Clinical guidelines or guidances are expert recommendations that help doctors care for people with PSC. Because PSC is a rare, long-term disease, guidelines play a key role in guiding treatment and monitoring. This is especially important for people with PSC who do not have access to a PSC specialist.
Two recent publications for PSC guidelines and guidance were published by the American Association for the Study of Liver Diseases (AASLD)1 and the European Association for the Study of the Liver (EASL)2. While written for health care providers, these publications help patients know what questions to ask their doctors about possible treatment and intervention options.
Why are guidelines (or guidances) important in managing PSC?
PSC is uncommon and unpredictable. Doctors may only care for a small number of people with PSC, and the disease can behave very differently from one person to another.
At present, there is no proven medication that reliably stops PSC from progressing. Research studies are often small or limited. Some treatments improve blood tests but have not been shown to improve long-term outcomes, such as avoiding liver transplantation. Guidelines help doctors navigate this uncertainty.
What’s the difference between a guideline and a guidance?
The terms “guidance” and “guidelines” are often used interchangeably, but they are not exactly the same, especially for a rare disease like PSC.
Guidelines
Guidelines are official, structured recommendations that are based on a systematic review of all available evidence and often graded to show the strength of the evidence.
For PSC, guidelines usually focus on what should and should not be done. They take a cautious approach because high-quality evidence is limited and they avoid recommending treatments unless there is proof of meaningful long-term benefit.
Guidance
By comparison, a guidance is broader and less rigid. It is often written to help clinicians manage areas where evidence is uncertain or incomplete. Guidances may be more willing to discuss options, emerging therapies, and real-world practice.
Guidances often acknowledge that evidence is limited. They describe how specialists approach care in practice and allow more room for individualised decision-making. A guidance may discuss treatments considered experimental or used in specialist centres.
This distinction matters because PSC is a rare and complex disease where large clinical trials are difficult, many decisions must be made with incomplete evidence, and patient experiences can vary widely. Guidelines prioritise safety and consistency whereas guidances help clinicians and patients make shared decisions when clear answers don’t exist.
Who creates PSC guidelines?
PSC guidelines are written by experts in the field. These include liver specialists (hepatologists) and researchers with expertise in PSC. These experts review all available scientific evidence, including clinical trials, long-term patient follow-up studies, safety data, and experience from specialist PSC centres.
Professional liver societies, such as international or national liver associations, for example AASLD (American Association for the Study of Liver Disease and EASL (European Association for the Study of the Liver) have produced recent guidelines.
To date, the Canadian professional association CASL (Canadian Association for the Study of the Liver) has not published a PSC guidance or guideline.
What do PSC guidelines recommend?
PSC guidelines typically focus on:
- Monitoring the disease carefully (regular blood tests, imaging, and cancer surveillance)
- Managing symptoms such as itching or fatigue
- Treating complications if and when they arise
- Identifying when referral for liver transplantation is appropriate
- Clearly stating which treatments are recommended, not recommended, or considered experimental or suitable only in research studies
Why are PSC guidelines often cautious about medications?
PSC is a rare disease impacting an estimated 1 in 10,000 Canadians. This makes large, definitive drug trials difficult to conduct. Some treatments show early promise but lack proof that they improve survival or prevent complications. As well, the long-term safety of many therapies is unknown.
Guidelines therefore tend to be conservative, prioritising patient safety and avoiding treatments that may cause harm without proven benefit.
This caution does not mean research has stopped—it reflects the current limits of medical knowledge.
Do PSC guidelines limit individual care?
PSC guidelines do not limit individual care. They are not rigid rules.
Guidelines are meant to support doctors in making informed decisions. They encourage honest discussions between patients and specialists as well as allow individualised care, especially in complex or unusual cases.
In some situations, a doctor and PSC patient may decide together to try a treatment outside of standard recommendations, often within a clinical trial or specialist centre.
Do PSC guidelines change over time?
PSC guidelines are regularly updated as new research becomes available. Sources of information can be ongoing clinical trials report results or a better understanding of the disease develops.
PSC treatments considered experimental today may become standard care in the future.
Clinical guidelines for treating PSC exist to promote safe, consistent, high-quality care. They reflect the current evidence and protect patients while research continues. Guidelines also support shared decision-making between patients and their care team.
PSC guidelines or guidances are a tool to guide care in a rare and complex disease—not a barrier to hope or progress.
Endnotes:
- PSC Clinical Guidance (2023) from the American Association for the Study of Liver Disease: https://journals.lww.com/hep/fulltext/2023/02000/aasld_practice_guidance_on_primary_sclerosing.29.aspx AASLD practice guidance on primary sclerosing cholangitis and cholangiocarcinoma., Bowlus, Christopher L. et al.,Hepatology 77(2):p 659-702, February 2023.
- PSC Clinical Guidelines (2023) from the European Association for the Study of the Liver: https://www.journal-of-hepatology.eu/article/S0168-8278(22)00326-9/fulltext EASL Clinical Practice Guidelines on sclerosing cholangitis, Chazouilleres, Olivier et al., Journal of Hepatology, Volume 77, Issue 3, 761 – 806
