Introduction: Primary sclerosing cholangitis (PSC) is an enigmatic disease with scarce therapeutic options. The clinical management of PSC remains challenging and may benefit from Outcome Indicators (OI) to assess the quality of care. Aims: This study aims to: (A) identify OIs for PSC, and (B) validate OIs in a clinical context. Methods: (A) A panel of experts generated a list of OIs by Delphi method. (B) OIs with the highest RAND/UCLA score were tested in an ongoing multicentric, prospective study (Value-based Medicine in Hepatology, VBMH). Results: Five OIs were identified having the highest rating values and low disagreement indexes: 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 and EQ-5D (OI#3); number of patients died for cholangiocarcinoma and colo-rectal carcinoma (OI#4); incidence and/or worsening of osteoporosis (OI#5). In the validation study, 63 consecutive patients with PSC enrolled in 3 tertiary centres in Northern Italy were evaluated for a median 24-months 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 reporting high 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). Outcome indicators in primary sclerosing cholangitis: Interim analysis of the value-based medicine in hepatology study. DIGESTIVE AND LIVER DISEASE, 47(Suppl 1), e16-e16 [10.1016/j.dld.2015.01.037].

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

Introduction: Primary sclerosing cholangitis (PSC) is an enigmatic disease with scarce therapeutic options. The clinical management of PSC remains challenging and may benefit from Outcome Indicators (OI) to assess the quality of care. Aims: This study aims to: (A) identify OIs for PSC, and (B) validate OIs in a clinical context. Methods: (A) A panel of experts generated a list of OIs by Delphi method. (B) OIs with the highest RAND/UCLA score were tested in an ongoing multicentric, prospective study (Value-based Medicine in Hepatology, VBMH). Results: Five OIs were identified having the highest rating values and low disagreement indexes: 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 and EQ-5D (OI#3); number of patients died for cholangiocarcinoma and colo-rectal carcinoma (OI#4); incidence and/or worsening of osteoporosis (OI#5). In the validation study, 63 consecutive patients with PSC enrolled in 3 tertiary centres in Northern Italy were evaluated for a median 24-months 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 reporting high 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
Oral contribution presented at the 48th national congress of the AISF (“Associazione Italiana per lo Studio del Fegato”). Roma (Italy), 19-20 February 2015
Fabris, L., Ciaccio, A., Okolicsanyi, S., Rota, M., Cortesi, P., Buonocore, M., et al. (2015). Outcome indicators in primary sclerosing cholangitis: Interim analysis of the value-based medicine in hepatology study. DIGESTIVE AND LIVER DISEASE, 47(Suppl 1), e16-e16 [10.1016/j.dld.2015.01.037].
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
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/10281/77629
Citazioni
  • Scopus ND
  • ???jsp.display-item.citation.isi??? 0
Social impact