RxPrep - Cystic Fibrosis

Is cystic fibrosis curable?

No

What is cystic fibrosis?

Hereditary disease caused by a genetic mutation; causes abnormal transport of Cl, Bicarb, Na - leads to thick, viscous secretions

Cystic fibrosis causes thick, viscous secretions. What organs does this affect?

Lungs, pancreas, liver, and intestine

What are the complications of cystic fibrosis?

Difficulty breathing

Lung infections

Digestive complications

Where does the name "cystic fibrosis" come from?

Characteristic scarring (fibrosis) and cyst formation in the pancrease

What is the average lifespan of cystic fibrosis? When are most people diagnosed?

35-40

Most diagnosed by age 2

Newborns in the US are screened for cystic fibrosis. If screening prompts additional testing, what is used?

The "sweat chloride test" confirms the diagnosis. This measures the amount of salt (chloride) in the sweat. Higher in patients with CF

What protein is mutated in CF?

cystic fibrosis transmembrane conductance regulator (CFTR)

What are the classic symptoms of CF?

*- Salty testing skin

- poor growth and weight gain

- thick and sticky mucus production*

- frequent lung infections

- coughing and SOB

- digestive symptoms

- steatorrhea

- malnutrition due to poor absorption of nutrients, including fat soluble vitamins.

If CF is not treated, what can occur?

Malnutrition and failure to thrive

What are the goals of CF therapy?

preventing/treating lung infections

maintain adequate nutrition

optimizing QOL

What is important when giving meds in CF?

The order!!

Giving the inhaled meds in the correct order is critical to maximize absorption and effect.

What is the order of inhaled CF meds?

1st - bronchodilator to open airway

2nd - HyperSal to mobilize mucus and clear airway

3rd - Dornase alfa to thin mucus and clear airway

4th - Chest physiotherapy to mobilize mucus

5th - Inhaled ABX to control airway infection

Do CF patients use oral meds?

Sometimes they receive oral pancreatic enzyme products or azithromycin. These can be given at any time without worrying about the order

What are the most common organisms seen in CF infections early on?

S. aureus

H. flu

Pseudomonas in teens and adults

What are the characteristics of acute pulmonary exacerbations of CF?

increased cough, sputum, change in color, SOB, rapid decline in FEV1

What do you do when a CF patient has a Pseudomonas infection?

2 IV drugs - need synergy and prevent resistance

Aminoglycosides are common, usually at larger-than-normal doses

Why do CF patients need larger doses of ABX?

Altered PK

Need therapeutic concentrations in the lungs

Overcome reduced susceptibilities of the bacteria that chronically colonize the airway

Eventually, airway infections in CF become chronic, and patients will require...

inhaled ABX, particularly with Pseudomonas

What is the schedule for inhaled ABX in chronic lung infection with CF?

28 days on, 28 days

this improves lung function and reduces exacerbations

Why is oral azithromycin used in CF?

It has no direct activity versus Pseudomonas, but it disrupts the biofilm formation

How is HyperSal delivered in CF?

Nebulizer

What is HyperSal?

Hypertonic saline

What is pulmozyme?

Dorase alfa

Can you mix Pulmozyme with other drugs in the nebulizer?

No!

How much Pulmozyme be stored?

The ampules must be stored in the fridge. Protect from light.

How does dornase alfa work?

Breaks down DNA content in sputum to decrease viscosity of the mucus

When is dornase alfa given in order?

After hypertonic saline

When are inhaled bronchodilators (albuterol) given in order?

1st to open the airway for the other drugs

When is hypertonic saline given in order?

After the bronchodilator and before Pulmozyme

When are inhaled ABX given in order?

Very last, after the chest physiotherapy

What are the inhaled ABX given in CF to target Pseudomonas?

TOBI (capsule or soln)

Aztreonam (soln, also comes IV for acute)

What are the side effects of TOBI?

Ototoxicity, tinnitus, voice alteration, mouth/throat pain d/t to inhalation

How is TOBI dosed?

Every 12 hours, but it must be at least 6 hours apart

How should TOBI solution be stored?

Fridge

Ok at room temp for up to 28 days

Do not mix with other drugs in the nebulizer

How should the TOBI Podhaler be stored?

Room temperature.

Should be used with the device it comes with.

Do not swallow the capsules

What are the side effects of aztreonam?

Allergic reactions, bronchospasm, fever, wheezing, cough, chest discomfort

How is aztreoman given?

Every 8 hours, but must be at least 4 hours apart

How is aztreonam stored (inhaled)?

Fridge.

Ok at room temp for 28 days

Don't mix with other drugs in the nebulizer

What is contained in pancrelipase?

Lipase, protease, and amylase

Harvested from porcine pancreatic glands

What is the purpose of the pancreatic enzyme products?

Helps breakdown fat, starches, and protein

Helps CF patients digest food, maintain weight, and improve nutrient absorption

Where are PEP formations designed to dissolve?

In the basic pH of the duodenum

How is PEP dosed?

What is the max dose for all ages?

Based on the lipase component and age

10,000 units/kg/day

How is the dose of PEP adjusted?

every 3-4 days until stools are normalized

What are the side effects of PEP?

ab pain, gas, nausea

What are the warnings associated with PEP?

Colonic strictures (with very high doses)

Mucosal irritation

Are PEP formulations interchangeable?

no

Which PEP comes as a tablet?

Viokace

How is Viokace given?

As a tablet, must be given with a PPI

Other than Viokace, what are the other PEP products?

All are capsules that cannot be crushed or chewed

If DR with microspheres: they can be opened and sprinkled on soft acidic foods (not dairy)

Do not retain contents in the mouth. Swallow and chase with water to avoid mucosal irritation.

How should PEPs be taken in regard to food?

Before or with all meals and snacks

Larger meals may need bigger doses

Use 50% of the mealtime dose with snacks

How should PEP be stored?

Protect from moisture

Dispense in original container

Do not refrigerate

What are some names of PEP products?

Zenpep

Creon

Viokace

What is Ivacaftor? How does it work?

Cystic fibrosis transmembrane conductance regulator modulator (CFTRM)

Increases the time the CFTR channels remain open, increases the amount of CFTR that is delivered to the cell surface

What is special about the CFTR modulators?

Each is approved for specific mutations; genetic testing is required first

What is the most common mutation in the CFTR gene?

Homozygous F508del mutation

(two copies of the same allele)

What is ivacaftor (mono) used for?

Other responsive mutations, NOT homo F508del

The combination ivacaftor products are approved for...

Homo F508del mutation (and some additional ones)

How should ivacaftor-containing products be administered?

with high-fat containing foods

What are some dietary concerns for CF?

High-fat, calorically dense diet is recommended

Vitamin supplement, esp ADEK

May need insulin d/t CF-induced DM

Possible transplant later on