Biggest hepatology breakthroughs in 2016: Nonalcoholic steatohepatitis, primary biliary cholangitis

John Vierling, MD, chief of hepatology at Baylor College of Medicine in Houston, spoke with Becker's ASC Review about new developments in the field.

Here's what he had to share about three key liver conditions:

1. Hepatitis C. This year brought additional therapeutic options for chronic hepatitis C to patients, including the FDA approval of Epclusa, a Gilead Sciences-developed tablet containing sofosbuvir and velpatasvir.

Outside of approvals, there has also been successful research done on both a third generation of direct-acting antivirals and on new therapies with triple or double drug regimens in a single pill.

"The dual and triple investigational DAAs have shown tremendous rates of success in the initial phases of their analyses," Dr. Vierling says. "We are anticipating, with some degree of certainty, that each of these regimens will meet the approval criteria for the regulatory agencies."

However, even as additional treatment options for hepatitis C are made available, a major concern is ensuring early diagnosis to prevent disease progression. At present, cirrhosis caused by chronic hepatitis C is the most common reason for a patient to need a liver transplant, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

"There's an important need for our society to decide, as individuals and collectively, to support transplantation activities by becoming organ donors and advocating for organ donation," Dr. Vierling says of the organ shortage in the United States.

"We have continued to suffer from inadequate organ donation. As our population continues to grow, waiting lists for those who need transplants have also increased; but sadly, the portion of our population that is donating these needed organs has decreased."

2. Nonalcoholic steatohepatitis. In recent years, NASH has emerged as an important public health consideration, according to Dr. Vierling. Along with being a progressive disease that can result in long-term damage like cirrhosis, NASH is also the second most common reason for liver transplantation in the United States.

"Today, there are numerous clinical trials investigating multiple individual drugs that interrupt pathways believed important in the pathogenesis of NASH," Dr. Vierling says. "Two trials have also investigated the ability to reverse cirrhosis, an advanced scarring reaction within the liver.

"However, the most important thing to recognize is that we do not have results of phase 3 trials for any of the investigational drugs, and therefore, as yet, we do not have any approved drugs for care. NASH will be our primary challenge every month and every year to come."

Since these potential therapies are not yet on the market, Dr. Vierling stresses that physicians should focus on diagnosing and managing the disease, with an emphasis on controlling diet and exercise.

"We have a serious problem with NASH; it's like a tsunami that we can see moving toward the shore, but that has not yet broken on the shore," Dr. Vierling says, regarding the growing rates of conditions associated with NASH, like obesity and type 2 diabetes.

"We need to have a multipronged approach to NASH. In addition to drug therapies, this must include effective education about appropriate diet and exercise regimens to prevent or retard the underlying root causes."

3. Primary biliary cholangitis. For the past few decades, the primary treatment for PBC was ursodeoxycholic acid, a therapy that slows progress of the disease in 60 percent of patients.

This year, the FDA approved Ocaliva, an Intercept Pharmaceuticals-developed oral treatment containing obeticholic acid. Ocaliva is targeted toward the 40 percent of patients who had a suboptimal response to ursodeoxycholic acid.

"There remains an unmet need in the population with PBC," Dr. Vierling explains, noting that about 50 percent of those treated with Ocaliva in clinical trials had suboptimal responses to both ursodeoxycholic acid and obeticholic acid.

"However, when we note that roughly 60 percent of PBC patients respond to ursodeoxycholic acid alone, and that half of the remaining 40 percent respond to the addition of obeticholic acid, that is an enormous breakthrough for patients with this disease."

Moving forward, Dr. Vierling maintains that effective diagnosis will be the primary hepatology concern for 2017.

"We now have therapies for hepatitis C, hepatitis B, PBC, autoimmune hepatitis, some of the genetic liver diseases and emerging therapies for NASH." he says.

"Part of our overall challenge is to apply these therapies in the earliest stages of the disease, which requires early, accurate diagnosis. Without improving the capacities of our healthcare system to identify and evaluate patients with liver disease and to diagnosis the root cause, all of our therapeutic options will remain somewhat moot."

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