PTU-035 Economic impact of non-response to ursodeoxycholic acid in primary biliary cholangitis patients
Bibliographic record
Abstract
Introduction NICE recommends the use of Obeticholic acid (OCA), as a second-line treatment for failed or intolerant ursodeoxycholic acid (UDCA) drug therapy in primary biliary cholangitis (PBC). This audit aims to determine the proportion of PBC patients, in a tertiary referral hospital experiencing failed UDCA drug therapy, to gauge the potential economic impact of a switch to OCA. Methods A total of 120 patients with PBC were identified from an existing patient database. 24 patients were excluded due to inappropriate diagnosis, missing data, non-attendance or failure to tolerate UDCA. Baseline characteristics, UDCA dosage and biochemical response were recorded for all patients. For the purpose of this study, failed UDCA drug therapy was defined as an alkaline phosphatase (ALP) level of greater than 1.67 times the upper limit of normal (ULN) (Toronto Criteria)1. Results Of the 96 patients included for analysis, 9 were male and the remaining 87 were female. The mean age and weight were 64.4± 11.9 years and 75.9± 15.4 kg respectively. The mean blood results for haemoglobin, platelets, bilirubin, ALP, alanine aminotransferase, and creatinine were 126.8± 15.0 g/L, 254.5± 94.3 x10^9/L, 9.4± 4.3 µmol/L, 144.4± 75.6 u/L, 30.3± 19.3 u/L, and 75.1± 28.3 µmol/L respectively. The mean UDCA dose for the cohort was 13.9± 5.9 mg/kg/day. Biochemical failure was documented in 13 patients who had an ALP of greater than 1.67 times the ULN at the end of 1 year of therapy with UDCA (13.5%). Of these 13 patients, 4 were found to be on suboptimal dosage of UDCA (<10 mg/kg/day), leading to a true non-response rate in 9/96 subjects (9.4%). Conclusions A substantial proportion of PBC patients were found to be biochemically unresponsive to UDCA. Shifting these patients to OCA would lead to significant drug expenditure for NHS Grampian. An intermediary step of shifting non-responsive patients to the cheaper drug, Bezafibrate, may prove to be cost-effective, but this drug has been revealed to have no benefit in a recent Cochrane review2. References Kumagi T, Guindi M, Fischer SE, et al. Baseline ductopenia and treatment response predict long-term histological progression in primary biliary cirrhosis. Am J Gastroenterol 2010; 105: 2186–2194. Rudic JS, Poropat G, Krstic MN, Bjelakovic G, Gluud C. Bezafibrate for primary biliary cirrhosis. Cochrane Database of Systematic Reviews 2012, Issue 1. Art. No.: CD009145.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.011 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.007 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".