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Online PSC registry offers ‘dynamic and ongoing’ research, PROs
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Analysis of a primary sclerosing cholangitis online registry provided efficient collection of patient-reported outcomes, analysis of treatment, disease progression and inclusion of previously underrepresented cases of PSC.
Alexander Kuo, MD, a gastroenterology and hepatology Fellow from the University of California, talked to Healio Gastroenterology and Liver Disease about the PSC Partners Patient Registry, a web-based patient-driven database established in 2014 in collaboration with the National Institutes of Health.
Healio: What is the focus of the registry’s design?
Kuo: This registry came about in the year 2014 and is an ongoing patient-recruitment process. Our first group of participants that we analyzed between 2014 and 2016 included a total of 870 patients. The method to which patients were recruited was entirely voluntary.
There has been an annual patient conference in which patients with PSC get together in one U.S. city. We have this large group of participants; we thought perhaps they could pool their experiences together and then submit their own history of PSC as a way to gain strength in numbers for studying the disease and hopefully find some patterns that may prove useful in clinical research.
Healio: How does this online registry differ from standard data collection related to PSC?
Kuo: Most of the existing patient data, because this disease is so rare, are only collected through large national disease registries or coronary care centers. So, a center that is known for caring for PSC patients over a course of 10 to 20 years will collect a similar number of patients, although this is much longer-term and tends to attract certain types of patients.
This being a very rare disease, previously large registries would probably number no more than 400 or 500 patients, so the PSC Patient Registry is definitely on the larger side. One key difference is that those previous registries were probably a little more objective in the sense that physicians recorded disease progression and laboratory findings, but those registries can tend toward a bias of patients that are sicker and require more direct care. Let’s say if you have a relatively asymptomatic patient, they might not be encouraged to go to these treatment centers and may never be included in a registry. In comparison, the hope with the PSC Patient Registry is that people who either know somebody or themselves are suffering from some stage of PSC are encouraged to tell their story and provide data that can be used in an organized manner.
Healio: What were the unique findings in the registry compared with previous reported data?
Kuo: PSC, historically, has a male predominance, whereas we found a female predominance in the registry. This was one interesting aspect. Another key difference we found was that patients were reported to survive longer — this includes both survival till death or liver transplantation. We reported as long as 21 years and this is significantly longer than previously reported. We’re actually quite encouraged by this result; we expect there is a lot to learn from these patients with longer survival.
We were also astonished to see is how prevalent the reported symptoms were among the cohort, including the common symptoms such as itching, abdominal pain, poor sleep and depression. Some of the things that might not directly relate to what hepatologists are studying but are nonetheless related to PSC diagnosis and care.
Healio: How can researchers use the current data for future studies?
Kuo: I think that remains to be seen. I think the main limitation is the lack of diagnostic confirmation in certain cases such as lab data or MELD scores, but where this registry thrives is that it is a dynamic and ongoing data collection. As we come out with new clinical endpoints and symptom parameters validated by clinical studies, we can look to the registry to compare data and return to patients for validation.
Healio: What should physicians know about this registry ?
Kuo: PSC studies are only as strong as we our ability to enroll patients, given how rare this disease is, so I would say that advocacy and recognition of this disease is the most important thing that this organization is striving for. Physicians should be encouraged to inform patients about participating in this registry.
Reference: www.pscpartners.org
Disclosure: The authors report no relevant financial disclosures.