There are a variety of medicines that are used to treat children with liver disease. Sometimes, these medicines provide specific treatment for the underlying liver disease. Unfortunately, this is not common. Vitamins or minerals, which may not be absorbed well due to poor liver function, may be supplemented. Some medicines are used to prevent complications of chronic liver disease. Others are used to treat the symptoms of chronic liver disease.
Antibiotics are frequently used in patients with liver disease. They are sometimes used preventively, when a patient has recurrent infection of the bile ducts (cholangitis), or recurrent infection in ascites called spontaneous bacterial peritonitis. Intravenous antibiotics can be used to treat serious infections which complicate the course of chronic liver disease. These might include severe cholangitis, bacteremia (bacteria in the bloodstream) or spontaneous bacterial peritonitis.
Drugs for Itching
These medicines are summarized in the section of this book on itching.
- Antihistamines hydroxyzine (Atarax) and diphenhydramine (Benadryl) are often the first drugs that are used. These may help the itching, usually by sedating the patient.
- Rifampin is an antibiotic which for some reason can also help itching. The patient should be monitored carefully since this drug can be hard on the liver.
- Ursodeoxycholic acid (Actigall or URSO) may also help itching by promoting bile flow.
- Questran is a material called a resin. This gritty substance binds bile salts in the stool, stimulating flow of bile from the liver. It should not be taken at the same time as certain other medicines such as URSO.
- Naloxone is an "anti-narcotic". It has been used in desperate situations to help itching. It can harm the liver.
Drugs for Metabolic Diseases
- NTBC is a drug used to treat a metabolic liver disease called tyrosinemia. This drug, which was developed in Sweden, can reverse the liver failure that can be seen in infants with tyrosinemia. The drug does cause accumulations of tyrosine in the bloodstream. Patients are kept on a low protein diet so that tyrosine crystals do not accumulate in the eyes. Growth and development must be monitored carefully while on this drug.
- Penicillamine is a commonly used medicine for Wilson's disease. This drug chelates or binds with copper and leads to its secretion from the body. Patients on penicillamine are monitored since this drug can sometimes be tough on the kidneys or bone marrow. It can also affect wound healing. It is important to take penicillamine consistently, since patients with Wilson's disease who stop it have developed liver failure.
- Zinc therapy prevents the body from absorbing copper in Wilson's.
- Trientine, which removes copper from the body, is a third drug for Wilson's.
Drugs that Suppress the Immune System
- Prednisone is a corticosteroid drug which is used to treat autoimmune hepatitis as well as to prevent rejection in liver transplant patients. Corticosteroids are normally made by the body in small amounts. Physicians may give these drugs in large amounts to suppress an overactive immune system. Prednisone causes bones to thin and may sometimes cause severe injury to bone. Prednisone can interfere with normal growth. Prednisone suppresses the immune system, making patients more susceptible to infection. Prednisone cannot be stopped suddenly since the adrenal glands, which normally make corticosteroids, may be suppressed. This can cause the patient to develop life-threatening adrenal insufficiency (the body goes into crisis due to lack of corticosteroids). Prednisone causes cosmetic changes such as weight gain, stretch marks and round, full cheeks. Facial acne may appear. Hypertension and cataracts are seen, mostly in adults, and mostly with prolonged treatment. Efforts are usually made to wean the dose down to the least amount that will keep the disease process under control.
- Azathioprine or Imuran (or a related drug called 6-MP or 6-mercaptopurine) may also be used to suppress the immune system, commonly in the context of autoimmune hepatitis. These drugs affect the DNA synthesis of rapidly dividing cells. Rarely, these drugs can cause hepatitis or pancreatitis. Low white counts are a more common problem, and blood work is monitored to assess this. An allergic reaction with fevers, rash, joint pains, gastrointestinal symptoms can develop 3 to 4 weeks after starting such a drug. Theoretically, since the drugs affect DNA repair, these drugs could be harmful to a developing infant, or even cause an increased risk of cancer. These are not common complications, although pregnancy should be undertaken with caution in patients on Imuran or 6-MP.
Drugs that Work Against Viruses
- Ribavirin is an oral drug used for treatment of chronic hepatitis C. This drug can cause anemia due to hemolysis, a process in which blood cells break down. Blood counts must be monitored during ribavirin therapy. Most importantly, ribavirin can cause severe damage to the developing fetus. It must not be taken by pregnant women and strict attention must be paid to birth control if a sexually active patient is taking ribavirin.
- Lamivudine is an oral drug used for treatment of chronic hepatitis B. It has very few side effects. Unfortunately, in a large fraction of treated patients the virus learns to mutate or change to avoid the drug's effects.
- Interferon injections are used for treatment of both chronic hepatitis B and hepatitis C, in different doses. As summarized in the section on viral hepatitis, interferon causes fevers, chills, and flu-like symptoms, especially with the first few doses. Drops in white blood cell counts can be seen and can require dose adjustment. Depression must be looked for carefully as it can be a serious complication. GI symptoms and weight loss are seen sometimes. Rare complications include autoimmune diseases, hair loss, bleeding into the eyes, heart, lung and neurological problems.
Drugs Used for Ascites
These "diuretic" drugs and others like them are used to help rid the body of excess fluid:
- Spironolactone (Aldactone) opposes a hormone that makes patients with cirrhosis retain fluid.
- Furosemide (Lasix) is another diuretic which makes the patient urinate extra fluid. It can cause abnormalities in the salts in the blood.
Drugs Used to Treat Encephalopathy
When the liver does not work well toxins accumulate which may make the brain function abnormally. Patients with liver disease may be confused, agitated or sleepy. One potential cause of this is accumulation of ammonia.
- Lactulose is sometimes used to help the body get rid of ammonia. Lactulose is given until the child has 3 or 4 loose stools per day. If the body gets rid of more ammonia, the child's mental state may improve.
- Neomycin is an oral antibiotic sometimes used to control the growth of ammonia-producing bacteria in the intestine.
Ursodeoxycholic Acid or URSO (trade name Actigall)
Ursodeoxycholic Acid or URSO (trade name Actigall) is used to promote bile flow. This drug helps water flow in bile ducts, and also makes bile less toxic to the liver when it backs up due to poor bile flow. Actigall has been used to treat a variety of liver diseases including liver disease related to cystic fibrosis, Alagille's syndrome and many other conditions in which bile flow is poor. Actigall has been used in sclerosing cholangitis where it has been shown to make the blood tests better, although has not improved the general course of the disease.
The liver makes bile salts, which help the body absorb fat from the diet. If liver function is poor or if bile flow is blocked in a condition such as biliary atresia, then fat cannot be absorbed. This causes the body to malabsorb (lose) fat-soluble vitamins that dissolve in fat. These include vitamins A, D, E and K.
- Vitamin A: Deficiency in vitamin A can cause problems with the eyes and skin. Deficiency of vitamin A is not as common as deficiency in the other fat-soluble vitamins below. Vitamin A can be supplemented orally with a dose of 5,000 to 10,000 IU/day. It is important not to give an excess of vitamin A since this can cause brain swelling.
- Vitamin D: A deficiency in vitamin D can lead to very weak bones (a disease called "rickets") or even bone fractures. Vitamin D can be supplemented using one of several vitamin D preparations. Often very large doses of vitamin D are needed in patients with blockage to bile flow (some jaundiced infants, who would normally need 400 IU of vitamin D, require as much as 8,000 IU of vitamin D). A few infants with blockage to bile flow cannot absorb vitamin D given orally and require a vitamin D shot every three months. Too much vitamin D, however, can cause too much calcium to appear in the blood.
- Vitamin E: Low vitamin E can cause problems with the nervous system as well as with the skin. Vitamin E can be supplemented orally through several forms. The best absorbed is called Liqui-E or TPGS-conjugated vitamin E. This is ideal for use in infants with liver disease. This preparation can even help other fat-soluble vitamins to be absorbed if they are taken together.
- Vitamin K: Vitamin K is needed for the liver to make the proteins that make the blood clot. Deficiency in vitamin K can cause severe bleeding in the internal organs or even in the brain, causing stroke. Vitamin K is easily supplemented by giving anywhere between 2.5 and 10 mg/day orally. An occasional patient will require monthly vitamin K injections when absorption is especially poor.