Patient-reported outcome measures used in patients with primary sclerosing cholangitis: a systematic review

Isa, Fatima; Turner, Grace; Kaur, Gurvinder; Kyte, Derek; Slade, Anita; Pankhurst, Tanya; Kerecuk, Larissa; Keeley, Thomas; Ferguson, James; Calvert, Melanie

doi:10.1186/s12955-018-0951-6

Cited by 15 publications

(20 citation statements)

References 67 publications

(71 reference statements)

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“…Although the CLDQ has been reported to have good test-retest reliability and cross-sectional validity, [52] this should be interpreted with caution as the evidence was obtained from studies that combined the results of patients with PSC with that of patients with primary biliary cirrhosis (PBC) and hepatitis B & C [53,54]. Preliminary validation of the PSC PRO, a new measure developed in accordance with FDA guidelines suggests good psychometric properties [55,56]. The PedsQL-TM has been reported to have good reliability and validity in young patients with transplant [34,35].…”

Section: Discussionmentioning

confidence: 99%

Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study

Aiyegbusi

Isa²,

Kyte

et al. 2020

Health Qual Life Outcomes

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Background: Rare diseases may be life-threatening or chronically debilitating conditions. Patient care needs are often complex and challenging to coordinate and deliver effectively. Rare diseases and their clinical management may therefore substantially impact on patients' health-related quality of life (HRQOL). The use of patient-reported outcome measures (PROMs) may complement clinical assessments by elucidating patients' perspectives on their health status and care priorities. This study explored the opinions of patients and clinicians on the use of PROMs in the management of patients with rare diseases in routine clinical practice. Methods: A total of 15 semi-structured one-to-one interviews were conducted with four patients with primary sclerosing cholangitis (PSC); five renal transplant recipients; and six PSC doctors from University Hospitals Birmingham (UHB) NHS Foundation Trust. A focus group session was also conducted with 10 clinical staff members (doctors, nurses and other allied health professionals from UHB). The suitability and acceptability of the Chronic Liver Disease Questionnaire (CLDQ) and the Short Form 12 (SF12) were assessed by patients with PSC and their doctors while the Paediatric quality of life inventory Transplant Module (PedsQL-TM) and the EuroQoL-5 dimensions (EQ. 5D) were evaluated by the renal transplant recipients and their doctors. The discussions were audio recorded and transcribed verbatim. Coding of the transcripts was done using the Nvivo 11 Plus software. Thematic analysis was conducted to identify the main themes and subthemes.

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Section: Discussionmentioning

confidence: 99%

Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study

Aiyegbusi

Isa²,

Kyte

et al. 2020

Health Qual Life Outcomes

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“…The review found that a wide diversity of PROMs were used to measure HRQoL and symptom burden in patients with PSC. However, the quality of the evidence to support reliability and validity in the most commonly used measures was generally poor and only one had been validated within this population following FDA guidelines [ 70 , 71 ].…”

Section: Introductionmentioning

confidence: 99%

Patient reported outcome measures in rare diseases: a narrative review

Slade

Isa

Kyte

et al. 2018

Orphanet J Rare Dis

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150

161

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BackgroundRare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome measures (PROMs) are used to capture the patient’s views about their health status and facilitate our understanding of the impact of these diseases and their treatments on patient’s quality of life and symptoms.Main textThis review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed. The review highlights potential solutions for some of the issues outlined in the review and the implementation of PROMs in research and clinical practice are discussed.ConclusionPatient input throughout the development of PROMs including qualitative research is essential to ensure that outcomes that matter to people living with rare disease are appropriately captured. Given the large number of rare diseases, small numbers of patients living with each condition and the cost of instrument development, creative and pragmatic solutions to PROM development and use may be necessary. Solutions include qualitative interviews, modern psychometrics and resources such as item banking and computer adaptive testing. Use of PROMs in rare disease research and clinical practice offers the potential to improve patient care and clinical outcomes.

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“…There was therefore a lack of clarity about how QoL had been measured in this population, which factors were important determinants of QoL in PSC, and how the condition affected QoL. Four more reviews by other groups have since been published, and were identified in later updates to the initial search: a systematic review assessing PROMs used in PSC [ 22 ], a systematic review identifying PRO instruments and concepts used in the PSC literature [ 23 ], a literature review exploring QoL in cholestatic disease [ 24 ], and a scoping review exploring the impact of PSC on psychological well-being [ 25 ].…”

Section: Introductionmentioning

confidence: 99%

Quality of life in primary sclerosing cholangitis: a systematic review

Marcus

Stone

Krooupa

et al. 2021

Health Qual Life Outcomes

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Background Primary sclerosing cholangitis (PSC) is a rare bile duct and liver disease which can considerably impact quality of life (QoL). As part of a project developing a measure of QoL for people with PSC, we conducted a systematic review with four review questions. The first of these questions overlaps with a recently published systematic review, so this paper reports on the last three of our initial four questions: (A) How does QoL in PSC compare with other groups?, (B) Which attributes/factors are associated with impaired QoL in PSC?, (C) Which interventions are effective in improving QoL in people with PSC?. Methods We systematically searched five databases from inception to 1 November 2020 and assessed the methodological quality of included studies using standard checklists. Results We identified 28 studies: 17 for (A), ten for (B), and nine for (C). Limited evidence was found for all review questions, with few studies included in each comparison, and small sample sizes. The limited evidence available indicated poorer QoL for people with PSC compared with healthy controls, but findings were mixed for comparisons with the general population. QoL outcomes in PSC were comparable to other chronic conditions. Itch, pain, jaundice, severity of inflammatory bowel disease, liver cirrhosis, and large-duct PSC were all associated with impaired QoL. No associations were found between QoL and PSC severity measured with surrogate markers of disease progression or one of three prognostic scoring systems. No interventions were found to improve QoL outcomes. Conclusion The limited findings from included studies suggest that markers of disease progression used in clinical trials may not reflect the experiences of people with PSC. This highlights the importance for clinical research studies to assess QoL alongside clinical and laboratory-based outcomes. A valid and responsive PSC-specific measure of QoL, to adequately capture all issues of importance to people with PSC, would therefore be helpful for clinical research studies.

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Patient-reported outcome measures used in patients with primary sclerosing cholangitis: a systematic review

Cited by 15 publications

References 67 publications

Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study

Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study

Patient reported outcome measures in rare diseases: a narrative review

Quality of life in primary sclerosing cholangitis: a systematic review

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