Cystic Fibrosis

How Is Cystic Fibrosis Treated?

Treatment for cystic fibrosis includes:

• Nutrition care. Good nutritional status is strongly associated with good respiratory status. The goal for all CF patients is to be well-nourished and at an appropriate weight for their height. Your child can achieve a well-nourished state by:
  • Eating a high-calorie diet
  • Routinely taking vitamins/minerals
  • Routinely taking pancreatic enzyme replacement medicines
  • Drinking nutrition supplements
  • Supplementing with tube feedings
• Medicines. Children with cystic fibrosis need different medicines to keep them healthy. These include:
  • Enzymes. Pancreatic enzymes are lacking in most children with CF, so these are taken to help absorb protein, carbohydrates and fat from food. The amount prescribed is based on your child's weight, and the dose will be adjusted often as your child grows. Generic enzymes should not be used for children with CF. Brand name enzymes include Creon, Pancreaze, Zenpep, Pertzye, Ultresa and Viokace.
  • Vitamins. Certain vitamins are poorly absorbed in children with CF. Extra vitamins are needed to keep children healthy. Vitamins prescribed to children with CF include AquaADEKs, Vitamax, MVW Complete Formulation, Libertas ABDEK and ChoiceFul.
  • Reflux Medicines. These medicines are needed to treat stomach reflux when it occurs. They are also prescribed to some children to help enzymes work better. Reflux medicines include Zantac (ranitidine), Pepcid (famotidine), Prevacid (lansoprazole), Prilosec (omeprazole), Protonix (pantoprazole) and Nexium (esomeprazole).
  • Laxatives. Laxatives are used to ease constipation. Types of laxatives include MiraLAX, docusate, senna and lactulose.
  • Actigall/Ursodiol. This medicine treats cystic fibrosis liver disease.
  • Medicines for the lungs. Inhaled or nebulized medications to maintain lung health include:
    • Pulmozyme. Pulmozyme is a preventive medication that helps break down mucus in the lungs making it easier to cough out.
    • Bronchodilators (Albuterol/Xopenex). Bronchodilators help open the airways to improve clearance of mucus.
    • Hypertonic saline. This is a strong salt solution that helps restore a normal environment in the lungs, makes the mucus more slick and easier to clear from the lungs and encourages a healthy cough.
  • Inhaled antibiotics. Antibiotics are chosen based on which bacteria are found on respiratory cultures, the symptoms during an illness, any allergies and a child's previous response to antibiotics. These medicines keep certain bacteria (pseudomonas) from growing in the lungs. Examples include TOBI (nebulized solution and TOBI Podhaler), Cayston and Colistin.
  • Steroids. Steroids are used less often in children who have asthma-like symptoms in addition to CF. They can be given for a short time by mouth or can be taken long term in an inhaled or nebulized form.
  • Oral antibiotics for respiratory illnesses. These medicines are used to treat bacteria in the lungs or sinuses that can cause infection. Common antibiotics used in CF are Augmentin, Omnicef, Bactrim, Ciprofloxacin and Linezolid.
  • CF modulator. Ivacaftor (Kalydeco) is a medicine taken by mouth that is approved for a small number of patients with CF. This medicine helps open the chloride channel that is broken in CF and leads to increased lung function and weight gain. The medication is taken with the other CF medicines and does not replace the other therapies. Patients who qualify for this treatment are older than 6 years old and have one of the following CF mutations: G551D, G178R, S549R, S549N, G551S, G1244E, S1251N, S1255P, G1349D or R117H.

The Cystic Fibrosis Foundation recommends that children with CF complete at least four clinical visits each year. Each patient’s visit will be different based on his or her specific needs. Children and their families can expect the following during regular appointments:

• Measurement. Your child’s weight and height will be measured and recorded without shoes.
• Body mass index (BMI). The Cystic Fibrosis Foundation recommends a BMI (weight in relation to height) in the 50^th percentile for all cystic fibrosis patients. New studies show that children with CF who are well-nourished have better lung function. A dietitian will calculate your child’s BMI and share it with the pulmonologist. Growth charts, body mass index percentiles and weight goals will be established at your child's annual screening. If there is a concern about weight, you and your child's doctor will discuss treatment options to improve nutritional status.
• Pulmonary function testing. Pulmonary function tests check how well the lungs are working. Spirometry is the most common test to check how well the lungs function. As part of pulmonary function testing, the doctor may also perform an oximeter reading. A pulse oximeter reading checks the level of oxygen in the blood. The reading is done by placing a pulse oximeter device on your child's finger. This test is painless.
• Airway clearance therapies (ACT). Airway clearance loosens mucus so it can be cleared by coughing or huffing. It moves mucus from small to central airways to be coughed or huffed out. The goal of ACT is to prevent and reduce lung infection and to improve and maintain lung function. When air gets behind mucus and airflow is increased, mucus is pulled along. The results are reduced lung infection and improved lung function. Types of airway clearance therapies include:
  • Coughing. This is the most basic airway clearance technique. It clears mucus with high-speed airflow. Huff coughing can be less tiresome and may work better. Huff coughing is a gentle way of coughing that speeds airflow while keeping the throat open.
  • Mucociliary clearance. Normally, mucus protects the airway from inhaled irritants. The mucus lies on the airway like a blanket of jelly. Hairs called cilia line the airway and sweep the mucus upward so that it can be coughed out.
• Laboratory work. Your child's doctor may perform several tests to monitor overall health and body functions. Types of lab tests that may be performed include blood tests, urinalysis, sputum or respiratory culture (expectorate or throat swab), stool tests and fecal elastase to assess pancreatic function.
• Radiology tests. Imaging of your child's lungs and bone density help monitor his or her condition. Your child may undergo tests such as a chest X-ray and a DEXA scan.

At each appointment, you and your child will meet with the following medical professionals to discuss and review your child's healthcare plan:

• Doctor. The doctor will perform medical exams and review the body's systems. He or she will also provide prescriptions for any new or continued treatment plans.
• Nurse. The nurse will:
  • Review medicines
  • Look at your child's health history
  • Perform a throat culture and explain why it is important
  • Explain or review your child's care plan
  • Give flu shots
• Dietitian. The dietitian will review weight, BMI status, eating practices, pancreatic enzymes, vitamins and stool/gastrointestinal history and make recommendations for improving or maintaining nutritional status.
• Pulmonologist. A pulmonologist will work with you and your child to monitor the results of pulmonary function tests over time and ensure that proper respiratory therapy for airway clearance is being followed.
• Respiratory therapist. A respiratory therapist will review respiratory treatments and medicines, review order of therapy, make recommendations for improving and maintaining respiratory status and review how to clean and maintain nebulizers and equipment.
• Social worker. Social workers provide assistance with insurance, financial concerns and personal concerns related to CF.
• Pharmacist. Treatment for CF involves many medicines. Pharmacists will help review and educate you about your child's medications.
• Research specialists. You and your child may be interested in research studies or clinical trials that may benefit your child's health. Research specialists coordinate these studies and trials.

Infection Control

More than 25 years of research has given the cystic fibrosis specialists at Riley Children's a better understanding of germs in CF, how germs are spread and the relationship between germs and lung health in CF.

Germs in CF can spread several ways, including:

• Direct contact with infected secretions. Direct contact with infected secretions happens when a person comes into contact with an infected person through kissing, touching, patient care or shaking hands.
• Indirect contact with infected secretions. Indirect contact with infected secretions happens when a person comes into contact with an infected object like an eating utensil, a drinking glass, respiratory equipment or a toy.
• Droplets of infected secretions. Droplets have the ability to travel distances of 6 feet, and germs within droplets can live for hours on surfaces.

The following guidelines help control infection:

• Perform respiratory cultures on a regular basis to watch for the presence of germs
• Promote the Six Foot Rule; keep a 6-foot distance from others with CF to minimize sharing of secretions
• Know your and your child’s culture results
• Bring your own books and/or toys to doctor appointments
• Get your and your child’s flu vaccine every year
• Pay attention to hand hygiene (cleaning hands) and always carry alcohol-based hand sanitizers and tissues

All staff at the Riley Cystic Fibrosis Program wear gowns and gloves. This is to protect you and your child from indirect contact with secretions. Patient rooms are cleaned between each patient, including all chairs, tables and desks. Patients must wear masks upon entering healthcare settings to comply with infection control recommendations.