Genetic Disorder Cystic Fibrosis

Description of the disease

Cystic fibrosis is a genetic disorder that is passed from parent to offspring. It is a hereditary, autosomal and recessive medical complication. Its main feature is the accumulation of more dense and sticky secretions in the lungs, digestive tract, and other areas of the body (Wilschanski et al., 2011). In fact, the clinical presentation of the disease is evident in various organs of the body as discussed in this paper.

Pathophysiology of Cystic Fibrosis

The CFTR channels may be affected in terms of functionality. In most cases, the cell surface of the CFTR gene is mutated. Eventually, the condition leads to abnormal chloride transport.

Airway

Most mortality cases are caused by the inflammation of the airways owing to the onset of pulmonary disease. When the size of the airway is interfered with, bacteria cannot be easily washed away from the airway passage.

Reproductive system

The pathophysiology of this disease is also evident in the human reproductive system. Vas deferens may be absent in the reproductive system of males and consequently usher in congenital bilateral conditions. It culminates into atrophy or blockage of vas deferens.

GI tract

Poor absorption of essential nutrients throughout the entire digestive system is one of the common GI tract abnormalities. Late in life, a condition known as distal intestinal obstruction syndrome may be experienced. In the initial years of the GI tract infection, reduced production of water and chloride in the digestive system is common (Sly et al., 2009).

Liver

Liver disease coupled with cirrhosis may develop among patients diagnosed with CF even though it is not a dominant condition. Bile can also become increasingly viscous as a result of an affected liver.

Clinical presentation

Most children with cystic fibrosis are diagnosed until the age of two. However, minimal cases are diagnosed at the age of 18 years or more. The affected persons may not grossly suffer from the disease at the initial stage of development (Giddens, 2013).

Family history is a major risk factor for cystic fibrosis since the disease is hereditary. The rate of infection is also determined by race. For instance, Caucasian people are highly vulnerable to the disease

In newborns, it is possible to observe bowel blockage, trouble gaining weight, coughing up secretions, and dehydration.

Over the years, a patient may experience weight loss, progressive malnutrition, chronic cough with a lot of secretion, sinusitis, chronic formation of nasal polyps, liver disease (biliary cirrhosis ), diabetes, respiratory infections, and infertility.

Clinical management

Diagnosis, tests, and eventually seeking medical help are highly recommended as part of the clinical management of Cystic Fibrosis. It is crucial to obtain immediate medical attention after being diagnosed with the disease. Regular screening to monitor the general body condition is prudent (Tuchman, Schwartz, Sawicki & Britto, 2010). Severe sputum of blood or difficulty in breathing is among the warning signs that individuals should take seriously.

It is vital to seek expert assistance if an adult or a child demonstrates the following signs:

  • Fever, cough increased, changes in saliva or blood in the saliva, loss of appetite, or other signs of pneumonia.
  • Marked weight loss
  • Bowels tend to move quite often
  • Bad smell generated from stool
  • The abdomen may be swollen

Patients are also advised to seek medical consultation with experts who handle this type of medical condition. Specialists who usually diagnose cystic fibrosis include:

  • General practitioners
  • Medical genetics
  • Gastroenterologists
  • Pulmonologists

It is pertinent to be prepared for the medical examination process. This implies that before going for a consultation, it is proper to be armed with some bit of information. Medical history, including other conditions of a patient and medications or supplements that are taken regularly, should be known by the medical personnel in charge. If possible, patients should be accompanied when going for consultation or during the diagnosis.

A series of questions may be asked by a medical specialist. These include:

  • When did the symptoms first begin?
  • What is the intensity of the symptoms?
  • Are the symptoms occasional or frequent?
  • Does the patient experience some fever, fatigue, loss of appetite, pain, or other symptoms that may be associated with pneumonia? During a medical checkup, patients should ask questions from the concerned doctors

Diagnosis is also part of the clinical management of Cystic Fibrosis.

To make the diagnosis, the doctor often carries out some specific tests, depending on the patient’s age:

  • Sweat test.
  • Newborn screening is usually done routinely in hospitals to identify three congenital diseases.
  • Genetic testing identifies only the most frequent types of disease but often helps diagnose the vast majority of cases of cystic fibrosis (Moran et al., 2009).

Other tests that identify problems that may be related to cystic fibrosis include:

  • Computed tomography or X-ray
  • fecal test
  • Function test of the lungs
  • Measurement of pancreatic function
  • Secretin stimulation test
  • Trypsin and chymotrypsin in the feces
  • Upper gastrointestinal series (GI) tract and small intestine

The sooner we find out about cystic fibrosis, the better the quality of life and the patient will live longer – since there is no cure for the disease.

Treatment of Cystic Fibrosis

Treatment for cystic fibrosis is multidisciplinary as it involves physicians from different specialties. The main goal of treatment is to improve the quality of life of the patient. In young children, the focus is on proper nutrition (Flume et al., 2009). The treatment for lung problems includes:

  • Antibiotics to prevent and treat infections in the lungs
  • Inhaled medicines to help open the airways
  • Enzyme replacement therapy
  • High concentration of salt solutions
  • Lung transplantation is an option in some instances, particularly when the case is advanced.

Treatment of intestinal and nutritional problems may include:

  • A special diet rich in protein and calories for patients who are above 18 years.
  • The use of pancreatic enzymes usually helps in the absorption of fats and protein (Khan et al., 2012).
  • Recommending vitamin supplements such as A, D, E and K.

Home care can help speed recovery and may include:

  • Keeping away from smoke, dust, dirt, and other household chemicals.
  • Clearing or removing mucus and other secretions that may block airways. Uptake of plenty of fluids.
  • Regular exercise such as through swimming, running and cycling.

References

Flume, P. A., Mogayzel Jr, P. J., Robinson, K. A., Goss, C. H., Rosenblatt, R. L., Kuhn, R. J., & Marshall, B. C. (2009). Cystic fibrosis pulmonary guidelines: treatment of pulmonary exacerbations. American journal of respiratory and critical care medicine, 180(9), 802-808.

Giddens, J. F. (2013). Concepts for nursing Practice. St Louis, MO: Elsevier.

Khan, T. Z., Wagener, J. S., Bost, T., Martinez, J., Accurso, F. J., & Riches, D. W. (2012). Early pulmonary inflammation in infants with cystic fibrosis. American Journal of Respiratory and Critical Care Medicine, 151(4).

Moran, A., Dunitz, J., Nathan, B., Saeed, A., Holme, B., & Thomas, W. (2009). Cystic fibrosis–related diabetes: current trends in prevalence, incidence, and mortality. Diabetes care, 32(9), 1626-1631.

Sly, P. D., Brennan, S., Gangell, C., de Klerk, N., Murray, C., Mott, L.,… & Ranganathan, S. C. (2009). Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. American journal of respiratory and critical care medicine, 180(2), 146-152.

Tuchman, L. K., Schwartz, L. A., Sawicki, G. S., & Britto, M. T. (2010). Cystic fibrosis and transition to adult medical care. Pediatrics, 125(3), 566-573.

Wilschanski, M., Miller, L. L., Shoseyov, D., Blau, H., Rivlin, J., Aviram, M.,… & Kerem, E. (2011). Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. European Respiratory Journal, 38(1), 59-69.

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