A new study published in Alimentary Pharmacology & Therapeutics found that while people with primary biliary cholangitis (PBC) who stayed on ursodeoxycholic acid (UDCA), obeticholic acid (OCA) or both were more likely to reach normal alkaline phosphatase (ALP) levels — an indicator of bile duct health — the majority of patients didn’t stay on treatment long-term.
Read more about PBC testing and diagnosis
Study investigators reviewed U.S. insurance and laboratory data collected over a span of five years to understand the real-world treatment patterns for more than 20,000 people living with PBC. The review was conduced before OCA was removed from the U.S. market due to safety concerns.
About half of the people who started on UDCA, OCA or both had stopped taking their respective treatment at the one-year mark. By the fifth year, fewer than 10% of people within the study remained on therapy. Stopping treatment was more common among people experiencing pruritus, fatigue or abdominal pain and in those with cirrhosis, portal hypertension or Medicaid coverage.
“This study highlights the high PBC treatment discontinuation rates and emphasises the importance of sustained treatment UDCA among individuals with PBC, given its association with being in the normal ALP range,” the study’s authors wrote.
Among participants with the most severely elevated ALP levels before starting treatment, 33% of those taking UDCA, 17% taking OCA and 11% taking both therapies achieved normal ALP levels within six to 24 months of consistent treatment. Those who stopped therapy for 60 days or longer were unlikely to reach normal ALP levels.
People whose ALP levels returned to normal had a noticeably lower chance of liver transplantation, hospitalization or death compared to those whose levels remained high. Improvements were observed in both younger and older adults, as well as in individuals with or without cirrhosis. These findings indicate that reaching normal ALP levels was associated with better outcomes across all groups.
The study’s authors emphasized that improving treatment persistence could help more people reach normal ALP levels and reduce serious liver complications. Better management of symptoms such as itching and fatigue, along with access to emerging therapies, may help patients remain on treatment longer and achieve better outcomes.
Maintaining consistent treatment offers people with PBC the best opportunity to keep their liver healthy and delay serious complications. As medications such as seladelpar and elafibranor become available, future research will explore access to treatment, how treatment changes or restarts and how the use of fibrates alongside standard therapy might influence ALP levels and long-term outcomes.
Sign up here to get the latest news, perspectives, and information about PBC sent directly to your inbox. Registration is free and only takes a minute.
