PROMs and PSC
Patient Reported Outcome Measures
PROMs for PSC?
Patient reported Outcome Measures (PROMs) are used in research and healthcare to better understand the lived experience of particular conditions. However, without a fully validated PROM specifically for PSC patients, researchers and healthcare providers often ask PSC Support which PROMs they should use. We're sharing our focus group findings for the benefit of other investigators and researchers.
Focus Group
In October 2019, PSC Support led a focus group of twenty PSC patients to identify which PROMs currently available assess the most relevant and important issues to people with PSC.
Recommendations
- Well-designed, validated PROMs should be used in clinical trials at the very least as exploratory endpoints and that the resulting data be consistently reported.
- Careful consideration should be given to what the clinical trial is hoping to demonstrate, and PROMs chosen carefully, on a case-by-case basis.
- For recording itch, based on our focus group findings, we would recommend a combination of 5D-Pruritus Scale and the Pruritus Visual Analogue Scale.
- For recording fatigue, based on our focus group findings, we would recommend the PBC-40 over the Fatigue Severity Scale and the PSC-PRO.
- For recording general quality of life, while both have their own limitations, our focus group favoured the PBC-40 and PSC-PRO over the SF-36.
- Patients on clinical trials should be offered the opportunity to complete PROMs electronically in their own time.
Patient Reported Outcome Measures
A Patient Reported Outcome Measure (PROM) is a question or set of questions that allow a patient to record how they feel and function in daily life. They can include any aspect of their health, such as reporting on specific symptoms like tiredness, itch and pain, how much pain or itch they are experiencing, or reporting more generally about daily life and activities.
Blood test, scan and biopsy results give us a great deal of information about the liver and bile duct damage in PSC, but they tell us nothing about the lived experience of the disease. In fact, some tests don't seem to reflect how patients feel and function at all, that is how quality of life is affected by PSC.
PROMs can be used in healthcare to understand how an individual's quality of life is improving or deteriorating over time. PROMs bridge the gap between the way a disease affects the life of the individual affected and what medical tests reveal. They allow doctors to better understand how our disease affects us and is a starting point to address those issues.
When PROMS are properly collected, analysed and reported, they can inform shared-decision making and help improve the quality of healthcare.
At a higher level, as well as improvements to individual care, the use of PROMs can support clinical guideline and health policy development.
We hope in PSC that PROM results could support welfare benefit claims.
In research, PROMs can be used to understand the impact of a new drug or intervention on patients' lives.
Creating a valid PROM takes time and expertise. High quality qualitative methodology is critical in the development of PROMs to ensure reliability, validity and acceptability 136.
This involves a process of asking willing patients what issues affect them, and then checking that the issues identified are relevant and important to other patients in a similar position.
Each question (or item) needs psychometric analysis and validation on yet another group of similar patients.
This takes time.
The UK-PSC quality of life measure is in the final stages of validation and international development but is not yet ready for use. The UK-PSC quality of life measure has had significant patient input from the outset, and has been entirely funded by patient organisations: PSC Support, the British Liver Trust and PSC Partners Seeking a Cure.
What PSC Patients think
Suboptimal PROMs
There are a number of PROMs available for use right now, yet none of them are perfect for use in PSC137. From a patient perspective, the use of a suboptimal or poorly selected PROM is more damaging than helpful. PROMs apply only to the populations in which they were validated during development, and therefore using a PROM developed for a different disease on PSC patients would not give a true picture.
Focus Group
In October 2019, we worked with 20 people affected by PSC to understand which PROMs they felt were most useful in a clinical trial. Asking patients to review PROMs is a big ask, and time-consuming for those involved. For that reason, we did not garner views using a survey. We chose to ask open ended questions via email.
Quality of life in PSC
Our own surveys show that PSC has a considerable impact on quality of life that that patients live with daily debilitating symptoms, namely fatigue, itch and pain. Therefore, we asked patients to review the following PROMs and tell us which would be most useful in a clinical trial for PSC:
- the PBC-40 - a quality of life questionnaire developed for people with primary biliary cholangitis (PBC) containing 40 questions.
- the PSC-PRO - a quality of life questionnaire containing 42 questions developed for PSC patients (without IBD) specifically for use in clinical trials.
- the SF-36 - a questionnaire that is not disease-specific that measures health-related quality of life with 36 questions.
- the 5-D Pruritus Scale - a questionnaire containing 5 questions to explore the duration, quality, impact and severity of itch.
- the Pruritus Visual Analogue Scale (VAS) - a scale consisting of a 10cm long line on which patients indicate the severity of itch (from 'no itch' to 'worst imaginable itch').
- the Fatigue Severity Scale (FSS) - a questionnaire containing nine statements that attempt to explore severity of fatigue symptoms.
Out of the 5-D Pruritus Scale, the Pruritus VAS and the PBC-40 itch questions, only one person in our focus group preferred the VAS alone, and one person preferred the PBC-40. Most of the remainder favoured the 5-D Pruritus Scale alone or in conjunction with the Pruritus VAS.
Generally, PSC patients found the Pruritus VAS too simplistic and felt it didn't capture the real impact of itch. They favoured the itch questions in the PBC-40 but generally felt the 5D-Pruritus Scale was more comprehensive and best for use in clinical trials.
Comments from the focus group members included:
"Strengths in both - my preference would be the 5D as it seems to have more useful information for the researchers (esp, location of the itch). The visual analogue is obviously better suited to quick filling in so lends itself to the daily period, but feels over simplistic. The 5D obviously, aimed at longer intervals, would worry me that I was losing details and is dependent on frame of mind at that time. Shame it’s not possible to combine aspects of the two!"
"Guess the choice here is between a questionnaire that’s very easy and quick to fill in vs one that’s more comprehensive but would take a little longer. If the researchers need detailed info on itch and how it affects patients I’d go for 5-D Pruritus scale, if this detail isn’t needed I’d go for the VAS."
"The 5-D itch scale seems to be a better indicator of not only itch severity but also how it affects life aspects which seems better than the VAS which is simply a measure of itch without measuring how it impacts on sleep and other factors. With VAS, I’m not sure about simply putting a mark anywhere on the 10cm line – it doesn’t seem definite enough for me. The PBC itch scale seems better than the VAS scale but not as good as the 5-D itch scale. Both VAS and PBC scales don’t include a patient reported improvement or worsening (the patient would need to complete two questionnaires a few weeks apart to see if there is movement in the improvement/worsening) whereas the 5-d scale does ask the questions about how itching has changed over the last 2 weeks, so I think 5-D itch scale is the best overall."
"Should we use 5-D itch Scale or Itch VAS or BOTH? The 5-D itch Scale is a lot more specific, so In my opinion this would give a lot more and more accurate data regarding the itch. I feel that PBC-40 captures itch experience better as well..."
"I’d opt to use the 5-D itch scale. I feel it gives a lot more data that could be compared in the trial than the itch VAS. Maybe I am slightly biased as pre transplant my itching was horrific, but I feel the 5-D itch scale gives a much rounder picture of the impact itch can have rather than the very limited information which would be obtained in the itch VAS."
"I feel that the 5-D itch Scale is the better Scale to use as it is more in depth and will give a better picture of how bad someones itch is and how long they have experienced the itch, also how it affects activities and where on the body they itch."
The focus group considered the Fatigue Severity Scale and the fatigue questions in the PBC-40 and the PSC-PRO.
Generally, people expressed concerns about the FSS and the fact that the questions suggested depression. They didn't feel that it captured fatigue as well as the PBC-40. Patients liked the fact that the PBC-40 captured the impact on daily life better. Some people did, however prefer the FSS but they were in the minority.
Comments included:
"With regards the PSC trial specifically, if it [the FSS] is simply to gauging the impact of fatigue on daily living, then it does seem a good set of factors to score against. If it’s aiming to do more than that (i.e. spot depression as well) I think it needs other supporting assessments."
"Definitely one I think should be used [PSC-PRO]. Looks like a great set of questions especially compared to the FSS. It clearly accepts the overlap between fatigue and depression, but makes better attempts to differentiate. A lot to fill in daily, but weekly certainly."
"...think the way the PBC-40 is written makes it easier to answer than FSS - questions are clearer and more prescriptive so answers likely to be better defined? Like the scale being words rather than numbers - may be easier to answer? Also liked the following 3 Qs (19-21) as these also capture how fatigue can impact on your quality of life."
"I don’t like the FSS very much so would say not to use it. PBC 40 captures it a little better in my view"
"In terms of the FSS Questionnaire I'm not sure it would be an accurate way to assess a patient. I could easily circle 7 for every question as I agree that every statement is appropriate to me, however I know that I could be worse so I would have to guess which number to circle trying to allow for next week where I may be worse."
'I personally quite like the ...FSS questionnaire"
"I believe that the PBC-40 captures the fatigue experience better than the FSS."
"I don't think you need the FSS if you are including the PBC-40 questionnaire. ...I think PBC-40 is better at fatigue than FSS"
"I initially thought the FSS scale seemed to work ok although when I tried it out for myself, I found I under- scored myself. When I reviewed my answers I realized I needed to adjust the scores upwards. Perhaps this is because when you live with fatigue daily for years, you get used to it so it's hard to be totally objective and it becomes your norm so you are subjective from that baseline. When I gave it further thought about how my fatigue compared to when I was completely healthy 5 years ago, I realized I needed to increase my scores. I think the fatigue section of the PBC scale is much better than the FSS scale because the statements reflect everyday life better and assumes fatigue will affect people in these ways (which is correct!) and then asks the patient to agree using the ‘never, rarely, sometimes, etc..’ response approach which I think is better than the number scoring system of FSS."
"I think it [PBC-40] captures fatigue experience more effectively [than the FSS]. It has more specific measures of fatigue, relevant/experienced by PSC patients."
"My initial reaction reading this questionnaire [FSS] was oh my goodness could I have been depressed!? On reflection with all the other symptoms I was having at the point I was listed for transplant onwards, and with my low quality of life, it seems completely reasonable. However, I am not sure I’d have coped well ‘diagnosing’ myself as clinically depressed from doing the survey. Therefore as much as I think the [FSS] questions give a rounded picture of the impact of fatigue on an individual’s life, I don’t like the idea of it being used if patients had the same wording as it could result in them self-diagnosing clinical depression."
"Should we use the FSS? Yes Does PBC-40 capture fatigue experience better than the FSS? No"
"Pbc 40 is better than fss as it does capture fatigue and itch better but think fss is still worth using so I'd stick it all in."
The focus group considered the PBC-40, the PSC-PRO and the SF-36.
Generally, the group did not favour the SF-36. There were varied views on the PSC-PRO and the PBC-40, with some saying the PBC-40 captured the lived experience of PSC better, and others saying that the PSC-PRO captured it better.
These answers reflected the need for patient engagement throughout the development of a quality of life measure, and that the questionnaires reviewed were not ideal for PSC.
"Not as keen on this one[PBC-40]. While it’s more detailed [than the PSC-PRO], I felt the questions are more leading and suggestive. Comparing 'Over the past 7 days, how often were you unable to enjoy life because of your PSC?' with 'Everything in my life is affected by PBC' - the latter is immediately negative, and would lead me to view things badly. However, the former is more objective, asking you to recall the specific bad instances which seems to be a more positive way of looking at it."
"I found the layout confusing. PSC-PRO includes the timeframe in every question. I found I kept having to refer to back to the section description in the SF-36 to remind myself. I very much liked the mix of ‘negative’ and ‘positive’ wording though. Avoids the problem I found in PBC-40."
"I would say certainly PSC-PRO and maybe SF-36 [preferred to PBC-40], but they are quite lengthy."
"PSC-PRO: Good as it covers a wide range of symptoms and impacts. Not ideal having 2 different timescales (24 hours and 7 days) and 2 different numbering systems (1-10 none to very bad and 1-5 never to always) which makes it harder to read and understand.PBC-40: Good as it covers a wide range of symptoms, and impacts. The way the questions are presented makes it easy to read and respond, better in this respect than PSC-PRO I think. Consistent times (four weeks) and answers (never- always) makes it easier to read and understand. I like this the best of the 3.SF-36: This doesn’t cover PSC type symptoms and seems to be a much more general questionnaire on abilities and mental health. The fact that questions are presented in different ways makes it harder to read and understand. Think the PBC-40 is better than this as it’s more specific and is written in a way that’s easier to read and complete."
"PSC-PRO looks good to me but I think If I was being ultra picky I would rather answer PBC-40 as I personally find the circle responses easier to decide on over a 'score'. In regards to the SF-36, I believe that the 'Limitations Of Activities' section is well covered in the other questionnaires but the 'Energy And Emotions' section could be beneficial."
"I think to include all 3 of the quality of life questionnaires would be too much. I do not think the SF -36 is particularly useful when the other 2 are available. If we could have the best of both worlds a combination of the other 2 would be great, however on balance from my own personal experience I think the PBC-40 one expresses better the challenges of living with PSC."
"Overall if I had to pick one questionnaire it would be PBC-40"
"I think some of this [PBC-40] is useful, didnt seem quite as focused as the PSC-PRO one"
"I would not object to completing it [SF-36] but it seems very generalised and would not capture my issues as well."
"At first I couldn’t make my mind up about SF36. It is asking about overall health and emotional wellbeing and ability to carry out tasks, etc… but isn’t as specific as the others. It approaches questions about health from a more ‘high-level’ point of view so thinking about it, it may be a good questionnaire to compliment the others which are more specific in that they address the symptoms more. SF36 – Yes, I think this would be good to complete alongside the chosen itch and fatigue questions."
"Should we use PSC PRO? Yes, the quality of life measures in here cover a lot of areas and have been designed specifically with PSC in mind.
Should we use PBC-40? Yes - The quality of life measures do capture a lot of things relevant to the experience of PSC. Some of the physical symptom measures may not be as important in PSC as PBC (dry eyes) but the measures of fatigue and tiredness look very good.
Should we use SF-36? No. I think PSC PRO and PBC40 give more disease specific measures of quality of life."
"Should we use PSC PRO? I think so. It does give a good idea of how a person with PSC is feeling and dealing with the condition. If the questionnaire is completed on a semi regular basis, then it would give both the specialists and the patient an idea as to how their health is. It's quite easy to get down and just think that everything is getting worse, but when you break it down into separate symptoms and can compare the answers, some issues might have gotten worse while others have improved. It could help a patient. Even a slight improvement somewhere can give someone's mental health a boost. On the other hand, if it's all going down, then it would be easier for a patient to feel very down or even get depressed over it.
Should we use PBC-40? same answer as the previous question, I think it might be good to use the questionnaire.
Should we use SF-36? This questionnaire covers a different time period, some questions relate to the last year, others to the last 4 weeks. It might paint a slightly different picture than the other 2. It probably wouldn't hurt to use this questionnaire."
"...if i had to choose between the 3 presented in the way as shown i would be more inclined to choose PBC-40 or SF-36 as they strike to be less intimidating and it would make me feel more relaxed"
"In the first section about PSC symptoms I’m wondering why the last three options don’t have ‘at its worst’ after them like all the others do. I think the questions help give a rounded description of the impact of PSC and particularly like the sections on emotion impact and quality of life. I prefer it to SF-36."
"PBC-40: I like that it includes questions about the itch such as finding it embarrassing ...and asking about itching so much you bled. I don’t recall these in the questionnaires I’ve already commented on. I feel it captures much of the same information as PSC PRO... If I had to pick one I would pick PSC PRO as I feel it gives more detail than in PBC-40."
"Should we use PSC PRO? Yes, I like this questionnaire, it covers most likely symptoms.
Should we use PBC-40? I am PSC/AIH but felt there were many useful aspects on here...dry mouth, and the itching descriptions.
Should we use SF-36? I think this could be left out."
"If we ask people to do all of them this would take a fair amount of time for them to do and to analyse the data too. Wonder if you would be gaining much as some of them would be very repetitive and may not add too much?"
"I’ve had a look at the questionnaire I really think keep it simple, I personally quite like the VAS scale and the FSS questionnaire otherwise I think people get bogged down. The SF-36 questionnaire Is great but not sure if people will take the time."
"If they were, say, filled in every two weeks (fill one in one week and the other the next week) it might well be not too onerous."
"I think to include all 3 of the quality of life questionnaires would be too much. I do not think the SF -36 is particularly useful when the other 2 are available. If we could have the best of both worlds a combination of the other 2 would be great, however on balance from my own personal experience I think the PBC-40 one expresses better the challenges of living with PSC."
"Would it be too much to include ALL of them (PSC PRO, PBC-40 and SF-36)? I think so."
"If I was asked to complete all 3 surveys, I would feel like there's a lot of repetition. The PBC-40 and SF-36 both ask to think about the last 4 weeks in quite a few questions."
"If we ask people to do all of them this would take a fair amount of time for them to do and to analyse the data too. Wonder if you would be gaining much as some of them would be very repetitive and may not add too much?"
"The bottom line is is I would be happy to answer all the questionnaires if they may help with the trials. They all ask relevant questions and although some are repeated I can't imagine anyone would mind that especially considering the worthy cause!"
"I would be happy to complete all of the forms as I would want to be able to give as clear a picture as possible of how I feel."
"There are a lot of questions and some duplication of questions/areas across the different surveys/scales. But as long as the purpose was explained to me, and I knew that there was going to some duplication of issues being surveyed (fatigue/itching appearing in 3 of the surveys), and how long it would take me to complete all of them (probably 20-30 minutes?) I would be very happy to fill these in as a patient. So, personally I don't think it would be too much..."
"I think anyone who is part of a trial would be willing to put in the work."
The PSC Support 2019 Research Survey revealed that out of 293 people who has said they were interested in taking part in research:
- 83% said they would prefer to complete questionnaires electronically, such as via email, on a website or using mobile/tablet app. 9% said they preferred to complete the questions on paper.
- 85% preferred to complete questionnaires in their own time (e.g. at home). 15% said they would prefer to complete them during their study appointment.
- 99.66% were prepared to log symptoms daily for a week, 90% said they would log them daily for a month, 73% daily for three months and 61% were prepared to log daily symptoms for as long as was needed.
- 78% preferred to complete logs electronically and 22% said they would prefer to complete them on paper.
Patient Reported Outcome Measures in PSC - Focus Group Findings (PSC Support). Original publication date: 29th March 2020