Cystic Fibrosis (CF)

Altered physiology

Clinical manifestations

Multi-system alterations in function

Treatment modalities

an autosomal recessive disorder of electrolytes and subsequently water transport that affects certain epithelial cells

most commonly affects Caucasians of European descent

most common lethal inherited disease in the group it affects

leads to the production of excessive and thick exocrine secretions which leads to

infection

inflammation

obstruction

caused by a mutation of the CF gene located on the long arm of chromosome 7

required on both inherited CFTR alleles

impaired electrolyte transportation across the epithelial cells on mucosal surfaces leads to thick secretions in

pancreas

gastrointestinal tract

respiratory tract

sweat glands

other exocrine tissues

most commonly associated with the mucous plugging, inflammation and infection in the lungs

mucous plugging is the formation of tenacious secretions which lead to

permanent lung damage

impairment of local immune function from infection

Presenting clinical manifestations depend on

Symptom severity

Rate of disease progression

Age at diagnosis

Associated with tenacious secretions leading to respiratory and gastrointestinal impairment

Clinical Manifestations

Gastrointestinal and Pancreas

Sweat glands

Respiratory

chronic cough accompanied with mucoid or purulent sputum

tachypnea

recurrent respiratory infections

recurrent wheezing or crackles

hemoptysis

dyspnea

chest pain

chest retractions

barrel chest

recurrent sinusitis

cyanosis

increased frequency of large, greasy, malodorous stools

distention

abdominal pain

poor absorption of fat soluble vitamins

weight loss

sweat abnormalities

excessive salt depletion

loss of appetite

Other

In males

undescended testicles

sterile

Sweat Glands

Respiratory

Pancreas and Gastrointestinal

Enzymes that are important for the breakdown of fats, proteins, and carbohydrates get blocked into tubes due to mucus obstruction

causes

inflammation

build up of enzymes

scarring

this scarring can lead to CF related diabetes

The CF gene that controls the movement of chloride and water is faulty

leads to

more sweat

high levels of chloride in sweat

not enough absorption of fats, proteins and carbohydrates

abnormal electrolyte transport causes cells in the respiratory system to absorb too much sodium and water

causes

secretions in the lungs to become very thick and hard to move

There is no cure for CF but treatment can ease symptoms

Treatments types

Goals of Treatment

Treating and preventing intestinal blockage

Removing and loosening mucus from the lungs

Providing adequate nutrition

Preventing and controlling infections that occur in the lungs

Medications

medications that target gene mutations

antibiotics

anti-inflammatory drugs

mucus-thinning drugs

bronchodilators

oral pancreatic enzymes

stool softeners

acid-reducing medications

drugs for diabetes or liver disease when needed

help improve function of the faulty CFTR protein and may improve lung function and weight, and reduce the amount of salt in sweat

Airway clearance techniques

Vest therapy

can relieve mucus obstruction

help to reduce infection and inflammation in airways

makes it easier to cough up mucus

other techniques to loosen and remove mucus

breathing and coughing techniques

tube that you blow into

clapping with cupped hands on the front and back of the chest

Pulmonary rehabilitation

may include

breathing techniques that may help loosen mucus and improve breathing

nutritional counseling

Physical exercise that may improve condition

counseling and support

education about condition

Surgical

Noninvasive ventilation

Feeding tube

Oxygen therapy

Bowel surgery

Nasal and sinus surgery

removal of nasal polyps that obstruct breathing

treat recurrent sinusitis

nasal polyps

improves oxygen intake to prevent high blood pressure in the lungs

a nose or mouth mask to provide positive pressure in the airways and lungs when breathing in

can increase air exchange in the lungs and decrease the work of breathing

helps with airway clearance

Lung transplant

Liver transplant

delivers extra nutrition

tube is inserted into the nose and guided into the stomach or tube may be surgically implanted in the abdomen

removes blockage in bowel

may be necessary if patient has

severe breathing problems

life-threatening lung complications

increasing resistance to antibiotics for lung infections

used for those with severe cystic-fibrosis liver disease