girl testing positive for MRSA, it is an even bigger question.  Based on the information provided, I gather that the 4 year old girl is relatively healthy.  I assume that her BMI is acceptable and that she is not having recurrent pulmonary symptoms.  Not sure if she is able to perform pulmonary function testing yet, given her age, so there may be little information available by which to judge her lung health.  I think it is safe to say that there is no consensus among CF Centers and physicians with what to do in this setting.  The CFF also has not provided recommendations.  There are studies out there that provide different results regarding the impact of MRSA on lung function and future health.  I guess things to think about would be:  Is the girl having problems or is she growing and developing on track with no pulmonary issues?  It is hard to think about aggressive IV antibiotics for a POTENTIAL pathogen that may or may not be causing problems.  Only a bronchoscopy would provide information to help decide if the MRSA is in her lungs or just in her throat.  A bronchoscopy involves sedation and is not without risk.  Most centers decide what to do based on a risk/benefit approach.  If the 4 year old is doing well, I can understand the “wait and see” approach.  If she is not, the center would likely become more aggressive in their approach to diagnosis and treatment.

Walter Van Praag
CF Blogger and Activist

Pwcf live with various untreatable lung infections. The name MSRA sounds terrible, but is no worse than all the other antibiotic resistant infections that we culture. Think of a pwcf as a trawling fishing net; when the nasties come near us we get them. All we can do is treat flare-ups with ‘tune-ups’ – aggressive IV and oral antibiotics treatment. 

I for one have tested positive for some nasty bugs in the past and when they make me sick I get bombarded with antibiotics the doctors choose for me. Other times the nasties don’t show up at all!! Some show up again occasionally over the year and some just disappear. 

Main thing is to treat aggressively when they cause problems and be diligent with regular daily treatments/drug regime. Ensure the pwcf remains as active as possible, eats as much as possible, plans for the future and has fun.  
Don’t dwell on things that aren’t affecting you yet!

Dr. Jerimiah Lysinger, MD
Director of the Billings Clinic Cystic Fibrosis Center

The answer to your question depends on what you mean by “bugs”.  In patients who have chronically grown bacteria seen in CF such as H. influenzae, S. aureus, P. aeruginosa, or MRSA it would be rare for them to suddenly have a clear culture.  What frequently happens is that the culture was run in a lab that does not specialize in CF cultures or does not realize what are CF related pathogens.  For example,H. influenzae may be reported as normal flora and occasionally, S. aureus  is as well.  Most labs who are used to running CF cultures will report out most of the bacteria that grows even if it is normally part of the normal flora.  Another instance where cultures may suddenly look clear is in a pt who has throat swabs and an adequate swab was not obtained.  Therefore, I have seen cultures suddenly appear clear in CF patients, but I am always suspicious of lab error or culture technique error.

Josh Mogren
CF Speaker, Blogger and Entertainer

“Can you get a culture with “normal flora” as in “no bugs to be seen anywhere in the the culture”? I’m no expert, but I would think if you had the right magical cocktail of drugs AND/OR the lab culture everything perfectly…then…yes, I would guess so. Is it really hard to do this? Moreover, If you do achieve this, is it easy to maintain that “normal flora”? The answer to both of those questions depends on “your CF”. Our bodies work differently. Different immune systems mean different long term responses to drug therapy, different abilities an individual’s body has to fight off infections from different bugs, and environmental factors that we have little control over. In other words…it’s not impossible, but it is very hard to do.
Don’t be discouraged by this answer, though. Just because your child’s culture isn’t “normal flora” doesn’t mean they aren’t healthy. Even as a CF adult, I usually have a pretty clear culture right after a treatment of oral or IV antibiotics. Yes the “bugs” come back, but that initial culture lets me know that I can get back to (or as close as possible) to square one. It’s harder as we get older, but so is everything with age. Hang in there, my distant friend.

Meg Benefield, MSW, LICSW
Clinical Social Worker, University of Minnesota Medical Center, Adult CF Program

Thank you for your excellent question; it is a common concern!  Anti-depressant therapy can be extremely helpful for people struggling with depression.  Finding the right medication can be challenging, but it is usually possible to find one that treats depressive symptoms without causing intolerable side effects or drug interactions.

Major Depression is one of the most common and treatable mental health diagnoses.  Depression is different from grief or sadness.  Unlike emotions that may come and go, depression is a “global” mood shift of sadness and/or loss of interest in activities that lasts for 2 or more weeks.  It also includes some combination of the following symptoms: weight/appetite change, fatigue, difficulty concentrating/making decisions, sleep problems, feelings of guilt/worthlessness, being excessively active or extremely slow-moving, and/or thoughts of self-harm/suicide.

Depression can vary from mild to life-threatening, but it always interferes with a person’s life.  In CF, depression can affect self-care through reducing motivation and energy to keep up with medical treatments.  It can worsen nutritional problems and interfere with getting enough rest.  Depression can make coping with the chronic demands of CF overwhelming.  For some people, depression is a recurrent problem that requires lifelong treatment.  For others, it may be a one-time experience, triggered by a stressful situation (i.e., health complications).  If you think you are depressed, it is very important to seek help.  People who are depressed may experience thoughts of self-harm or suicide.  Left untreated, these thoughts can lead to actions.

The first step to getting effective treatment for depression is receiving a diagnosis from a skilled professional.  Diagnosing depression can be complicated, especially if the symptoms overlap with health issues.  In diagnosing depression, it is important to rule out medical factors (i.e., underactive thyroid).  It is also important to assess whether a person has any other mental health concerns, like anxiety.  Life stressors should also be noted.  When possible, seek evaluation from a licensed mental health professional (psychiatrist, psychologist, therapist or clinical social worker).  Primary care or other physicians may also help diagnose depression, but they have not necessarily received training to do a thorough evaluation.

Once diagnosed, treatment for depression ideally includes both medication and counseling.  Counseling helps address stressors and strengthen a person’s coping skills.  Medication helps stabilize mood and reduce the other symptoms of depression.

There are many antidepressant medications.  Antidepressants act on specific neurotransmitters in the brain that influence mood.   Antidepressants fall into different classes depending on how they affect the neurotransmitter systems in the brain.

Some of the more commonly used classes:

Selective serotonin reuptake inhibitors-SSRIs: Prozac, Zoloft, Paxil, Celexa, Lexapro and Luvox

Serotonin and norepinephrine reuptake inhibitors-SNRIs: Cymbalta and Effexor

Norepinephrine-dopamine reuptake inhibitor (NDRI): Wellbutrin

Antidepressant medications generally take 2-4 weeks to work and even longer to have their full effect.  Different people respond better to certain medications.  Sometimes the first medication prescribed is effective, while other times, it takes several trials to find the best medication.  Some people benefit from taking more than one antidepressant or taking other medications to address anxiety or sleep problems.

Concern about side effects is a common barrier to people trying or staying on antidepressants.  Each medication has a list of possible side effects, which can include things like nausea, nervousness, insomnia, and low libido.  Generally, side effects are worst in the first 1-2 weeks and lessen with time.  Side effects can be addressed in several ways.  If the symptoms are tolerable, simply waiting may allow them to naturally subside.  If the symptoms are intolerable or do not improve, changing the time of day when the medication is given, changing the medication dosage or changing the medication itself might help.  Due to safety issues, you should never change, wean, or stop antidepressant medication without professional guidance.

If you are on certain medications or have other underlying medical issues, some antidepressants may not be safe.  Medication safety is completely dependent on your specific medical history and list of medications.  For example, having significant CF liver disease complicates medication safety.  Therefore, you need a physician and pharmacist to review any new medication.  However, most people with CF can safely use one or more classes of antidepressants.

Prescribing and managing antidepressant medication is complex, so seeing a psychiatrist is recommended if possible.  A psychiatrist is a medical doctor with advanced training in the diagnosis and treatment of mental health conditions.  Psychiatrists are especially helpful when making medication adjustments or dealing with more complex issues (i.e., both anxiety and depression).  Since people with CF have so many other appointments, they can be understandably reluctant to add another.  In the case of psychiatry, I think it is worth the upfront time investment.  Once you’re on an antidepressant that works, you won’t need frequent psychiatry appointments.

Given all the above challenges, in your words, “How do you get a cf adult to consider trying such?”

My approach is to educate people and let them make an informed decision.  When someone is experiencing depression, I want them to understand the condition and the treatment options.  Unless someone is threatening self-harm, my role is to counsel, educate and then respect the person’s decision.  Some people will not try medication at all, saying the benefits simply do not outweigh the risks.  I have seen other people suffering from depression regain energy, enthusiasm and the ability to cope with life demands after receiving antidepressant treatment.  Some people recover after the first medication is prescribed.  Other people have to try several medications before finding something that works.  Some people are on medication short-term, others life-long.

Antidepressants can be extremely helpful and should always be discussed and considered as a treatment option for depression.  The final outcome of what is tried and what works is truly individual.

Resources:

Information on Depression/treatment options:

http://www.nami.org/Content/NavigationMenu/Mental_Illnesses/Depression/Mental_Illnesses

_What_is_Depression.htm

Safety information for specific medications:

http://www.fda.gov/Drugs/DrugSafety/ucm085729.htm

Crisis/Suicide Hotline (national):

1-800-273-TALK (8255)

http://www.suicidepreventionlifeline.org/

Walter Van Praag
CF Blogger, Published Author and Activist

My biggest concern is that doctors prescribe non-compatible mediations. PWCF frequently visit multiple medicos and get prescribed tons of different medications. It is really important that we as the final swallower check to make sure nothing interacts. Best way to do this is to use a drug interaction checker such as http://reference.medscape.com/drug-interactionchecker.I have it on my iPhone so when i get prescribed something at the doctor’s surgery I can immediately add it to the list and see if there are any undesirable a) side effects or b) interactions.

Often there are minor interactions and you can read the possible consequences, which may be better than not taking them, but often it just tells you to take it 3 hours or more apart. In those cases I try separate some medication by a full day at least just to be on the safe side. I was recommended to take a Typhoid inoculation prior to travel recently and decided against it as it had possibly ‘life threatening’ interactions with the antibiotics i am on. I do not wish to stop the antibiotics for three days and get sick before I travel and hoped the natural immunity pwcf might have against typhoid fever would suffice. It is a gamble! Same with anti depressants, choose wisely and check for possible interactions! And remember that simple pills like Calcium Carbonate and Salt tablets should also be added to the interaction checker!

Dr. Michael S. Schechter, MD, MPH
Director of the Emory Cystic Fibrosis Clinic, Associate Chief of the Division of Pulmonary, Allergy/Immunology, Cystic Fibrosis and Sleep, and Associate Professor of Pediatrics and Environmental Health at Emory UniversityThere is no CF Guideline or formal consensus on bronchoscopy, so I will offer my opinion:

The most common reason to perform a bronchoscopy is to get a better idea of what bacteria are growing in the bronchial tubes.  In younger children, we usually get throat cultures to learn what bacteria that inhabit their respiratory tract.  These are not 100% accurate, but they provide adequate information in children who are doing well.  However, if a child is experiencing symptoms that do not resolve with oral antibiotic treatment chosen based upon previous throat cultures, it suggests that there might be other bacteria in the airways that need specific treatment.  So in that situation, particularly before initiating IV antibiotics, a bronchoscopy is typically done to ensure that we know what bacteria need to be treated. If a child is able to cough up (expectorate) sputum, cultures are more reliable and it might not be necessary to perform a bronchoscopy on them.

When my CF patients are having anesthesia for other reasons (sinus surgery, ear tubes, feeding tubes, etc), I will usually try to use that opportunity to perform a bronchoscopy even in children who are doing well, because the procedure is safe and adds no additional risk to the procedure that is primarily being done.  I do sometimes learn something unexpected when I look down into the airway “just for the sake of looking”.

Another, less common situation where a bronchoscopy may be helpful is when the chest x-ray indicates that an area of the lung might be plugged with mucus, and vigorous airway clearance therapy combined with antibiotics and mucus-clearing medications has not resolved the problem.  In that case, a bronchoscopy might be done to help clear out the mucous plug.

Please note that this is not an exhaustive list of reasons to do a bronchoscopy – just the most common reasons.

Katie M. McDonald, PhD, RD
Clinical Dietitian – Primary Children’s Medical Center

Cystic Fibrosis Related Diabetes (CFRD) results from gradual pancreatic destruction in cystic fibrosis causing progressive insulin deficiency.  CFRD is more common with pancreatic insufficiency and occurs with increasing frequency as people with CF age. Approximately 43% of adults with CF over age 30 have CFRD. Although CFRD is similar to the more common types of diabetes type I (insulin-dependent) or type II (non-insulin dependent)significant differences exist as well.  Dietary management is important in CFRD; generally caloric restriction is not recommended.  Most people with CFRD require some insulin each day.

Currently, there are no proven specific measures to prevent CFRD.   Potential strategies to delay the onset of CFRD may include:

• maintenance of a healthy body weight,
• daily moderate to vigorous exercise,
• healthy eating habits/schedules with balanced meals, possible avoidance of high carbohydrate loads (such as sugary drinks or candies) especially when no other foods (such as proteins or fats) are consumed,
• maintain adequate serum vitamin D levels.

Remember, early identification and treatment of impaired glucose tolerance or CFRD can prevent decline in lung function or unhealthy weight loss.  Be alert for symptoms of diabetes such as increased thirst and increased urination. Other symptoms of CFRD may include excessive fatigue, unintentional weight loss and unexplained decline in lung function.  Annual monitoring for CFRD with an oral glucose tolerance test (OGTT) is recommended for adults with CF.

Aimee Lecointre
Adult with CF

I take the glucose test twice yearly and am always in the ‘gray area’. I have been in the ‘gray area’ for years and years now. My doctors and I have discussed this and really there is nothing to do to reverse this or prevent my body from making that final leap to being diagnosed with CFRD, and there is no way of knowing if and when that may or may not happen. I think exercise and a balanced diet are important for everyone, but I don’t know that there is a way to prevent the onset of CFRD. I personally try and stay away from consuming too many sugary sweets and opt for ‘sugar-free’ and ‘no sugar added’ options when possible. I have talked to a few friends who have been diagnosed with CFRD and the majority of them have been told not to restrict calories are given insulin to help control their CFRD.

Douglas Conrad, MD

You ask a very important question.  First of all congratulations on the successful control of your M abscessus infection.  I know that took a tremendous effort but in my opinion will be critical to your long-term health.

As you know, mycobacteria are a large family of bacteria found throughout our environment.  In my practice, I refer to them as “cousins” of the bacteria that cause tuberculosis.  Unlike those with tuberculosis, patients with these “cousins” of TB (or as they are sometimes referred to “non-tuberculous mycobacteria”)  do not pose a public health threat.  In other words, patients with these non-tuberculous mycobacteria are not harmful to others in that we do not believe they will spread infections to family, friends, household residents or people they meet casually.  In my experience, it is very rare that someone would have two simultaneous mycobacterial infections as you have had.

Treating patients with mycobacteria can be very challenging.  The therapy typically includes treatment with at least three drugs for many months… in some cases up to 18-24 months.  Many of the antibiotics require intravenous administration.  Generally speaking therapy is continued for 12 months after sputum cultures become negative for the mycobacteria.

Antibiotic selection is a challenge for many reasons.  One major reason is that the antibiotic susceptibility tests performed in the microbiology lab don’t always predict medical outcome.  For example, just because a mycobacterial isolate in the lab is sensitive to ethambutol doesn’t guarantee that the treatment will be a clinical success.  In some cases there is a correlation of sensitivity with clinical therapy success such as the case of clarithromycin susceptibility of  MAC/MAI isolates.  The microbiology lab can provide clinicians with a list of antibiotics that the isolate is susceptible at clinically relevant blood levels.  The challenge then is to find 3-4 of these antibiotics that the patient can take given their underlying medical problems and tolerance issues.  Another challenge is that each clinician rarely has a large number of patients with similar isolates and underlying medical condition.  The result is a lot of empiric therapy.  I believe this is why you received two different recommendations.

MAC/MAI turns out to be the most common non-tuberculous mycobacteria and as a result there are some guidelines for treating patients.  If a CF patient has persistently positive sputum cultures which are sensitive to clarithromycin, they likely fulfill the criteria by the American Thoracic Society and the Infection Disease Society of America for treatment with clarithromycin, ethambutol and rifampin.  The previous therapy for the M abscessus or complications of the CF might confound these recommendations.  I think it is very wise to seek out multiple opinions and would consider those opinions from clinicians with the greatest experience in this field.  Along these lines I think it is a very good idea to seek out advice from the NIH as you are.  Other institutions with a lot of experience in this field are located at National Jewish Hospital in Denver and University of Texas at Tyler.  I wish you well and hope this was helpful.

Angie Bates, DPT

I agree that both Cranio-sacral (CS) and myofascial release (MFR) treatments are both effective and beneficial for many people.  Physical therapists may not advertise as being MFR or CS trained since it is just another “tool” in their bag.   These are courses that are available to PT’s, massage therapists, chiropractors, and other ”body workers” who tend to advertise this specialty more.  As a PT, we are trained to use a variety of techniques including strengthening, soft tissue mobilization, joint mobilization and other manual therapy techniques, which include CS and MFR.  You won’t find many PT’s who only use one treatment protocol.    Unfortunately, there is not much research behind CS or MFR, so a group like the American Medical Association or American Physical Therapy Association may not promote it. CS therapy has been compared more to “energy work” than a manual therapy technique.  It is very subtle and gentle.  MFR is a well known and used manual therapy technique.  The bottom line is to use your best judgment and find a therapist who uses the right “tools” for your needs.  Ask what manual therapy techniques they use and what they think of MFR and CS.  No matter what, your PT, massage therapist, or chiropractor should be able to provide a very specific treatment plan to address your particular needs.

Sharlie Ross Kaltenbach

I have had cranio-sacral work and lymph massage as well as other treatments like accupressure, massage, applied kinesiology and chiropractic work as well as other “energy work.” I believe this is one of the reasons that despite my low lung function, I seem to do so well and have been able to put off transplant for more than 10 years when they first listed me. I would not say I am an expert but I highly recommend pursuing complimentary treatments. My experience in discussing these treatments with my doctors is that they really aren’t educated enough about them to give me any input and in general are pretty skeptical : )  I have never allowed my doctor to persuade me from trying something new for better or for worse… I have gone with my instincts and stayed very in tune with my body and what feels good. For the most part “eastern” or “alternative” treatments have been around much longer than “traditional” medicine and I think it’s great to explore other healing methods out there.

Josh Mogren