Patient ownership of primary biliary cholangitis long-term management

Abstract

ObjectivePatient ownership of disease is vital in rare diseases like primary biliary cholangitis (PBC). This survey of UK members of the PBC foundation aimed to assess patients’ perception of their disease management, focusing on key biomarkers and problematic symptoms.DesignRegistered PBC foundation members were surveyed on their experiences on their most recent clinic visit, covering the type of hospital and clinician and whether biochemical response and symptom burden were discussed, including who initiated these conversations. Respondents were also asked about their willingness to initiate these conversations.ResultsAcross 633 respondents, 42% remembered discussing alkaline phosphatase, the key biochemical response measure, and the majority of discussions were initiated by the healthcare provider. 56% of respondents remembered discussing itch, a key PBC symptom. There was no distinction between the grade of healthcare professional, but both patients and clinicians were significantly more likely to discuss symptoms over disease progression. Reassuringly, 84% of respondents felt willing to initiate conversations about their illness, regardless of the grade of managing clinician.ConclusionsThis work lays a positive foundation for patient education and empowerment projects, likely to improve clinical outcomes. Key aspects of management (biochemical response to treatment and symptom burden) should be emphasised as topics of discussion to both patients and clinicians managing PBC. We suggest a simple cue card to prompt patient-led discussion.

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

“…A previous study showed that patients were more inclined to rely on their physicians and ignored their own role in the management of disease. 12 However, as with many other chronic diseases, patient ownership is crucial. 21,25 Leighton et al suggested a cue card to prompt patient-led discussion regarding ALP levels with their physicians, 12 which may be used initially to encourage patients to engage in disease management and later expanded progressively to cover more content.…”

“…However, only a minority of patients discuss their ALP value, which is the most important biochemical indicator of PBC, with their physicians. 12 Moreover, a few patients request an improved relationships with their doctors. 13 The lack of communication between doctors and patients inevitably leads to a different understanding of the disease and its management.…”

Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study

“…Improvement in pruritus has been previously observed with treatment with seladelpar 10 mg QD through 1 year. [20] Pruritus is a significant burden for patients with PBC that is often undertreated, [28][29][30] suggesting that seladelpar has the potential to add this benefit to that of improving biomarkers associated with disease progression. The PBC-40 total score, which sums diverse individual quality-of-life domain scores, did not improve when examined for the entire population at month 3.…”

“…ENHANCE Study Group: Raul Adrover, 1 Saurabh Agrawal, 2 Elmar Aigner 3 Agustin Albillos Martinez, 4 Abu-Mouch Saif Alden, 5 Raul Jesús Andrade Bellido, 6 Marco Antonio Arrese Jiménez, 7 Walid Ayoub, 8 Seth J. Baum, 9 Ziv Ben-Ari, 10 Marina Berenguer, 11 Christoph Berg, 12 Fernando Oscar Bessone, 13 Alan Bonder, 14 Brian B. Borg, 15 Carlos Gustavo Bresky Ruiz, 16 Peter Buggisch, 17 Jose Luis Calleja Panero, 18 Elizabeth J. Carey, 19 Michal Carmiel-Haggai, 20 Francesca Carubbi, 21 Pilar Castillo Grau, 22 Chin Lye Ch'ng, 23 Nicoleta-Claudia Cimpoeru, 24 Raúl Contreras Omaña, 25 Lynsey Corless, 26 Charlotte Costentin, 27 Marc Deschênes, 28 Yvonne Dörffel, 29 Predrag Dugalic, 30 Geoffrey Charles Farrell, 31 José Luis Fernández, 32 Annarosa Floreani, 33 Sven Francque, 34 Bradley L. Freilich, 35 Francisco Alejandro Fuster Saldias, 36 Michael R. Galambos, 37 Joseph Galati, 38 Andrea Galli, 39 Nathalie Ganne-Carrie, 40 Natalia Geyvandova, 41 Liliana-Simona Gheorghe, 42 Richard Gilroy, 43 Aparna Goel, 44 Tobias Goeser, 45 Susan Greenbloom, 46 Waldemar Halota, 47 Stephen A. Harrison, 48 Marek Hartleb, 49 Jeong Heo, 50 Harald Hofer, 51 Gábor Horváth, 52 Jonathan C. Huang, 53 Jason Lee Huffman, 54 Béla Hunyady, 55 Steven Johnson, 56 Yiannis Kallis, 57 Arun Khazanchi, 58 Kyung-Ah Kim, 59 Seung Up Kim, 60 Yoon Jun Kim, 61 Anita Kohli, 62 Nicholas Kontorinis, 63 John R. Lake, 64 Kwan Sik Lee, 65 Tomasz Mach, 66 Richard Manch, 67 Yaakov Maor-Kendler, ...…”

The relationship between disease activity and UDCA response criteria in primary biliary cholangitis: A cohort study

“…This may have contributed to the lack of understanding, on behalf of both clinicians and patients, regarding optimal management of PBC. 7 , 8 All the current UDCA response criteria also accept some degree of ongoing liver blood biochemistry abnormality (typically of alkaline phosphatase) in “responding” patients. Recently, the Global PBC Study Group proposed that the biochemical cut-off for UDCA response should be the upper limit of normal (ULN) for alkaline phosphatase and bilirubin, or even 0.6 times the ULN of bilirubin together with a normal alkaline phosphatase, as any degree of ongoing liver biochemistry abnormality is associated with an increased risk of adverse outcomes.…”