Prospective evaluation of <scp>PBC</scp>‐specific health‐related quality of life questionnaires in patients with primary sclerosing cholangitis

Raszeja‐Wyszomirska, Joanna; Krawczyk, Marek; Rigopoulou, Eirini I.; Bogdanos, Dimitrios P.; Milkiewicz, Piotr

doi:10.1111/liv.12730

Cited by 32 publications

(58 citation statements)

References 30 publications

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“…Few reports have already indicated the negative impact of PSC on HRQoL [3,4]. In our previous study, we observed an impairment in quality of life for patients with PSC compared to healthy individuals, and our data highlighted a significant impact of female gender in predicting worse quality of life [2]. Our current project increases our knowledge on the impact of genetic variations in PSC on patients’ well-being, as this is the first study the focus on HRQoL assessment in this context.…”

Section: Discussionsupporting

confidence: 56%

“…Both questionnaires were recently validated by our group for use in PSC [2]. PBC-40 consists 40 questions in 5 domains including Cognition , Itch , Fatigue , Social-Emotional and Other Symptoms that are assessed using a five-point scale (1 = never to 5 = always), with higher scores denoting a greater symptom impact and poorer HRQoL.…”

Section: Methodsmentioning

confidence: 99%

“…It occurs predominantly in males and often remains asymptomatic in the early stages of the disease. Nevertheless, progressing biliary tree damage ultimately leads to chronic cholestasis, recurrent cholangitis and liver cirrhosis in a high proportion of affected individuals [1], impairing the health-related quality of life (HRQoL)[2–4]. Moreover, patients with PSC are at an increased risk of cholangiocarcinoma, a primary biliary cancer with a fatal prognosis [5].…”

Section: Introductionmentioning

confidence: 99%

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ApaI polymorphism of vitamin D receptor affects health-related quality of life in patients with primary sclerosing cholangitis

Kempińska-Podhorodecka

Milkiewicz

Jabłoński

et al. 2017

PLoS ONE

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BackgroundPolymorphisms of vitamin D receptor (VDR) contribute to the pathogenesis of multiple autoimmune conditions.MethodsWe investigated the incidence of VDR polymorphisms (rs1544410-BsmI; rs7975232-ApaI; rs731236-TaqI) in a group of patients with primary sclerosing cholangitis (PSC, n = 275) and in healthy controls (n = 376). Additionally, correlations of the VDR polymorphisms with clinical and biochemical factors of the disease were analysed.ResultsThe genotype and allele distributions of these polymorphisms in PSC patients were similar to those observed in controls. However, the ApaI polymorphism was associated with an impaired health-related quality of life (HRQoL). The generic SF-36 questionnaire showed that the Role-Physical (p = 0.01), Role-Emotional (p = 0.01), Physical Component Summary (p = 0.01) and Mental Component Summary (p = 0.003) scores were significantly affected. Similarly, the disease-specific questionnaires, PBC-40 and PBC-27, demonstrated that carriers of the C allele suffered from more severe Itch (p = 0.03 assessed by PBC-40 and PBC-27), more Fatigue (p = 0.02 assessed by PBC-40 and PBC-27) and Impaired Cognitive Capacity (p = 0.04 and p = 0.03). Correspondingly, individuals who were AA homozygotes (non-carriers of the C allele of ApaI) had higher summary scores for the Physical (p = 0.01) and Mental Components (p = 0.006) measured with SF-36. Moreover, they experienced less itch (p = 0.03) and less Fatigue (p = 0.03) and had better Cognitive Abilities (p = 0.04) as assessed by the PBC-40 and PBC-27 questionnaires. No associations between other VDR polymorphisms and clinical or laboratory findings were made.ConclusionIn summary, this study is the first to show that the ApaI polymorphisms in VDR may exert an effect on disease-related symptoms and quality of life in patients with PSC.

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Section: Discussionsupporting

confidence: 56%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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ApaI polymorphism of vitamin D receptor affects health-related quality of life in patients with primary sclerosing cholangitis

Kempińska-Podhorodecka

Milkiewicz

Jabłoński

et al. 2017

PLoS ONE

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“…In a small, retrospective, case–control study, the presence of PSC in patients with IBD did not significantly impair HRQOL but was associated with higher rate of work disability . However, two more recent and larger studies demonstrated a significant association between PSC and impaired HRQOL . Older age, female gender, large duct disease, severity of disease, concomitant IBD and systemic symptoms (eg pruritus, fatigue, ascites) have all been identified as strong predictors of impaired HRQL in patients with PSC (Figure ) .…”

Section: Primary Sclerosing Cholangitismentioning

confidence: 99%

“…Of note, the PBC‐40 and PBC‐27 questionnaires are PBC‐specific validated measures used to quantify HRQOL in patients with PBC, with the latter being shorter but associated with somewhat lower sensitivity in the assessment of cognition and fatigue severity . The PBC‐40 and PBC‐27 may also be potentially used for assessment of HRQOL in PSC …”

Section: Primary Biliary Cholangitismentioning

confidence: 99%

Complications, symptoms, quality of life and pregnancy in cholestatic liver disease

Zakharia

Tabibian

Lindor

et al. 2017

Liver International

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Cholestatic liver diseases (CLDs) encompass a variety of disorders of bile formation and/or flow which generally result in progressive hepatobiliary injury and ultimately end-stage liver disease. Many patients with CLD are diagnosed between the ages of 20-50 years, a particularly productive period of life professionally, biologically and in other respects; it is not surprising, thus, that CLD is often associated with impaired health-related quality of life (HRQOL) and uncertainty regarding implications for and outcomes of pregnancy. Primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC) are the most prominent CLDs, both having considerable morbidity and mortality and representing major indications for liver transplantation. These disorders, as a consequence of their complications (eg ascites, hepatic osteodystrophy), associated conditions (eg inflammatory bowel disease) and symptoms (eg pruritus and fatigue), can significantly impair an array of domains of HRQOL. Here we review these impactful clinical aspects of PSC and PBC as well as the topics of fertility and pregnancy.

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A Systematic Review of Patient‐Reported Outcomes in Primary Biliary Cholangitis and Primary Sclerosing Cholangitis

et al. 2020

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Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are associated with decreased health-related quality of life and debilitating symptoms. These experiences can be defined as patient-reported outcome (PRO) concepts and measured using PRO instruments. We identified all PRO concepts and instruments used in the PBC and PSC literature. This systematic review identified PBC and/or PSC studies from January 1, 1990, to May 6, 2019, that measured at least one PRO concept. Study population, design, PRO concept, PRO instrument, and validation data for PRO instruments were investigated. We provided descriptive statistics of PRO concepts and instruments used, stratified by population type. Use of PRO concepts and instruments were assessed over time. The search yielded 318 articles (69% in PBC, 18% in PSC, 13% in both, and 24% in drug trials). Forty-nine unique PRO concepts were identified. The five most common PRO concepts included pruritus (25%), fatigue (19%), broad health-related quality of life (16%), gastrointestinal adverse events (6%), and physical adverse events (6%). Only 60% of PRO concepts were measured with a PRO instrument, most of which were nonvalidated visual analogue or numeric rating scales. Only three of 83 PRO instruments were developed with feedback from the target populations (one for PBC, one for PSC, and one for both), and only six documented any psychometric testing in the target populations. Use of PRO instruments increased over time from 30% in the 1990s to 67% by 2019. Conclusion: The overwhelming majority of PRO instruments used in PBC/PSC were nonspecific and lacked patient validation or empirical justification. Significant opportunities exist to use qualitative methods to better understand patient experiences, and translate this knowledge into meaningful, patient-driven study outcomes. (Hepatology Communications 2020;4:1502-1515). P rimary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are chronic cholestatic liver diseases associated with significant morbidity, including decreased quality of life, (1-6) pruritus, (7-9) and fatigue, (10-14) among other symptoms. The primary outcomes of most clinical trials include disease endpoints or surrogates (e.g., alkaline phosphatase), rather than patient-reported outcomes (PROs), which capture how patients feel or function. PROs reflect a patient's health status, as it comes directly from the patient, without interpretation by anyone else including the clinician. (15) Patient experiences and perceptions are best evaluated using validated PRO instruments that were developed with Abbreviations: FDA, Food and Drug Administration; HRQOL, health-related quality of life; NOS, not otherwise specified; PBC, primary biliary cholangitis; PRO, patient-reported outcome; PSC, primary sclerosing cholangitis.

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Prospective evaluation of PBC‐specific health‐related quality of life questionnaires in patients with primary sclerosing cholangitis

Abstract: This is the first study directly comparing PBC-40, PBC-27 and SF-36 in PSC. PSC patients, especially females, show HRQoL impairment. PBC-40 and PBC-27 questionnaires could be of potential use for HRQoL assessment in PSC.

Cited by 32 publications

References 30 publications

ApaI polymorphism of vitamin D receptor affects health-related quality of life in patients with primary sclerosing cholangitis

ApaI polymorphism of vitamin D receptor affects health-related quality of life in patients with primary sclerosing cholangitis

Complications, symptoms, quality of life and pregnancy in cholestatic liver disease

A Systematic Review of Patient‐Reported Outcomes in Primary Biliary Cholangitis and Primary Sclerosing Cholangitis

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