Background and Aims: Primary sclerosing cholangitis (PSC) is an enigmatic disease with scarce therapeutic options, potentially evolving to severe and life-threatening complications, including malignancies like cholangiocarcinoma (CCA) or colon cancer. The clinical management of PSC remains challenging and may benefit from identification of outcome indicators to assess the quality of care. This study aims to: (A) identify Outcome Indicators (OIs) for PSC, and (B) validate OIs in a clinical context with a preliminary interim analysis. 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 (Value-based Medicine in Hepatology, VBMH). Results: Five OIs were identified on the basis of the highest rating values and disagreement indexes next to 0. They include: annual rate of acute cholangitis episodes (OI#1); mortality rate for patients not yet listed for liver transplantation (OI#2); rate of quality of life improvement, 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 according to 3 levels of severity) (OI#3); number of patients died for CCA and CRC (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 tertiary 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, OI#4: no patients died without being listed for transplantation or because of cancer during study time; OI#3: 38.9% and 19.4% of patients showed an improvement in EQ-VAS and EQ-5D parameters, respectively; OI#5: 3% of patients developed or worsened osteoporosis. Conclusions: Five OIs for PSC were identified on a highly shared consensus. Albeit the study population is small (as in the case of rare diseases) 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.

Fabris, L., Ciaccio, A., Okolicsanyi, S., Rota, M., Cortesi, P., Buonocore, M., et al. (2015). P1169 : Outcome indicators in primary sclerosing cholangitis: Interim analysis of the value-based medicine in hepatology study. JOURNAL OF HEPATOLOGY, 62(Suppl 2), S791-S791 [10.1016/S0168-8278(15)31365-9].

P1169 : Outcome indicators in primary sclerosing cholangitis: Interim analysis of the value-based medicine in hepatology study

CIACCIO, ANTONIO
Secondo
;
OKOLICSANYI, STEFANO;ROTA, MATTEO;CORTESI, PAOLO ANGELO;GEMMA, MARTA;Fagiuoli, S;SCALONE, LUCIANA;VALSECCHI, MARIA GRAZIA;MANTOVANI, LORENZO GIOVANNI
Penultimo
;
STRAZZABOSCO, MARIO
Ultimo
2015

Abstract

Background and Aims: Primary sclerosing cholangitis (PSC) is an enigmatic disease with scarce therapeutic options, potentially evolving to severe and life-threatening complications, including malignancies like cholangiocarcinoma (CCA) or colon cancer. The clinical management of PSC remains challenging and may benefit from identification of outcome indicators to assess the quality of care. This study aims to: (A) identify Outcome Indicators (OIs) for PSC, and (B) validate OIs in a clinical context with a preliminary interim analysis. 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 (Value-based Medicine in Hepatology, VBMH). Results: Five OIs were identified on the basis of the highest rating values and disagreement indexes next to 0. They include: annual rate of acute cholangitis episodes (OI#1); mortality rate for patients not yet listed for liver transplantation (OI#2); rate of quality of life improvement, 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 according to 3 levels of severity) (OI#3); number of patients died for CCA and CRC (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 tertiary 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, OI#4: no patients died without being listed for transplantation or because of cancer during study time; OI#3: 38.9% and 19.4% of patients showed an improvement in EQ-VAS and EQ-5D parameters, respectively; OI#5: 3% of patients developed or worsened osteoporosis. Conclusions: Five OIs for PSC were identified on a highly shared consensus. Albeit the study population is small (as in the case of rare diseases) 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.
No
Abstract in rivista
Scientifica
outcome indicators; primary sclerosing cholangitis; value-based medicine; health care
English
Poster n° P1169 presented at the 50th annual meeting of the EASL (“European Association for the Study of the Liver”). London (United Kingdom), 22-26 April 2015
Fabris, L., Ciaccio, A., Okolicsanyi, S., Rota, M., Cortesi, P., Buonocore, M., et al. (2015). P1169 : Outcome indicators in primary sclerosing cholangitis: Interim analysis of the value-based medicine in hepatology study. JOURNAL OF HEPATOLOGY, 62(Suppl 2), S791-S791 [10.1016/S0168-8278(15)31365-9].
Fabris, L; Ciaccio, A; Okolicsanyi, S; Rota, M; Cortesi, P; Buonocore, M; Gemma, M; Giani, P; Belli, L; Fagiuoli, S; Scalone, L; Valsecchi, M; Mantovani, L; Strazzabosco, M
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/10281/83980
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