Background
A number of rare conditions such as renal dysplasia, cystinosis, focal segmental glomerulosclerosis, reflux nephropathy, post urethral valves and nephronophthisis may lead to babies being born with poorly developed kidneys or developing significant kidney damage in childhood [10]. These patients may require a renal transplant at some point to improve their chances of survival and quality of life [11]. | |
In the UK, the five-year graft survival rates following the first adult deceased and live donor kidney transplant are 86 and 92% respectively [12]. | |
However, coping with the demands of preserving a renal transplant can be challenging for adolescents especially during the transition from paediatric to adult care [13]. There is a high rate of graft failure and acute or chronic rejection due to poor adherence to prescribed medical regimen [14]. Studies have found that the transitioning process may increase the risk of allograft loss [15]. Rejection episodes may be life threatening to patients and a significant burden to the health system [16]. |
Primary sclerosing cholangitis (PSC) is a progressive disease of the liver and gallbladder characterized by inflammation and scarring of the bile ducts [17]. This may lead to the accumulation of bile which in turn may lead to liver damage and in the long term, cirrhosis, portal hypertension, liver tumours and liver failure [17]. | |
PSC is associated with a high level of morbidity which may have a significant impact on HRQOL [20]. Tiredness or fatigue may be an early symptom while pruritus, jaundice, abdominal pain, weight loss, fevers, hyperpigmentation, vitamin deficiencies and metabolic bone disease may occur as disease progresses [21]. | |
Methods
Setting
Participants
Data collection
Measure | Description |
---|---|
Medical Outcomes Study Short-Form 12 (SF-12) | A 12-item generic HRQOL measure derived from the SF-36 [31, 32]. There are 8 dimensions namely: (i) physical functioning (ii) physical role (iii) bodily pain (iv) general health (v) vitality (vi) social functioning (vii) emotional role (viii) mental health. These can be computed into 2 distinct clusters, PCS-12 and MCS-12 with higher values indicating better HRQOL [31]. |
EuroQOL 5-dimension (EQ-5D) | A utility measure with a self-classifier and a visual analogue scale (VAS) which can be used to value health states [33]. The self-classifier includes 5 dimensions: (i) mobility (ii) self-care (iii) usual activities (iv) pain/discomfort (v) anxiety/depression. Each dimension has 3 levels of severity (no problems, some problems, and severe problems) and it is possible to describe 243 health states between 0 (dead) and 1 (perfect health) [33]. |
Chronic Liver Disease Questionnaire (CLDQ) | A 29-item liver disease–specific HRQOL questionnaire with 6 dimensions [27] namely: (i) abdominal symptoms (ii) activity (iii) emotional function (iv) fatigue (v) systemic symptoms (vi) worry. Summary scores for each domain range from 1 (most impairment) to 7 (least impairment). |
Paediatric Quality of Life Inventory - Transplant Module version 3.0 (PedsQL-TM 3.0) | A 46-item transplant specific module of the PedsQL questionnaire [34] with 8 dimensions namely: 1) about my medicines I (barriers to medical regimen adherence), 2) about my medicines II (medication side effects), 3) my transplant and others (social relationships and transplant), 4) pain and hurt (physical discomfort), 5) worry (worries related to health status), 6) treatment anxiety (fears regarding medical procedures), 7) how I look (impact of transplant on appearance), and 8) communication (communication with medical personnel and others regarding transplant issues) [34]. A 5-point response scale is utilized Items are reverse-scored and linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0) with higher scores indicating better HRQOL [35]. |
Data analysis
Results
Potential benefits of PROMs in the management of rare diseases
“Yeah, that’s what I’m kind of suggesting as well so like I said if you fill it in before then you can do it, discuss it in the consultation … .” (PSC patient 4)
“….you don’t even have to assess that information in clinic because that will take some time but between clinics or post clinic at some point. I am sure clinicians do that.” (PSC patient 1)
“No I feel like that would be stretching it too much, I feel like the Doctors do need to see you regularly. I feel like you need to have that connection with your Consultant because he’s there for you” (Renal transplant patient 1)
Potential benefits of PROMs
“So I feel like it’s really good and like I feel like, if you get more people to fill it in, you’d know how it is for a renal patient live their life … ” (Renal transplant patient 1)
“ … It’s always about having a conversation … So I notice here you’ve put ‘I have no problems walking about’. ‘Can you just tell me a little bit more about that?’ … .if we don’t start the conversation from it, we miss the richness that’s going to come from the patient.” (MDT focus group participant)
“PROMs are important because it impacts the patient’s journey and a patient should feel that us as clinicians are listening to them and we are listening to what concerns them, not just what we see on a screen and concerns us.” (PSC doctor 1)
“ … ..so you get in a habit of always discussing their care with the parent because they know exactly how to pinpoint the problem … .where we have to discuss it with a young person, they will give you monotone answers, not even care about discussing their problem … ..” (MDT focus group participant)
A clinician believed the use of PROMs could influence the approach to patient care by enabling clinicians explore other domains of HRQOL such as psychological wellbeing as well as the physical symptoms.“(referring to patients) … ..they’ll put things down on a sheet that they won’t actually say to you in person.” (MDT focus group participant)
“Er, it’s asking about mood which I think is something that maybe as clinicians we’re not very good at asking our patients about directly, er, we generally tend to say something you know, how are you feeling and it doesn’t really, we maybe don’t go necessarily down into mood and anxiety so this is a good way of picking.” (PSC doctor 6)
“It might be a way of getting other allied health professionals on board” (PSC doctor 4)
Patients and clinicians believed that PROMs could improve symptom reporting and assist with monitoring the evolution of symptoms over time. They also felt PROMs could be used to assess patient response to treatment and the side effects of medication.“This questionnaire is good. It kind of jogs your memory.” (PSC patient 1)
“I think also it would be useful as a tool sort of over time to see how things change for individual patients and to get a better idea of how symptoms progress with time” (PSC doctor 2)
A clinician suggested that PROMs may facilitate the stratification of patients so that the most symptomatic patients are seen by consultants while more stable patients are either seen at nurse-led clinics and/or seen less frequently in clinic.“ … ..a lot of the young adults and older adults do have significant side effects from the drugs, even though our objective markers are good. So I think exploring that further, you know could be really very helpful.” (MDT focus group participant)
“You can use it to improve the care of the patients in clinic … you can stratify if patients have a lot of symptom burden or anxiety or something, you can make sure that they’re seen by the consultant. If someone’s very minimally symptomatic and is actually very well, maybe they could have nurse-led care or you know come less frequently” (PSC doctor 3)
Views on selected PROMs
Views on CLDQ and SF12 (patients with PSC and their clinicians)
“(referring to CLDQ) Yeah, I think that one’s quite good because all those questions are quite relevant to the stuff that I kind of sometimes feel.” (PSC patient 4)
“…the questionnaire (CLDQ) is fine; it’s just, er, whether, there’s no way that says that we know that it relates to PSC.” (PSC patient 2)
“I mean a lot of these things in SF12, apart from question 2 and perhaps 1, a lot of them are related to patient ……..motivation. They don’t really talk about things like cholangitis flares, itching.” (PSC doctor 1)
Patients and clinicians suggested including a free text box where patients can mention other issues not covered in the questionnaire. PSC doctors suggested that patients would prefer questionnaires tailored to their needs and commented on the absence of direct questions about patient fears.“But it’s very generic (referring to SF12), but it’s then, so it’s very easy for someone to fill this in and for you to then compare this with diabetes, obesity, whatever or healthy people, but it’s not liver and it’s not PSC IBD” (PSC doctor 3)
“….it is a good start because it gives you a baseline, but if you really wanted to go the whole way, I think patients would like to have something that is specific to them.” (PSC doctor 3)
“I think what’s missing is the direct, what is the fear that you have of dying, what’s the fear of you of getting colon cancer, what’s the fear of you getting bile duct cancer, and how does that fear manifest itself?” (PSC doctor 3)
Views on PedsQL and EQ-5D (renal transplant recipients and MDT participants)
“I feel like this one’s really good because I can relate to so many things in here…” (Renal transplant patient 1)
Patients suggested adding questions that inquire about patient experience of the receiving a transplant. They also suggested including questions that explore family views or a separate questionnaire for family members.“I think it would be more relevant people who have just recently had their transplants so people who are being discharged from hospital should be given these questionnaires.” (Renal transplant patient 3)
An MDT participant suggested adding questions that went beyond physical concerns and delved into patient fears about their future. This suggestion echoed the point made by a PSC doctor about the need for questions addressing patient fears of developing other conditions in future.“See there could be another survey for parents ….basic questions to see how parents feel as well as the patient, I know you’re trying to see how the patient feels but then again the services could also be developed for parents.” (Renal transplant patient 3)
Concerning the EQ-5D, an MDT participant felt anxiety and depression, which were combined in question number five, were very different concepts. Therefore, responding to the question would be challenging.“I suppose sort of identity, which is difficult to grasp, and also fears for the future, Will I need a transplant in the future? Will my children get this in the future? Will anybody find me attractive in the future? I know you’ve got visible difference here…I suppose are we meeting their unmet needs?” (MDT focus group participant)
“Certainly with the anxiety and the depression one on the EQ5D, anxiety and depression are two really different things, so if you’re ticking one, is it because you’re anxious, because you’re depressed or actually you don’t really know what you’re feeling?” (MDT focus group participant)
Practical considerations for implementation
“I don’t mind paper, but I would prefer electronically. First of all, it’s environmentally-friendly and – it’s also much better to keep the record electronically, isn’t it? less hassle, it’s much quicker, it’s more efficient, more accurate…” (PSC patient 3)
“I think paper based, yeah…” (Transplant patient 1)
A majority of the study participants felt that the local myHealth@QEHB patient portal was suitable and acceptable platform for delivery of ePROMs. myHealth@QEHB is a secure electronic patient records portal developed by the in-house Technical Development and Informatics team at University Hospitals Birmingham NHS Foundation Trust (UHB) [40]. It allows patients in long-term care to remotely access much of their clinical information held at the hospital, including their letters and laboratory results [40].“I think they’re a lot more engaging on an iPad, yeah. I just think they’re a bit more … I honestly think they hate pen and paper” (MDT focus group participant)
Concerning the frequency of administration, participants suggested time intervals ranging from every three months to yearly. The general opinion was that the timing should coincide with the existing frequency of follow-ups appointments.“So we could do it through MyHealth [patient portal], it’s just a bit of a faff for us to do this in the clinic because the patient has to have set up their MyHealth account and then remember their log-in.” (MDT focus group participant)
“… Yeah, I come in every year anyway so I’d be good to do it every year and then you can see what happened last year to the next year can’t you? If it’s getting worse or better…” (PSC patient 4)
“Normally I’d just do it beforehand or like, do you know, while I’m waiting for the Doctor to call me in after my bloods, I’d just complete it then. (Transplant patient 1)
“I think like I said maybe if you do it a couple of weeks before or something and then go through it when we’re there at the consultation and that’d probably be quite good…” (PSC patient 4)
“To get the most honesty out of people, do the questionnaires at the patients’ own comfort and their own space.” (Transplant patient 3)
“….any problems I’d like the consultant at Queen Elizabeth Hospital [to know] they’re marvellous and they sound as though they care.” (PSC patient 2)
“I don’t really mind. I mean, if it helps in research or anything, then, you know, I’m all for it.” (PSC patient 3)
“But it depends what we’re identifying doesn’t it? If it’s, you know my tummy aches when I stand, that’s something that we can say we’ll look into it. If somebody declares they’re literally suicidal, I … we have no … realistic option but to deal with that there and then.” (MDT focus group participant)
“And then with someone with special needs, again you have to decide who is going to advocate for them because, I mean you want to get a true … opinion” (MDT focus group participant)
Patients becoming bored completing the same questionnaires over time and privacy issues were mentioned as issues that warranted careful consideration. A transplant patient, who is on immunosuppression therapy, raised the issue of hygiene if ipads were provided for use in clinic.“And I guess it may be useful for those patients, for whom English is not their first language, to have some of these questions and there, available in their own language.” (PSC doctor 2)
“I wouldn’t mind but I don’t know, it might get a bit boring [laughs], you know what I mean?” (Transplant patient 5)
“I suppose, and obviously…..if … there’d be certain questions that I wouldn’t want somebody to fill in if they felt there was an observer.” (MDT focus participant)
“I have to be very aware of hygiene and stuff so if someone has just used that iPad with a cough or cold I would be very reluctant to use that one.” (Transplant patient 3)
Facilitators and barriers of implementation
“So I guess us being enthusiastic about it [laughs].” (PSC doctor 2)
“If you could reduce the size of the questionnaire, depending on their answers, so you know computer adaptive testing, that would be helpful.” (PSC doctor 5)
A lack of awareness among patients and clinicians of the importance and potential benefits of collecting ePROMs was cited as a potential barrier. The time constraints during clinics could prevent clinicians from acting on ePROM results and this could become a barrier to the use of ePROMs. Patients’ computer literacy levels, language and access to internet or computer or phones were other potential barriers identified.“I suppose the main barrier is that people have very good intentions to fill them out and then just forget…” (PSC doctor 6)
“They won’t understand how it helps them. You might discover that clinicians don’t think it’s important, because they’ll say, well this is just academic, it’s just a way of measuring something that I know, it’s designed to find out more symptoms than are relevant, so they’ll just say, well I don’t think it’s important.” (PSC doctor 3)
“And when you need to stop the consultation for…you need half an hour consultation if somebody reveals that they’re feeling suicidal and you know in the middle of a big transplant clinic … I just think it needs to be thought out, you can’t just introduce it and then just sort of, it’s like an unexploded bomb then.” (MDT focus group participant)
“There will be a group who is not, who aren’t as comfortable, so it’s important to have the other option, as well as the electronic option.” (PSC doctor 5)