Researchers: Drug will 'dramatically reduce' need for liver transplants

Researchers: Drug will 'dramatically reduce' need for liver transplants

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SALT LAKE CITY — The need for severely ill patients to receive a liver transplant can be dramatically lowered by a new drug, according to the findings of an Intermountain Healthcare study unveiled Friday at a medical conference in the Czech Republic.

The study found that seriously ill patients who exhibited a positive reaction to the drug sofosbuvir were also 50 times less likely to die from hepatitis C-caused severe cirrhosis than similar patients never given the drug, said Dr. Michael Charlton, the lead researcher and associate director of the Intermountain Medical Center Transplant Program.

The study's findings relate specifically to patients with cirrhosis brought on by a hepatitis C infection. Other causes of the liver illness include alcoholism and nonalcoholic staetohepatitis.

The study is a major landmark in providing evidence that the treatment is a cure for the large majority of patients specifically suffering from hepatitis C-caused cirrhosis to a severe degree, as opposed to only helping those with moderate symptoms, Charlton said.

"The benefit is quick and it's dramatic," he told the Deseret News. "If you work in the liver disease field, it's … a really good thing to be be able to offer your patients."

Only 3 percent of those who had been treated with sofosbuvir ended up needing a liver transplant, compared to 40 percent in the control group. The sharp difference is striking and indicates a major breakthrough in targeting cirrhosis, Charlton said.

"Treating these patients (using sofosbuvir) had a tremendous impact," he said. "There was a tremendous benefit."

Charlton said reducing the need for transplants is critical because 20,000 Americans die each year from cirrhosis, but the country's health system as of now can only accommodates about 1,500 transplant procedures per year.

"We don't have enough livers in the United States," he said. "Nowhere in the world does."

In all, about 2 million Americans and between 300 million and 500 million people worldwide suffer from the closely related hepatitis C, Charlton added. About 100,000 Americans are ill enough with cirrhosis due to hepatitis C that they match up with the severity-of-illness parameters used for the test group examined in the study, he said.

"We think we can dramatically reduce (cirrhosis deaths) by treating this population" with sofosbuvir, Charlton said.

The U.S. Food and Drug Administration first allowed sofosbuvir to be made commercially available in December 2013. Its effectiveness at treating hepatitis C-caused cirrhosis with less than severe symptoms is already proven and "very straightforward," Charlton said. But some governments around the world have balked at sofosbuvir's use for severe cirrhosis cases because of how expensive it is and a lack of information about whether seriously ill patients can be significantly helped, he said.

This is true especially in Asia, where many countries, including wealthy nations such as Japan and South Korea, are "essentially preventing access of treatment to these (most seriously ill) patients" because of cost concerns, Charlton said.

"I think they didn't know what the benefit will be. (They worry that) these patients (are too) sick — that you're going to give them the treatments, that you're going to spend all the money on the therapy, but without the ability to reverse the underlying illness," he said.

But the new findings provide a resounding answer to that concern, meaning the study ought to be a significant factor for those governments' decision-making, Charlton said.

"The uncertainty about the benefits, about treating the sickest group of patients, is removed. … I think (those countries) should reconsider their position," he said. "I think what we've shown definitively here is … these (most seriously ill) patients benefit tremendously."

Because the treatment is now shown to work, it's clear that its use will actually save countries money by reducing other costs associated with transplant procedures and additional hospitalizations, he said.

"This is a really good use of health care resources," he said.

If successful, weekly oral intake of sofosbuvir for 12 weeks completely eliminates the hepatitis C virus, and the infection never comes back unless they are re-exposed to it, according to Charlton. Its common side effects — fatigue and headache — are considered relatively mild.

Charlton said it's also possible for doctors to screen patients for how likely it is that the sofosbuvir treatment would be successful and make decisions together based on those odds.

The previous leading treatment, which employed a drug called interferon, was administered using an injection that typically had more serious side effects and was not as effective, Charlton said. The new drug is "so dramatically better than what we had before," he said.

In the United States, the drug is extremely expensive. The full 12-week regiment required for taking the pill costs $84,000, equating to $1,000 per pill, according to detailed information about the drug posted online by the University of Washington. Such a price also presents a formidable barrier for some Americans with regard to actually getting the treatment, depending on their health care coverage.

The large price tag is alarming, Charlton said, but there are no easy answers when it comes to getting the cost down.

"The high price is easily the biggest drawback to this medication," he said. "On the other hand, the development costs of this drug were about $12 billion and the window which companies have to recoup those costs are very hard to predict. … You really don't want to limit innovation. We're very lucky to have these spectacular new drugs be developed, and they come at a price, and the price is high."

Charlton, who is also president of the International Liver Transplantation Society, presented the study's findings Friday to that organization, as well as others, at a medical conference in Prague.

Subjects in the study were patients with late stage hepatitis C-caused cirrhosis in the United States, western Europe, Australia and New Zealand. There were 1,857 subjects treated between 2008 and 2013, who were not treated with sofosbuvir and were set aside as a control group. The 623 test group subjects who did get the drug were pulled from the integrated database of four other studies.

The study began in 2013 and was conducted through late 2016. Its complete analysis was finalized in February and presented for the first time Friday. Charlton said the study was assisted by researchers from Mayo Clinic, Harvard and Stanford, among others.aid the study was assisted by researchers from Mayo Clinic, Harvard and Stanford, among others.

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