Alagille syndrome medical therapy: Difference between revisions
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==Overview== | ==Overview== | ||
Alagille syndrome may be managed through medical therapies that involve improving bile flow and reducing itching. There is controversy over the degree of success of such treatments. | There is no known cure for Alagille's Syndrome. Most of the treatments available are aimed at improving the functioning of the heart, and reducing the effects of impaired liver function. Alagille syndrome may be managed through medical therapies that involve improving bile flow and reducing itching. There is controversy over the degree of success of such treatments. | ||
==Medical Therapy== | ==Medical Therapy== | ||
The medical management of Alagille Syndrome is complex and continues to generate controversy. The significant variability of organ involvement requires the managing physician to have an understanding of the breadth and interplay of the variable manifestations. Furthermore, the liver disease in particular requires an appreciation of the natural history and evolution of the profound cholestasis. | The medical management of Alagille Syndrome is complex and continues to generate controversy. The significant variability of organ involvement requires the managing physician to have an understanding of the breadth and interplay of the variable manifestations. Furthermore, the liver disease in particular requires an appreciation of the natural history and evolution of the profound cholestasis. Treatment for Alagille syndrome is aimed at increasing the flow of [[bile]] from the [[liver]], promoting growth and development, and making the person as comfortable as possible. [[Ursodiol]] (Actigall, Urso) is the only drug approved by the [[U.S. Food and Drug Administration]] to increase bile flow. Other treatments address specific symptoms of the disease. | ||
'''Pruritus.''' Itching may improve when the flow of [[bile]] from the [[liver]] is increased. Medications such as [[cholestyramine]] ([[Questran]], [[Prevalite]]), [[rifampin]] ([[Rifadin]]), [[naltrexone]] ([[ReVia]], [[Depade]]), [[antihistamines]], [[Ursodiol]] (Actigall),[[Hydroxyzine]] (Atarax), [[Cholestyramine]], [[Rifampicin]], and [[Phenobarbitol]]may be prescribed to relieve [[pruritus]]. Hydrating the skin with [[moisturizers]] and keeping fingernails trimmed to prevent skin damage from scratching are important. | |||
If severe [[pruritus]] does not improve with medication, a procedure called [[partial external biliary diversion]] (PEBD) may provide relief from itching. [[PEBD]] involves surgery to connect one end of the [[small intestine]] to the [[gallbladder]] and the other end to an opening in the abdomen—called a [[stoma]]—through which bile leaves the body and is collected in a pouch. | |||
A [[liver transplant]] may be necessary for a person with [[liver failure]] and severe [[pruritus]] that does not improve with medication or [[PEBD]]. | |||
'''Malabsorption and growth problems.''' Infants with Alagille syndrome are given a special formula that allows the absorption of much-needed fat by the [[small intestine]]. Infants, children, and adults can benefit from a high-calorie diet, [[calcium]], and [[vitamins]] A, D, E, and K. If oral doses of vitamins are not well tolerated, a health care provider may need to give the person injections for a period of time. A child may receive additional calories through a tiny tube that is passed through the nose into the stomach. If extra calories are required for a long time, a tube, called a [[gastrostomy tube]], may be placed directly into the stomach through a small opening made in the abdomen. The child’s growth may improve if nutrition status improves and the flow of bile from the liver increases. | |||
Many patients with Alagille's Syndrome will also benefit from a high dose of a multivitamin such as ADEK (contining high levels of vitamins A, D, E, and K), as the reduced bile flow makes it difficult to absorb and utilize these vitamins.It is equally important to optimize nutrition to maximize growth and development. Those with splenomegaly need to use spleen guard during activnities. Routine follow up with a pediatrician is necessary. | Many patients with Alagille's Syndrome will also benefit from a high dose of a multivitamin such as ADEK (contining high levels of vitamins A, D, E, and K), as the reduced bile flow makes it difficult to absorb and utilize these vitamins.It is equally important to optimize nutrition to maximize growth and development. Those with splenomegaly need to use spleen guard during activnities. Routine follow up with a pediatrician is necessary. | ||
'''Xanthomas.''' These fatty deposits typically worsen over the first few years of life and then improve over time, or they may eventually disappear in response to PEBD or the medications used to increase bile flow. | |||
'''Liver failure.''' In some cases, Alagille syndrome will progress to end-stage [[liver failure]] and require a [[liver transplant]]. A liver transplant is when the diseased liver is removed and replaced with a healthy one from an organ donor. | |||
The health care team carefully considers the risks and benefits of a transplant and discusses them with the patient and family. People with Alagille syndrome and heart problems may not be candidates for a transplant because they could be at high risk for complications during and after the procedure. | |||
==References== | ==References== | ||
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{{WH}} | {{WH}} | ||
{{WS}} | {{WS}} | ||
Latest revision as of 19:59, 27 August 2012
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Alagille syndrome Microchapters |
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Risk calculators and risk factors for Alagille syndrome medical therapy |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Overview
There is no known cure for Alagille's Syndrome. Most of the treatments available are aimed at improving the functioning of the heart, and reducing the effects of impaired liver function. Alagille syndrome may be managed through medical therapies that involve improving bile flow and reducing itching. There is controversy over the degree of success of such treatments.
Medical Therapy
The medical management of Alagille Syndrome is complex and continues to generate controversy. The significant variability of organ involvement requires the managing physician to have an understanding of the breadth and interplay of the variable manifestations. Furthermore, the liver disease in particular requires an appreciation of the natural history and evolution of the profound cholestasis. Treatment for Alagille syndrome is aimed at increasing the flow of bile from the liver, promoting growth and development, and making the person as comfortable as possible. Ursodiol (Actigall, Urso) is the only drug approved by the U.S. Food and Drug Administration to increase bile flow. Other treatments address specific symptoms of the disease.
Pruritus. Itching may improve when the flow of bile from the liver is increased. Medications such as cholestyramine (Questran, Prevalite), rifampin (Rifadin), naltrexone (ReVia, Depade), antihistamines, Ursodiol (Actigall),Hydroxyzine (Atarax), Cholestyramine, Rifampicin, and Phenobarbitolmay be prescribed to relieve pruritus. Hydrating the skin with moisturizers and keeping fingernails trimmed to prevent skin damage from scratching are important.
If severe pruritus does not improve with medication, a procedure called partial external biliary diversion (PEBD) may provide relief from itching. PEBD involves surgery to connect one end of the small intestine to the gallbladder and the other end to an opening in the abdomen—called a stoma—through which bile leaves the body and is collected in a pouch.
A liver transplant may be necessary for a person with liver failure and severe pruritus that does not improve with medication or PEBD.
Malabsorption and growth problems. Infants with Alagille syndrome are given a special formula that allows the absorption of much-needed fat by the small intestine. Infants, children, and adults can benefit from a high-calorie diet, calcium, and vitamins A, D, E, and K. If oral doses of vitamins are not well tolerated, a health care provider may need to give the person injections for a period of time. A child may receive additional calories through a tiny tube that is passed through the nose into the stomach. If extra calories are required for a long time, a tube, called a gastrostomy tube, may be placed directly into the stomach through a small opening made in the abdomen. The child’s growth may improve if nutrition status improves and the flow of bile from the liver increases.
Many patients with Alagille's Syndrome will also benefit from a high dose of a multivitamin such as ADEK (contining high levels of vitamins A, D, E, and K), as the reduced bile flow makes it difficult to absorb and utilize these vitamins.It is equally important to optimize nutrition to maximize growth and development. Those with splenomegaly need to use spleen guard during activnities. Routine follow up with a pediatrician is necessary.
Xanthomas. These fatty deposits typically worsen over the first few years of life and then improve over time, or they may eventually disappear in response to PEBD or the medications used to increase bile flow.
Liver failure. In some cases, Alagille syndrome will progress to end-stage liver failure and require a liver transplant. A liver transplant is when the diseased liver is removed and replaced with a healthy one from an organ donor.
The health care team carefully considers the risks and benefits of a transplant and discusses them with the patient and family. People with Alagille syndrome and heart problems may not be candidates for a transplant because they could be at high risk for complications during and after the procedure.