Outcome Indicators in Primary Sclerosing Cholangitis: Interim Analysis Of The Value-Based Medicine In Hepatology (VBMH) Study

Primary sclerosing cholangitis (PSC) is an enigmatic disease with scarce therapeutic options, potentially evolving in life-threatening complications, including cholangiocarcinoma (CCA). The clinical management of PSC remains challenging and will benefit from identification of outcome indicators to assess the quality of care and of the outcomes. This study aims at: A) identifying Outcome Indicators (OIs) for PSC, and B) validating OIs in a real-life context, by performing an interim analysis of the VBMH study. METHODS. A) A panel of experts generated a list of OIs using a modified version of the Delphi method. B) OIs with the highest RAND/UCLA score were tested in an ongoing multicentric and prospective study (VBMH). RESULTS. Five OIs were identified on the basis of the highest rating values (close to 9) and the lowest disagreement indexes (next to 0). They include: annual incidence of acute cholangitis episodes (OI#1); mortality rate for patients not yet listed for LTx (OI#2); improvement in patient reported quality of life measured by EQ-VAS (a visual assessment scale ranging from 0 to 100 where the patient points out his present-day health status) and EQ-5D (assessment of 5 domains that measure daily performance: mobility, self care, anxiety/depression, usual activities and pain/discomforts) (OI#3); number of patients died for CCA or colorectal cancer (OI#4); incidence and/or worsening of osteoporosis, expressed as T-score differential over a 2-year interval (OI#5). In the validation study, 63 consecutive patients with PSC enrolled in 3 liver centres in Northern Italy were evaluated for a 24-month follow-up period. For each OI, the following values were reported: OI#1) cumulative incidence of 5.2%, resulting in 0.029 cholangitis/patient; OI#2 and OI#4) no patients died without being listed for transplantation or because of cancer during the study time; OI#3) 38.9 and 19.4% of patients showed an improvement in EQ-VAS and EQ-5D, respectively; OI#5) 3% of patients developed or worsened osteoporosis. In CONCLUSION, five OIs for PSC were identified using the Delphi method and received a highly shared consensus. Albeit the study population is small (consistent with the rarity of PSC) and the follow-up time is short as compared to the long natural history of the disease, these OIs have proven to be easy to collect and to work appropriately. Therefore, they are suitable to be extended to specialized centres involved in PSC management to further validate their clinical usefulness in a larger scale. Patient reported outcomes showed improvements in a significant proportion of patients, indicating the importance to refer these patients to specialized centers