Effects of Primary Biliary Cholangitis on Quality of Life and Health Care Costs in the United Kingdom

Rice, Stephen; Albani, Viviana; Minos, Dimitrios; Fattakhova, Gulnar; Mells, George; Carbone, Marco; Flack, Steven; Varvaropoulou, Nikoletta; Badrock, Jonathan; Spicer, Ann; Sandford, Richard; Shirley, Mark; Coughlan, Diarmuid; Hirschfield, Gideon; Taylor-Robinson, Simon D; Vale, Luke; Jones, David

doi:10.1016/j.cgh.2020.06.025

Cited by 11 publications

(11 citation statements)

References 22 publications

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“…Patients presenting with symptoms are typically younger and may have more aggressive disease [ 6 ], whilst fatigue related to PBC is associated with increased all-cause mortality [ 7 ]. Recent data exploring health utility confirms that the greatest impact on functional status in PBC comes from fatigue and cognitive symptoms [ 8 ], with resultant social isolation giving rise to additional burdens and worsening quality of life [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

“…Cognitive symptoms remain a significant problem for many patients with PBC, with a detrimental impact on quality of life [ 8 , 35 , 36 ], no effective treatments [ 21 , 37 ], and limited understanding of the underlying pathogenic mechanisms [ 38 ]. In this study, the relationship between PBC-associated cognitive symptoms and allopregnanolone was explored to examine whether this potentially modifiable pathway could be a target for novel therapies.…”

Section: Introductionmentioning

confidence: 99%

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Neurosteroid Activation of GABA-A Receptors: A Potential Treatment Target for Symptoms in Primary Biliary Cholangitis?

Wetten

Ogle

Mells

et al. 2022

Canadian Journal of Gastroenterology and Hepatology

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Background and Aims. A third of patients with primary biliary cholangitis (PBC) experience poorly understood cognitive symptoms, with a significant impact on quality of life (QOL), and no effective medical treatment. Allopregnanolone, a neurosteroid, is a positive allosteric modulator of gamma-aminobutyricacid-A (GABA-A) receptors, associated with disordered mood, cognition, and memory. This study explored associations between allopregnanolone and a disease-specific QOL scoring system (PBC-40) in PBC patients. Method. Serum allopregnanolone levels were measured in 120 phenotyped PBC patients and 40 age and gender-matched healthy controls. PBC subjects completed the PBC-40 at recruitment. Serum allopregnanolone levels were compared across PBC-40 domains for those with none/mild symptoms versus severe symptoms. Results. There were no overall differences in allopregnanolone levels between healthy controls (median = 0.03 ng/ml (IQR = 0.025)) and PBC patients (0.031 (0.42), p = 0.42 ). Within the PBC cohort, higher allopregnanolone levels were observed in younger patients (r (120) = −0.53, p < 0.001 ) but not healthy controls (r (39) = −0.21, p = 0.21 ). Allopregnanolone levels were elevated in the PBC-40 domains, cognition (u = 1034, p = 0.02 ), emotional (u = 1374, p = 0.004 ), and itch (u = 795, p = 0.03 ). Severe cognitive symptoms associated with a younger age: severe (50 (12)) vs. none (60 (13); u = 423 p = 0.001 ). Conclusion. Elevated serum allopregnanolone is associated with severe cognitive, emotional, and itch symptoms in PBC, in keeping with its known action on GABA-A receptors. Existing novel compounds targeting allopregnanolone could offer new therapies in severely symptomatic PBC, satisfying a significant unmet need.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Neurosteroid Activation of GABA-A Receptors: A Potential Treatment Target for Symptoms in Primary Biliary Cholangitis?

Wetten

Ogle

Mells

et al. 2022

Canadian Journal of Gastroenterology and Hepatology

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“…Disease registries for RDs are now being used not only to understand the patient journey and the life cycle of the disease but also to shape the care of patients and provide insights into the available treatments. For example, some of the outcomes of the United Kingdom Primary Biliary Cholangitis (UK-PBC) registry include the development of financial models for healthcare costs for patients with PBC, 18 assessment of the quality of life of patients 19 and the exploration of new pharmacotherapy targets for this rare condition. 20 Similarly, registries for α1-antitrypsin deficiency are looking to understand the natural history of the disease across its different phenotypes and to provide insights into the effectiveness of augmentation therapy as a treatment modality for this cohort of patients.…”

Section: Introductionmentioning

confidence: 99%

Defining and characterising a toolkit for the development of a successful European registry for rare liver diseases: a model for building a rare disease registry

et al. 2022

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IntroductionA rare disease is defined by the European Health Commission as a disorder affecting less than 5/10,000 of the population. There are at least 20 rare liver diseases (RLDs) seen frequently in the adult and paediatric liver clinic, signifying that the hepatology community can be influential in developing such patient databases for registering patients with rare hepatic conditions. The aim of this review was, first, to identify registries for RLDs in Europe, and, second, to design a universal blueprint for the development of a registry for RLD by using lessons learnt from the European registries that have already been established. MethodsWe searched PubMed, Google Scholar and clinicaltrials.gov using the MESH terms 'registries', 'database management systems', 'database' and the non-MESH terms 'database$', 'registry', 'repository' and 'repositories'. We only included studies in English from countries/consortia of the European Union (EU). Our literature search was performed in 2020. ResultsWe identified 37 registries for RLDs in Europe. Using information from the design of these registries we designed a blueprint for the development of a patient registry for an RLD consisting of a theoretical, technical and maintenance phase. DiscussionIt is believed that rare diseases may affect as much as 6-8% of the EU population across its 28 member states. Here we have provided a toolkit for designing a registry for an RLD. Our article will complement the efforts of loco-regional, national and international groups seeking to establish robust systems for data collection and analysis for orphan liver diseases.

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“…quality of life (QoL) of the patients. [1][2][3][4] Recently, the prevalence and incidence of PBC have been increasing in the Asia-Pacific region, with a relatively high prevalence in China. 5 Ursodeoxycholic acid (UDCA) is currently the only first-line treatment for PBC.…”

mentioning

confidence: 99%

Differences in the perceptions of patients with primary biliary cholangitis and physicians from various hospital departments: An online survey

Liu,

Jia,

Ding

et al. 2024

J of Digest Diseases

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ObjectivesPrimary biliary cholangitis (PBC) is a chronic autoimmune liver disease that affects the quality of life (QoL) of patients. This study aimed to evaluate the differences in perceptions of PBC among physicians from different hospital departments and patients with PBC.MethodsAn online survey regarding the general knowledge, diagnosis, and management of PBC was completed by physicians and patients.ResultsA total of 239 patients with PBC and 239 physicians from eight hospital departments (gastroenterology, infectious diseases, rheumatology, hepatobiliary surgery, pathology, clinical laboratory, ultrasound, and radiology) completed the survey. The results showed that physicians from departments other than gastroenterologists and rheumatologists lacked knowledge of PBC, and that junior gastroenterologists were uncertain about the diagnostic and treatment pathways of PBC. Importantly, the lack of knowledge significantly impacted the QoL of patients, especially the emotional scores of PBC‐40 (odds ratio −2.556, 95% confidence interval −3.852 to −1.260, P < 0.001). In addition, there was a perceived knowledge gap between patients and gastroenterologists.ConclusionsPhysicians must improve their awareness of PBC. Patient education and patient–physician communication are important for improving the patient's QoL.

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Effects of Primary Biliary Cholangitis on Quality of Life and Health Care Costs in the United Kingdom

Cited by 11 publications

References 22 publications

Neurosteroid Activation of GABA-A Receptors: A Potential Treatment Target for Symptoms in Primary Biliary Cholangitis?

Neurosteroid Activation of GABA-A Receptors: A Potential Treatment Target for Symptoms in Primary Biliary Cholangitis?

Defining and characterising a toolkit for the development of a successful European registry for rare liver diseases: a model for building a rare disease registry

Differences in the perceptions of patients with primary biliary cholangitis and physicians from various hospital departments: An online survey

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