Clinical Outcomes of Integrated Treatment strategies in Progressive Familial Intrahepatic Cholestasis: Single Center Experience

Hafiz Bilal Ahmed; Syed Hasnain Abbas; Hafiz Aamir Bashir; Muhammad Usman Ali Rizvi; Syed Waqas Hassan; Muhammad  Umar

doi:10.51253/pafmj.v76iSUPPL-1.13280

Authors

Hafiz Bilal Ahmed Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan
Syed Hasnain Abbas Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan
Hafiz Aamir Bashir Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan
Muhammad Usman Ali Rizvi Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan
Syed Waqas Hassan Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan
Muhammad Umar Department of Liver Transplant & HPB Surgery, Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences, Gambat Pakistan

DOI:

https://doi.org/10.51253/pafmj.v76iSUPPL-1.13280

Keywords:

Biliary tract surgical procedures, Cholestasis, Liver transplantation, Progressive familial intrahepatic cholestasis, Pruritus.

Abstract

B

Objective: To evaluate the effectiveness of integrated treatment modalities based on outcomes and survival.

Study Design: Progressive Longitudinal.

Place and Duration of Study: Pir Abdul Qadir Shah Jeelani Institute of Medical Sciences (PAQSJIMS), Gambat, Pakistan, from Jan 2019 to Dec 2023.

Methodology: This study enrolled 43 patients with Progressive Familial Intrahepatic Cholestasis (PFIC), who were categorized into three treatment groups: medical management (n=22), Partial Internal Biliary Diversion (PIBD) (n=9), and Living Donor Liver Transplantation (LDLT) (n=12). Outcomes were assessed using pruritis scores, survival rates, and morbidity using Calvin-Dindo classification.

Results: In the medical management group, mean follow-up was 39.14±20.49 months, with a 90.90% survival rate and significant pruritis improvement (2.09±1.01, p=0.006) while the PIBD group had a mean follow-up of 49.89±21.07 months, a 100% survival rate, and significant pruritis reduction (0.78±0.83, p=0.021) but the LDLT group had a mean follow-up of 24.42±12.42 months, a 91.66% survival rate, and major morbidity (III-b). Overall survival across groups was 68.61±2.44 months.

Conclusion: Medical management and PIBD could be considered as initial treatments for well-compensated PFIC patients, while liver transplant is recommended only for cases with treatment failure.

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References

1. Alsohaibani FI, Peedikayil MC, Alfadley AF, Aboueissa MK, Abaalkhail FA, Alqahtani SA. Progressive familial intrahepatic cholestasis: A descriptive study in a tertiary care center. Int J Hepatol 2023; 2023: 1960152.

https://doi.org/10.1155/2023/1960152

2. Yu L, Liu Y, Wang S, Zhang Q, Zhao J, Zhang H, et al. Cholestasis: Exploring the triangular relationship of gut microbiota-bile acid-cholestasis and the potential probiotic strategies. Gut Microbes 2023; 15(1): 2181930.

https://doi.org/10.1080/19490976.2023.2181930

3. Sahloul A, Lainka E, Kathemann S, Swoboda S, Dröge C, Keitel V et al. Progressive familial intrahepatic cholestasis—outcome and time to transplant after biliary diversion according to genetic subtypes. Front Surg 2023; 10: 1074229.

https://doi.org/10.3389/fsurg.2023.1074229

4. Lemoine C, Bhardwaj T, Bass LM, Superina RA. Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis. J Pediatr Surg 2017; 52(2): 268-272.

https://doi.org/10.1016/j.jpedsurg.2016.11.021

5. Alam S, Lal BB. Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies. World J Hepatol 2022; 14(1): 98-114.

https://doi.org/10.4254/wjh.v14.i1.98

6. Davit-Spraul A, Fabre M, Branchereau S, Baussan C, Gonzales E, Stieger B, et al. ATP8B1 and ABCB11 analysis in 62 children with normal gamma-glutamyl transferase progressive familial intrahepatic cholestasis (PFIC): phenotypic differences between PFIC1 and PFIC2 and natural history. Hepatology 2010; 51(5): 1645-1655. https://doi.org/10.1002/hep.23539

7. Amirneni S, Haep N, Gad MA, Soto-Gutierrez A, Squires JE, Florentino RM et al. Molecular overview of progressive familial intrahepatic cholestasis. World J Gastroenterol 2020; 26(47): 7470-7484. https://doi.org/10.3748/wjg.v26.i47.7470

8. Baker A, Kerkar N, Todorova L, Kamath BM, Houwen RH. Systematic review of progressive familial intrahepatic cholestasis. Clin Res Hepatol Gastroenterol 2019; 43(1): 20-36.

https://doi.org/10.1016/j.clinre.2018.07.010

9. Henkel SA, Squires JH, Ayers M, Ganoza A, McKiernan P, Squires JE et al. Expanding etiology of progressive familial intrahepatic cholestasis. World J Hepatol 2019; 11(5): 450-463.

https://doi.org/10.4254/wjh.v11.i5.450

10. Van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL. Impact of genotype, serum bile acids, and surgical biliary diversion on native liver survival in FIC1 deficiency. Hepatology 2021; 74(2): 892-906.

https://doi.org/10.1002/hep.31787

11. Prince A, Traughber CA, Shiravand Y, Bhandari N, Khan MR, Sharma S, et al. Enigmatic functions of ATP8B1: cholestasis, inflammation, phosphoinositide flipping, and cellular homeostasis. Cell Cycle 2025: 1-13.

https://doi.org/10.1080/15384101.2025.2574275

12. Varol Fİ, Selimoğlu MA, Güngör Ş, Yılmaz S, Tekedereli İ. Single-center experience in management of progressive familial intrahepatic cholestasis. Arab J Gastroenterol 2021; 22(4): 310-315. https://doi.org/10.1016/j.ajg.2021.05.021

13. Agarwal S, Lal BB, Rawat D, Rastogi A, Bharathy KG, Alam S et al. Progressive familial intrahepatic cholestasis (PFIC) in Indian children: clinical spectrum and outcome. J Clin Exp Hepatol 2016; 6(3): 203-208.

https://doi.org/10.1016/j.jceh.2016.05.003

14. Gunaydin M, Bozkurter Cil AT. Progressive familial intrahepatic cholestasis: diagnosis, management, and treatment. Hepat Med 2018; 10: 95-104.

https://doi.org/10.2147/HMER.S137209

15. Foroutan HR, Bahador A, Ghanim SM, Dehghani SM, Anbardar MH, Fattahi MR, et al. Effects of partial internal biliary diversion on long-term outcomes in patients with progressive familial intrahepatic cholestasis: experience in 44 patients. Pediatr Surg Int 2020; 36(5): 603-610.

https://doi.org/10.1007/s00383-020-04641-z

16. Namgoong JM, Hwang S, Kwon H, Ha S, Kim KM, Oh SH, et al. Liver transplantation in pediatric patients with progressive familial intrahepatic cholestasis: Single center experience of seven cases. Ann Hepatobiliary Pancreat Surg 2022; 26(1): 69-75. https://doi.org/10.14701/ahbps.21-114

17. Hang C, Jin Y, Luo Y, Feng M, Zhou T, Zhu J, et al. Long-term results of pediatric liver transplantation for progressive familial intrahepatic cholestasis. J Clin Med 2022; 11(16): 4684.

https://doi.org/10.3390/jcm11164684

18. Davit-Spraul A, Gonzales E, Baussan C, Jacquemin E. Progressive familial intrahepatic cholestasis. Orphanet J Rare Dis 2009; 4: 1.

https://doi.org/10.1186/1750-1172-4-1