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Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder that particularly affects the digestive system and makes kids who have it more vulnerable to repeated lung infections. Now, thanks to high-tech medical advances in drug therapy and genetics, children born with CF can look forward to longer and more comfortable lives. In the last 10 years, research into all aspects of CF has helped doctors to understand the illness better and to develop new therapies. Ongoing research may someday lead to a cure.

What Is Cystic Fibrosis?

Currently affecting more than 30,000 children and young adults in the United States, cystic fibrosis makes kids sick by disrupting the normal function of epithelial cells — cells that make up the sweat glands in the skin and that also line passageways inside the lungs, liver, pancreas, and digestive and reproductive systems.

The inherited CF gene directs the body's epithelial cells to produce a defective form of a protein called CFTR (or cystic fibrosis transmembrane conductance regulator) found in cells that line the lungs, digestive tract, sweat glands, and genitourinary system. When the CFTR protein is defective, epithelial cells can't regulate the way chloride (part of the salt called sodium chloride) passes across cell membranes. This disrupts the essential balance of salt and water needed to maintain a normal thin coating of fluid and mucus inside the lungs, pancreas, and passageways in other organs. The mucus becomes thick, sticky, and hard to move.

Normally, mucus in the lungs traps germs, which are then cleared out of the lungs. But in CF, the thick, sticky mucus and the germs it has trapped remain in the lungs, which become infected.

In the pancreas, thick mucus blocks the channels that would normally carry important enzymes to the intestines to digest foods. When this happens, the body can't process or absorb nutrients properly, especially fats. Kids with CF have problems gaining weight, even with a normal diet and a good appetite.

A Family's Risk for CF

Humans have 23 pairs of chromosomes made of the inherited genetic testing, which can be done in kids both before and after birth and in adults thinking about starting or enlarging their families.

Of all ethnic groups, Caucasians have the highest inherited risk for CF, and Asian Americans have the lowest. In the United States today, about 1 of every 3,600 Caucasian children is born with CF. This compares with 1 of every 17,000 African Americans and only 1 of every 90,000 Asian Americans. Although the chances of inherited risk may vary, CF has been described in every geographic area of the world among every ethnic population.

Scientists don't know exactly why the CF gene evolved in humans, but they have some evidence to show that it helped to protect earlier generations from the bacteria that cause cholera, a severe intestinal infection.

How CF Affects Kids

The diagnosis of CF is being made earlier and earlier, usually in infancy. However, about 15% of those with CF are diagnosed later in life (even adulthood). Symptoms usually center around the lungs and digestive organs and can be more or less severe.

A few kids with CF begin having symptoms at birth. Some are born with a condition called meconium ileus. Although all newborns have meconium — the thick, dark, putty-like substance that usually passes from the rectum in the first few days of life — in CF, the meconium can be too thick and sticky to pass and can completely block the intestines.

More commonly, though, babies born with CF don't gain weight as expected. They

Cystic fibrosis is the most common cause of pancreatic insufficiency in children, but a condition called Shwachman-Diamond Syndrome (SDS) is the second most common cause. SDS is a genetic condition that causes a reduced ability to digest food because digestive enzymes don't work properly. Some of the symptoms of SDS are similar to those of CF, so it may be confused with cystic fibrosis. However, in children with SDS, the asthma-like symptoms. As CF symptoms progress, the child may develop a chronic cough that produces globs of thick, heavy, discolored mucus. They may also suffer from repeated lung infections.

As chronic infections reduce lung function, the ability to breathe often decreases. A person with CF may eventually begin to feel short of breath, even when resting. Despite aggressive medical therapy, lung disease develops in nearly all patients with CF and is a common cause of disability and shortened life span.

Identifying a Child With CF

By performing genetic tests during pregnancy, parents can now learn whether their unborn children may have CF. But even when genetic tests confirm CF, there's still no way to predict beforehand whether a specific child's CF symptoms will be severe or mild. Genetic testing can also be done on a child after birth, and can be performed on parents, siblings, and other relatives who are considering having a family.

After birth, the standard diagnostic test for CF is called the sweat test — an accurate, safe, and painless way to diagnose CF. In the sweat test, a small electric current is used to carry the chemical pilocarpine into the skin of the forearm. This stimulates sweat glands in the area to produce sweat. Over a period of 30 to 60 minutes, sweat is collected on filter paper or gauze and tested for chloride.

To diagnose CF, two sweat tests are generally performed in a lab accredited by the Cystic Fibrosis Foundation. A child must have a sweat chloride result of greater than 60 on two separate sweat tests to make the diagnosis of CF. Sweat test normal values for infants are lower.

Several other tests are standard parts of the routine care used to monitor a child's CF:

  • chest X-rays
  • blood tests to evaluate nutritional status
  • bacterial studies that confirm the growth of Pseudomonas aeruginosa, Staphylococcus aureus, or Haemophilous influenza bacteria in the lungs (these bacteria are common in CF but may not affect healthy people exposed to CF)
  • pulmonary function tests (PFTs) to measure the effects of CF on breathing (PFTs are done as soon as the child is old enough to be able to cooperate in the testing procedure; infant PFTs are currently being studied)

Treating Kids With CF

When kids are first diagnosed with CF, they may or may not have to spend some time in the hospital, depending on their condition. If they do, they'll have diagnostic tests, especially baseline measurements of their breathing (lung function) and a nutritional assessment. Before they leave, their doctors will make sure that their lungs are clear and that they've started a diet with digestive enzymes and vitamins that will help them to gain weight normally. Afterward, they'll probably see their doctor for follow-up visits at least once every 1 to 3 months.

The basic daily care program varies from child to child, but usually includes pulmonary therapy (treatments to maintain lung function) and gene therapy, and test trials are underway in at least nine different U.S. medical centers and other centers around the world. Another new therapy, called protein repair therapy, aims at repairing the defective CFTR protein. Numerous medications, including a spice called curcumin, are also being tested.

Caring for a child with CF can be tough at times, but parents need not feel alone. Doctors can usually refer them to a local support group linked to the Cystic Fibrosis Foundation.

Reviewed by:

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Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder that particularly affects the digestive system and makes kids who have it more vulnerable to repeated lung infections. Now, thanks to high-tech medical advances in drug therapy and genetics, children born with CF can look forward to longer and more comfortable lives. In the last 10 years, research into all aspects of CF has helped doctors to understand the illness better and to develop new therapies. Ongoing research may someday lead to a cure.

What Is Cystic Fibrosis?

Currently affecting more than 30,000 children and young adults in the United States, cystic fibrosis makes kids sick by disrupting the normal function of epithelial cells — cells that make up the sweat glands in the skin and that also line passageways inside the lungs, liver, pancreas, and digestive and reproductive systems.

The inherited CF gene directs the body's epithelial cells to produce a defective form of a protein called CFTR (or cystic fibrosis transmembrane conductance regulator) found in cells that line the lungs, digestive tract, sweat glands, and genitourinary system. When the CFTR protein is defective, epithelial cells can't regulate the way chloride (part of the salt called sodium chloride) passes across cell membranes. This disrupts the essential balance of salt and water needed to maintain a normal thin coating of fluid and mucus inside the lungs, pancreas, and passageways in other organs. The mucus becomes thick, sticky, and hard to move.

Normally, mucus in the lungs traps germs, which are then cleared out of the lungs. But in CF, the thick, sticky mucus and the germs it has trapped remain in the lungs, which become infected.

In the pancreas, thick mucus blocks the channels that would normally carry important enzymes to the intestines to digest foods. When this happens, the body can't process or absorb nutrients properly, especially fats. Kids with CF have problems gaining weight, even with a normal diet and a good appetite.

A Family's Risk for CF

Humans have 23 pairs of chromosomes made of the inherited genetic testing, which can be done in kids both before and after birth and in adults thinking about starting or enlarging their families.

Of all ethnic groups, Caucasians have the highest inherited risk for CF, and Asian Americans have the lowest. In the United States today, about 1 of every 3,600 Caucasian children is born with CF. This compares with 1 of every 17,000 African Americans and only 1 of every 90,000 Asian Americans. Although the chances of inherited risk may vary, CF has been described in every geographic area of the world among every ethnic population.

Scientists don't know exactly why the CF gene evolved in humans, but they have some evidence to show that it helped to protect earlier generations from the bacteria that cause cholera, a severe intestinal infection.

How CF Affects Kids

The diagnosis of CF is being made earlier and earlier, usually in infancy. However, about 15% of those with CF are diagnosed later in life (even adulthood). Symptoms usually center around the lungs and digestive organs and can be more or less severe.

A few kids with CF begin having symptoms at birth. Some are born with a condition called meconium ileus. Although all newborns have meconium — the thick, dark, putty-like substance that usually passes from the rectum in the first few days of life — in CF, the meconium can be too thick and sticky to pass and can completely block the intestines.

More commonly, though, babies born with CF don't gain weight as expected. They

Cystic fibrosis is the most common cause of pancreatic insufficiency in children, but a condition called Shwachman-Diamond Syndrome (SDS) is the second most common cause. SDS is a genetic condition that causes a reduced ability to digest food because digestive enzymes don't work properly. Some of the symptoms of SDS are similar to those of CF, so it may be confused with cystic fibrosis. However, in children with SDS, the asthma-like symptoms. As CF symptoms progress, the child may develop a chronic cough that produces globs of thick, heavy, discolored mucus. They may also suffer from repeated lung infections.

As chronic infections reduce lung function, the ability to breathe often decreases. A person with CF may eventually begin to feel short of breath, even when resting. Despite aggressive medical therapy, lung disease develops in nearly all patients with CF and is a common cause of disability and shortened life span.

Identifying a Child With CF

By performing genetic tests during pregnancy, parents can now learn whether their unborn children may have CF. But even when genetic tests confirm CF, there's still no way to predict beforehand whether a specific child's CF symptoms will be severe or mild. Genetic testing can also be done on a child after birth, and can be performed on parents, siblings, and other relatives who are considering having a family.

After birth, the standard diagnostic test for CF is called the sweat test — an accurate, safe, and painless way to diagnose CF. In the sweat test, a small electric current is used to carry the chemical pilocarpine into the skin of the forearm. This stimulates sweat glands in the area to produce sweat. Over a period of 30 to 60 minutes, sweat is collected on filter paper or gauze and tested for chloride.

To diagnose CF, two sweat tests are generally performed in a lab accredited by the Cystic Fibrosis Foundation. A child must have a sweat chloride result of greater than 60 on two separate sweat tests to make the diagnosis of CF. Sweat test normal values for infants are lower.

Several other tests are standard parts of the routine care used to monitor a child's CF:

  • chest X-rays
  • blood tests to evaluate nutritional status
  • bacterial studies that confirm the growth of Pseudomonas aeruginosa, Staphylococcus aureus, or Haemophilous influenza bacteria in the lungs (these bacteria are common in CF but may not affect healthy people exposed to CF)
  • pulmonary function tests (PFTs) to measure the effects of CF on breathing (PFTs are done as soon as the child is old enough to be able to cooperate in the testing procedure; infant PFTs are currently being studied)

Treating Kids With CF

When kids are first diagnosed with CF, they may or may not have to spend some time in the hospital, depending on their condition. If they do, they'll have diagnostic tests, especially baseline measurements of their breathing (lung function) and a nutritional assessment. Before they leave, their doctors will make sure that their lungs are clear and that they've started a diet with digestive enzymes and vitamins that will help them to gain weight normally. Afterward, they'll probably see their doctor for follow-up visits at least once every 1 to 3 months.

The basic daily care program varies from child to child, but usually includes pulmonary therapy (treatments to maintain lung function) and gene therapy, and test trials are underway in at least nine different U.S. medical centers and other centers around the world. Another new therapy, called protein repair therapy, aims at repairing the defective CFTR protein. Numerous medications, including a spice called curcumin, are also being tested.

Caring for a child with CF can be tough at times, but parents need not feel alone. Doctors can usually refer them to a local support group linked to the Cystic Fibrosis Foundation.

Reviewed by:

Note: All information is for educational purposes only. For specific medical advice, diagnoses, and treatment, consult your doctor.

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