A new drug offers promise of helping to cure patients suffering from Hepatitis C and other liver diseases who otherwise would not have been able to get treatment, a study released Wednesday found.
The drug also appears to be an effective treatment for a common blood disorder which causes bruising and excessive bleeding, according to a second study which was also published in the New England Journal of Medicine.
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The drug, called eltrombopag, serves to boost blood platelet counts, a critical component to blood clotting.
When platelet counts get too low there is a risk of serious bleeding. Patients with low platelet counts are often either deemed unfit for treatment or must receive costly and complex transfusions prior to undergoing surgery.
This is particularly problematic in the estimated 170 million patients worldwide infected with the Hepatitis C virus. The potentially fatal virus damages the liver and can also cripple the body's ability to manufacture platelets.
Only about half of those diagnosed are able to be cured with standard antiviral treatments.
"There's this huge group of patients with Hepatitis C where we try to treat them and their platelet counts drop or we're afraid to treat them," said lead author John McHutchison, an Australian researcher at Duke University in North Carolina.
"For 20 years of looking after people with liver disease we've watched their platelet counts and never had a drug that could treat that," he said in a telephone interview.
"Potentially, it could be a very important advance in many, many areas."
McHutchison and his colleagues have already started Phase III clinical trials to confirm the results and see if the drug is able to safely maintain platelet counts long enough for patients with Hepatitis C to undergo a full course of treatment and be cured.
Further studies will be needed to see if the drug can be safely used in other treatments, such as boosting platelet counts in cancer patients undergoing chemotherapy and those suffering from other liver diseases, he said.
"It could be a number of years before we have the results," McHutchison cautioned.
The double-blind study looked at 74 Hepatitis C patients in 22 centers in the United States and Europe who had developed cirrhosis of the liver and had very low platelet counts.
Platelet counts were raised significantly in at least 75 percent of those receiving the drug, which is taken orally once a day, while 95 percent of those receiving the highest dose saw their platelet counts jump in the four weeks of treatment.
Side effects included headaches, abdominal pain, nausea and dry mouth.
The drug was also found to be effective in treating the common blood disorder Idiopathic Thrombocytopenic Purpura which causes bleeding and bruising because the blood does not clot properly due to low platelet counts.
The second study published in the New England Journal of Medicine found that eltrombopag was effective in significantly boosting the platelet counts and reducing bleeding incidents in 80 percent of patients receiving either 50 or 75 milligrams a day within 15 days of treatment.
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