Primary sclerosing cholangitis (PSC) is a rare and serious liver disease that has been difficult to recognize and adequately treat non-invasively. In many cases, PSC patients can remain asymptomatic for years without being diagnosed, and if they cannot receive a liver transplant, their life expectancy is just over ten years.
The agenda for the American Association for the Study of Liver Diseases (AASLD) 2023 Liver Meeting in Boston this weekend includes substantial discussion of the current status of PSC detection, management, and investigation. But one of the key sessions focused on patient burden—from patients and key advocates themselves.
during an interview HCP live During the Liver Conference, Ricky Safer, CEO and founder of PSC Partners Seeking a Cure, discussed the current state of PSC patients and today’s diagnostic strategies. Safer, who was diagnosed with the disease in 2003 before founding her nonprofit, recalled that clinicians told her that about two-thirds of patients with the disease were in their 20s and 30s.
In patient advocacy groups, it’s a different reality.
“I know that in our community and in the people who are actively engaged in our activities, we see a much higher representation of women in everything we do,” Seifer said. “Part of the reason is that men tend to be less willing to talk about it, so they don’t come to us as easily.”
Although PSC affects every gender, race, ethnicity and socioeconomic background, the “true diversity” of the disease (beyond hospitalization and transplant data) is poorly understood, Safer said. This poor representation affects organizations like Safer’s, as well as many clinical trial projects currently under investigation.
Regarding pharmacological studies, Safer said that because understanding of PSC is still evolving, the goals for patients and clinicians are limited to symptom relief. Still, it’s a niche field, and investigators have time and again shown a willingness to “give up on themselves” while pursuing larger goals, Seifer said.
“To me, there’s so much research going on… we’re seeing progress, and I think part of it is because everyone is working together,” she said.
Safer acknowledged that modern PSC imaging capabilities have taken a “huge leap” compared to when they were first diagnosed in 2023. Even as clinicians gain a deeper understanding of the diseases patients are already facing, predictive biomarkers in PSC are still under investigation. Before being diagnosed in 2003, she had been symptom-free for 13 years—a trend not well known in the PSC patient community.
“Many of us face this condition and because we don’t have symptoms for so long, we don’t realize we have it,” Sarver said. “The only thing I notice is my stomach Gut noticed that my (alkaline phosphatase) was elevated. I had liver biopsies and ongoing blood tests, and it wasn’t until I was hospitalized for my first cholangitis that I realized, ‘This GI can’t fix this. . I need to see a hematologist.”
It is fairly common for PSC partners to help new patients connect with hepatology specialists, but this task is made difficult by the lack of availability and distance of specialists compared to where patients live in the United States.
“It’s very difficult to diagnose this rare disease, although I do feel that the U.S. military and general practitioners have a much better understanding of what it is and what these very early symptoms may mean than they did 20 years ago,” Safer said.