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Cystic Fibrosis

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Cystic Fibrosis (CF) is a rare, genetic disorder that is passed from parents to children. 

It is caused by inheriting two defective (or abnormal) copies of the gene cystic fibrosis transmembrane conductance regulator (CFTR) protein. This mutation makes mucus and other secretions much thicker and difficult to clear. 

As a result, there is a build-up of mucus and thick fluids, which creates blockage and obstructs vital organs. CF is a multi-systemic disease that affects the respiratory tract, pancreas, digestive system, and liver. 

Doctors can screen for and diagnose CF at birth with a positive newborn screening test. Diagnosis is further confirmed with a pilocarpine-induced sweat test and genetic testing. 

More than 30,000 people in the U.S. are living with cystic fibrosis and around 1,000 people are diagnosed with CF each year.

Pharmacist in a white coat holding a clipboard and a medication box while checking products on pharmacy shelves.

Related Symptoms and Health Concerns

People experience different CF symptoms because CF affects multiple organs:

  • Lungs: chronic inflammation and obstruction, lung infections, and trouble breathing. Common symptoms include coughing up thick mucus, wheezing, difficulty breathing, frequent lung infections, and airway inflammation.
  • Pancreas: low enzyme and vitamin levels needed for absorbing nutrients. Damage can lead to CF-related diabetes.
  • Liver: blocked bile ducts, which can cause damage to the liver.
  • GI tract: constipation or obstruction in the intestines. Symptoms include abdominal pain, constipation, malnutrition, or inadequate weight gain.
  • Miscellaneous: fertility problems, urinary incontinence, and increased risk of GI-related cancers

Treatment

Cystic Fibrosis is treatable, but it does not have a cure. Cystic Fibrosis is more manageable with newer drugs coming to market.

  • Oral medications:
    • Pancreatic enzymes to increase absorption of necessary nutrients.
    • CFTR modulators that target and improve function of defective protein.
    • Anti-inflammatory to reduce inflammation.
    • Antibiotics to help treat or prevent infection and inflammation.
    • Nutritional supplements with high dose fat-soluble vitamins.
  • Air clearance: Loosens mucus build up in the lungs.
  • Inhaled medications: Thins mucus and opens airways.

Treatment Goals

The primary goal of cystic fibrosis treatment is to reduce symptom severity, slow disease progression, and increase survival by…

Maintaining lung function

Preventing and treating lung infections

Loosening and removing inflammation-causing mucus from the lungs

Preventing and treating of blockages in the intestines

Promoting adequate nutrition to maintain growth and appropriate BMI

Encouraging adequate hydration

Improving or maintaining quality of life

Increasing survival and life expectancy

Maintaining optimal therapy adherence

Preventing, minimizing, and managing side effects

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Strategies to Achieve
Treatment Goals

Adhere to drug therapy
Improve diet and nutrition to maintain growth and appropriate BMI
Prevent, minimize, and manage side effects
Prevent and treat lung infections
Minimize disruption to daily life due to CF symptoms and treatment burden

Additional Resources

Patient Resources

Patient Guide

Cayston Patient Authorization Form

Arikayce Enrollment Form

Cystic Fibrosis Enrollment Form

Vertex GPS Enrollment Form

Additional Resources

Sources

  1. Lahiri, Thomas, et al. “Clinical Practice Guidelines From the Cystic Fibrosis Foundation for Preschoolers With Cystic Fibrosis.” American Academy of Pediatrics, American Academy of Pediatrics, 23 Mar. 2016, pediatrics.aappublications.org/content/early/2016/03/22/peds.2015-1784.
  2. Mogayzel, Peter J, et al. “Cystic Fibrosis Pulmonary Guidelines Chronic Medications for Maintenance of Lung Health.” American Journal of Respiratory and Critical Care Medicine, 3 Jan. 2013, www.atsjournals.org/doi/full/10.1164/rccm.201207-1160OE.
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Severe Asthma

Disease State Overview

Asthma is a long-term condition that affects the airways in your lungs.  These airways can become inflamed and narrowed.  This makes it harder to move air in and out of the lungs.  It can affect people of all ages, although it often begins in childhood.  Certain things – called triggers – can worsen asthma symptoms.  When symptoms suddenly get worse, you may have an asthma attack.

There are several types of asthma, and each may require different treatment or management strategies.  These include:

  • Allergic asthma
  • Aspirin-induced asthma
  • Cough-variant asthma
  • Exercise-induced asthma
  • Nighttime (or nocturnal) asthma
  • Steroid-resistant asthma
  • Occupational asthma (related to workplace exposure)

Experts have started to recognize the impact of inflammation in asthma.  Four biological pathways of inflammation have been identified: eosinophilic, neutrophilic, mixed eosinophilic and neutrophilic, and non-inflammatory.  

When asthma does not improve with long-term controller medications, it may be a sign of severe asthma.  There are 2 main types of severe asthma: Type-2 inflammation and Non-Type-2 inflammation.  Those patients with Type-2 severe asthma respond well to biologic medications.  These treatments target specific parts of the immune system and may be obtained at your specialty pharmacy.

Related Health Symptoms and Health Concerns

Asthma can cause a variety of breathing problems.  Common symptoms include:

  • Wheezing (a whistling sound when you breathe)
  • Coughing
  • Shortness of breath
  • A feeling of tightness in your chest

For some people, an asthma attack may begin with a cough that doesn’t go away.  Your chest may start to feel tight — some people describe it as having the air “sucked out” of them.  You may also begin to wheeze or feel lightheaded. 

Having and following your asthma action plan is very important.  It helps to know what steps to take when symptoms start, when to use your rescue medication, and when to seek emergency care. 

Call 9-1-1 right away if you are struggling to breathe, your symptoms are getting worse quickly or your rescue inhaler is not helping.  

Treatment

While there is no cure for asthma, there are many treatments that can help you manage your symptoms and live a healthy, active life.  Treatment usually includes monitoring your breathing, avoiding triggers, and using medications to both prevent and treat symptoms.

Most people use a combination of medications to keep their asthma under control. You may have a quick relief (rescue) medication to help stop symptoms and a long-term control medication to help prevent symptoms from happening in the first place.

The types of medications include:

  • Bronchodilators
  • Corticosteroids (steroids)
  • Anticholinergies
  • Biologics
  • Antibiotics

Treatment Goals

The goals of treating asthma include:

  • Improved breathing
  • Reduce airway inflammation
  • Reduce asthma symptoms
  • Improve physical function and quality of life
  • Maintaining optimal therapy adherence
  • Preventing, minimizing, and managing side effects of therapy

Strategies to Achieve Treatment Goals

  • Have an asthma action plan
  • Adherence to therapy
  • Monitoring and follow-up with physician
  • Know and reduce asthma triggers
  • Reducing the risk of additional lung damage through smoking cessation
  • Immunization against flu and pneumonia
  • Appropriate management of other underlying diseases such as allergies, high blood pressure, anxiety, etc.

Additional resources

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Rheumatology – Chronic Inflammatory Disease 

Rheumatology – Chronic Inflammatory Disease 

Ankylosing Spondylitis (AS), Gout, Psoriatic Arthritis (PsA), Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE), Systemic Juvenile Idiopathic Arthritis (sJIA)

Disease State Overview

Chronic inflammatory diseases cause long-lasting inflammation in the body. This inflammation can damage healthy cells and tissues over time.  Many areas of the body can be affected including, but not limited to, the digestive system, joints, skin, and respiratory system. Researchers believe that these conditions may be related to an overactive immune system, but more research is needed to understand what triggers this disruption of the immune system. 

The term rheumatism is used to describe many conditions that cause inflammation in the joints, muscles, and connective tissues.  It includes a wide range of diseases such as ankylosing spondylitis (AS), psoriatic arthritis (PsA), rheumatoid arthritis (RA), and systemic lupus erythematosus (SLE).

The most common symptom of rheumatologic inflammatory diseases is joint pain. Joints may also feel warm, tender, stiff, or swollen. Other symptoms may include feeling unusually tired, having low energy, occasional fevers, loss of appetite, skin rashes and vision changes.  In most cases, these conditions can be cyclical.  There are periods where symptoms are worse, called flares.  When symptoms ease and feel better, it is called remission.

Related Symptoms and Health Concerns

If left untreated,

  • Inflammation can lead to permanent joint damage or deformity
  • physical disability, making everyday activities and work more difficult
  • negative impact on mental and social well-being
  • patients may be at an increased risk of heart, lung and kidney problems
  • patients may be at an increased risk of uveitis, which can cause eye pain, sensitivy to light, and blurry vision

Treatment

Most chronic inflammatory diseases have no cure. Lifestyle changes and early treatment may help manage and prevent complications. Treatment largely depends on the severity of symptoms. Research shows that people with rheumatoid arthritis who are diagnosed early and treated aggressively do better over the long-term.

Topical creams or ointments, as well as nonsteroidal anti-inflammatory drugs (NSAIDs), may help with pain and reduce inflammation. If symptoms are widespread, more aggressive treatment may be required. Depending on the diagnosis, treatment options may include immunosuppressive therapies, such as steroids, disease-modifying antirheumatic drugs (DMARDs), or immunomodulators.  Immunomodulators may help decrease the body’s hyperactive immune response in the skin, joints and other organs.  Suppressing inflammation not only offers relief from common symptoms, but it also allows the tissues to heal.

Specialty medications used to treat chronic inflammatory rheumatologic conditions include, but are not limited to:

  • Oral immunomodulators
  • Injectable immunomodulator therapies

Treatment Goals

The primary goals of treating inflammatory rheumatic conditions are to:

  • Reduce or eliminate symptomatic disease activity
  • Manage exacerbating conditions, such as depression, anxiety, or sleep loss due to disease symptoms
  • Prevent flares (maintain remission)
  • Reduce risk of long-term complications, such as bone deformities
  • Maintain as normal a lifestyle as possible.

Strategies to Achieve Treatment Goals

  • Adhere to therapy
  • Keep appointments, and follow-up with physician
  • Report side effects or unmanaged symptoms to your pharmacist, or healthcare team
  • Avoid known triggers, such as cigarette smoke, heavy alcohol consumption, and food sensitivities
  • Practice stress management and relaxation techniques
  • Participate in regular exercise and physical activity programs
  • Eat a healthy diet, and maintain a healthy weight

Sources

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Atopic Dermatitis (AD), Chronic Idiopathic Urticaria, Pemphigus Vulgaris, Plaque Psoriasis

Atopic Dermatitis (AD), Chronic Idiopathic Urticaria, Pemphigus Vulgaris, Plaque Psoriasis

Disease State Overview

Chronic inflammatory diseases cause ongoing inflammation in the body, damaging healthy cells of the digestive system, joints, skin, respiratory system, and other organs. Researchers believe that a hyperactive immune response may play a role in these conditions. More research is required to determine what triggers this disruption of the immune system.

Atopic Dermatitis (AD), Chronic Idiopathic Urticaria, Pemphigus Vulgaris, and Plaque Psoriasis are inflammatory diagnoses that mainly affect the skin. Symptoms may include red, dry patches of skin, burning, itching, swelling, blistering, pain and/or sensitivity. In most cases, there are periods of time when the disease is worse, called flares. Then there are periods when symptoms improve, called remissions. Chronic inflammatory dermatologic conditions are not contagious.

Related Symptoms and Health Concerns

If left untreated,

  • rashes on the body may break open, weep fluid and bleed when scratched, making skin vulnerable to infection;
  • skin  may become dry and discolored, and repeated scratching  may cause thickening and hardening;
  • constant itching or pain  may  make it difficult to concentrate, taking a toll on time, energy and money;
  • unmanaged symptoms may  result in poor sleep, causing daytime lethargy, mental status changes and irritation.

Treatment

Most chronic inflammatory diseases have no cure. Lifestyle changes and early treatment may help manage and prevent issues.

Treatment largely depends on the severity of symptoms, as well as other diseases present.  Mild cases of skin inflammation may be treated with topical creams or ointments.

If symptoms are widespread, or joint pain is present along with skin irritation, more aggressive treatment may be required. Depending on the diagnosis, treatment options may include immunosuppressive therapies. These are steroids, methotrexate, or cyclosporine, or immunomodulators. Immunomodulators help lower the body’s hyperactive immune response in the skin and other organs. These are used for moderate to severe disease. 

Suppressing inflammation offers relief from common symptoms. It also allows the skin tissue to heal.

Specialty medications used to treat chronic inflammatory dermatologic conditions include, but are not limited to:

  • Oral immunomodulators
  • Injectable immunomodulator therapies

Treatment Goals

The primary goals of treating inflammatory dermatologic conditions are to:

  • Reduce or eliminate symptomatic disease activity
  • Manage exacerbating conditions, such as depression, anxiety, or sleep loss due to disease symptoms
  • Prevent secondary infections, which may occur as a result of scratching or blistering
  • Prevent flares (maintain remission)
  • Reduce risk of long-term complications, such as involvement of the joints

Strategies to Achieve Treatment Goals

  • Adhere to prescribed therapy
  • Keep appointments, and follow-up with physician
  • Report side effects or unmanaged symptoms to your pharmacist, or healthcare team
  • Avoid known triggers, or irritants, such as: harsh soaps/detergents, perfumes, cosmetics, cigarette smoke, heavy alcohol consumption, and food allergies
  • Use appropriate skin care, such as regular use of hypoallergenic moisturizers for dry skin
  • Practice stress management and relaxation techniques
  • Eat a healthy diet

Additional Resources

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