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Expert Review of Pharmacoeconomics & Outcomes Research Volume 21, 2021 - Issue 2
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Study protocol

Burden of illness of progressive familial intrahepatic cholestasis in the US, UK, France, and Germany: study rationale and protocol of the PICTURE study

Leonardo Ruiz-Casasa HCD Economics, Madrid, SpainView further author information
,
Sonia O’Harab HCD Economics, Daresbury, UKORCID Iconhttps://orcid.org/0000-0002-9119-8336View further author information
ORCID Icon,
Claudia Mighiub HCD Economics, Daresbury, UKCorrespondence[email protected]
View further author information
,
Alan Finneganc Faculty of Health and Social Care, University of Chester, UKView further author information
,
Alison Taylord Children’s Liver Disease Foundation, Birmingham, UKView further author information
,
Emily Venturae PFIC Advocacy and Resource Network, KY, United StatesView further author information
,
Anil Dhawanf Pediatric Liver GI and Nutrition Center, King’s College Hospital, London, UKView further author information
,
Karen F Murrayg Pediatric Institute, Cleveland Clinic Children’s Hospital, Cleveland, OH, United StatesView further author information
,
Jorn Schattenbergh University Medical Center of the Johannes Gutenberg-University, Mainz, GermanyORCID Iconhttps://orcid.org/0000-0002-4224-4703View further author information
ORCID Icon,
Jose Willemsei Dutch Liver Patient Association (NLV), Hoogland, NetherlandsView further author information
,
Melanie Karakaidose PFIC Advocacy and Resource Network, KY, United StatesView further author information
&
Harpreet Brrangd Children’s Liver Disease Foundation, Birmingham, UKView further author information
 show all
Pages 247-253 | Received 28 Oct 2020, Accepted 01 Dec 2020, Published online: 07 Jan 2021

References

  • Davit-Spraul A, Gonzales E, Baussan C,et al. Progressive familial intrahepatic cholestasis. Orphanet J Rare Dis. 4(1):1 (2009).
  • Srivastava A. Progressive familial intrahepatic cholestasis. J Clin Exp Hepatol. Elsevier; Vol.4 p. 25–36 (2014).
  • Gunaydin M, Bozkurter Cıl AT. Progressive familial intrahepatic cholestasis: diagnosis, management, and treatment. Hepatic Med Evid Res. Volume 10:95–104 (2018).
  • Bull LN, Thompson RJ. Progressive Familial Intrahepatic Cholestasis. Clinics in liver disease. W B Saunders; Vol.22, p. 657–669 (2018).
  • Gomez-Ospina N, Potter CJ, Xiao R,et al. Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis. Nat Commun. 7(1):1–8 (2016).
  • Sambrotta M, Strautnieks S, Papouli E,et al. Mutations in TJP2 cause progressive cholestatic liver disease. Nat Genet. 46(4):326–328 (2014).
  • Baker A, Kerkar N, Todorova L,et al. Systematic review of progressive familial intrahepatic cholestasis. Clin Res Hepatol Gastroenterol. 43(1):20–36 (2019).
  • Jacquemin E, Hermans D, Myara A,et al. Ursodeoxycholic acid therapy in pediatric patients with progressive familial intrahepatic cholestasis. Hepatology. 25(3):519–523 (1997).
  • Jacquemin E. Progressive familial intrahepatic cholestasis. Clin Res Hepatol Gastroenterol 36(SUPPL.1):S26–35 (2012).
  • Yasuda I, Iwashita T, Ohnishi T,et al. ENDOSCOPIC NASOBILIARY DRAINAGE: CURRENT INDICATIONS AND EVALUATION OF THE PRODUCTS. Digestive Endoscopy 18(s1):S92–5 (2006).
  • Al-Dury S, Marschall HU. Ileal bile acid transporter inhibition for the treatment of chronic constipation, cholestatic pruritus, and NASH. Frontiers in Pharmacology. Frontiers Media S A. 9: 931 (2018).
  • Bustorff-Silva J, Neto LS, Olímpio H,et al. Partial internal biliary diversion through a cholecystojejunocolonic anastomosis-a novel surgical approach for patients with progressive familial intrahepatic cholestasis: a preliminary report. J Pediatr Surg. 42(8):1337–1340 (2007).
  • Hegade VS, Kendrick SFW, Dobbins RL,et al. Effect of ileal bile acid transporter inhibitor GSK2330672 on pruritus in primary biliary cholangitis: a double-blind, randomised, placebo-controlled, crossover, phase 2a study. Lancet. 389(10074):1114–1123 (2017).
  • Mayo MJ, Pockros P, Jones D,et al. Clarity: A phase 2, randomized, double-blind, placebo-controlled study of lopixibat chloride (Formerly Lum001), a novel apical sodium-dependent bile acid transporter inhibitor, in the treatment of primary biliary cirrhosis associated with itching. J Hepatol. 64(2):S 197 (2016).
  • Al-Dury S, Wahlström A, Wahlin S,et al. Pilot study with IBAT inhibitor A4250 for the treatment of cholestatic pruritus in primary biliary cholangitis. Sci Rep. 8(1):6658 (2018).
  • Lee WS, Chai PF, Looi LM. Progressive familial intrahepatic Cholestasis in Malaysian patients – A report of five cases. Med J Malaysia. 64(3): 216 (2009)
  • Brouwer WBF, Van Exel NJA, Van Gorp B,et al. The CarerQol instrument: A new instrument to measure care-related quality of life of informal caregivers for use in economic evaluations. Qual Life Res. 15(6):1005–1021 (2006).
  • Elman S, Hynan LS, Gabriel V,et al. The 5-D itch scale: A new measure of pruritus. Br J Dermatol. 162(3):587–593 (2010).
  • Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. Pharmacoeconomics. 4(5):353–365 (1993).
  • Jo C. Cost-of-illness studies: concepts, scopes, and methods. Clin Mol Hepatol. Korean Association for the Study of the Liver; 20:327–337 (2014).
  • Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC:: Albireo Pharma official website (September 2020). [cited 2020 Nov 11]. Available from: http://ir.albireopharma.com/newsreleases/news-release-details/albireo-phase-3-trial-meets-both-primary-endpoints-odevixibat

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