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First Treatment Approved for Disease Linked to Stem Cell Transplant

Liver condition is rare, but deadly

Please note: This article was published more than one year ago. The facts and conclusions presented may have since changed and may no longer be accurate. And "More information" links may no longer work. Questions about personal health should always be referred to a physician or other health care professional.

WEDNESDAY, March 30, 2016 (HealthDay News) -- Defitelio (defibrotide sodium) has been approved by the U.S. Food and Drug Administration to treat a rare, but usually fatal liver disease that affects some people who have had a stem cell transplant.

The condition, fatal in as many as 80 percent of cases, is called hepatic veno-occlusive disease (VOD). It affects people who have had a stem cell transplant to treat cancers of the blood or bone marrow.

Hepatic VOD causes blockage of blood vessels in the liver, which could lead to damage of the liver, kidneys and lungs, the FDA said.

Defitelio was investigated in 528 people with hepatic VOD who participated in three clinical studies. The most common side effects included low blood pressure, diarrhea, vomiting, nausea and nosebleed.

More serious side effects could include internal bleeding and allergic reactions, the FDA said.

Defitelio is marketed by Jazz Pharmaceuticals, based in Palo Alto, Calif.

More information

The FDA has more about this approval.


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