Add-on immunosuppressive therapy may benefit selected patients with primary biliary cholangitis and autoimmune phenomena

Mengqi Li; Sha Chen; Shuxiang Li; Tingting Lv; Buer Li; Shan Shan; Min Li; Na Zeng; Qianyi Wang; Yuanyuan Kong; Hong Ma; Xinyan Zhao; Xiaojuan Ou; Hong You; Weijia Duan; Jidong Jia

doi:10.1177/17562848231224840

Add-on immunosuppressive therapy may benefit selected patients with primary biliary cholangitis and autoimmune phenomena

Therap Adv Gastroenterol. 2024 Jan 18:17:17562848231224840. doi: 10.1177/17562848231224840. eCollection 2024.

Authors

Mengqi Li^{1

2

3}, Sha Chen^{1

2

3}, Shuxiang Li^{1

2

3}, Tingting Lv^{1

2

3}, Buer Li^{1

2

3}, Shan Shan^{1

2

3}, Min Li^{4

3}, Na Zeng^{4

3}, Qianyi Wang^{1

2

3}, Yuanyuan Kong^{4

3}, Hong Ma^{1

2

3}, Xinyan Zhao^{1

2

3}, Xiaojuan Ou^{1

2

3}, Hong You^{1

2

3}, Weijia Duan^{5

2

3}, Jidong Jia^{5

2

3}

Affiliations

¹ Liver Research Center, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
² Beijing Key Laboratory of Translational Medicine on Liver Cirrhosis, Beijing, China.
³ National Clinical Research Center for Digestive Diseases, Beijing, China.
⁴ Clinical Epidemiology and EBM Unit, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
⁵ Liver Research Center, Beijing Friendship Hospital, Capital Medical University, 95 Yong-an Road, Beijing 100050, China.

Abstract

Background: Mildly elevated levels of transaminase and/or immunoglobulin G (IgG) are common in patients with primary biliary cholangitis (PBC). It is still unclear whether adding immunosuppressive therapy to ursodeoxycholic acid (UDCA) benefits those patients who are not fulfilling the diagnostic criteria of PBC with autoimmune hepatitis (AIH) features.

Objectives: To assess the efficacy of adding immunosuppressive therapy to UDCA for patients with PBC and autoimmune phenomena but not fulfilling the diagnostic criteria of PBC with AIH features.

Design: This is a retrospective-prospective cohort study in a tertiary medical center.

Methods: Patients with PBC and autoimmune phenomena were defined by the elevation of IgG and/or transaminase but did not fulfill the diagnostic criteria of PBC with AIH features. We grouped these patients based on with and without add-on immunosuppressive therapy and balanced their baseline characteristics using inverse probability treatment weighting (IPTW).

Results: A total of 652 patients with PBC and autoimmune phenomena were included, with a median follow-up of 4.08 years. After IPTW, the pseudo sample size in the add-on therapy and monotherapy groups was 558 and 655, respectively. After 1 year of observation, patients in the add-on therapy group had a higher biochemical response rate (normalization of transaminase and IgG levels) (49% versus 17%, p < 0.001). Furthermore, add-on therapy improved the transplant-free survival in the subgroup of patients with PBC and transaminase ⩾3 × upper limit of normal (ULN) or IgG ⩾1.3 × ULN (p = 0.033).

Conclusion: Add-on immunosuppressive therapy may improve the normalization rates of transaminase and IgG levels in all patients with PBC and mildly elevated transaminase and IgG levels and the long-term outcomes in the subgroup of the patients with transaminase ⩾3 × ULN or IgG ⩾1.3 × ULN.

Keywords: immunosuppressant; long-term outcome; primary biliary cirrhosis; second-line therapy.

Plain language summary

A look at add-on immunosuppressive therapy in primary biliary cholangitis patients Adding immunosuppressive therapy may enhance the normalization of ALT, AST and IgG levels in all PBC patients with mild elevation and improve long-term outcomes in those with more severe elevation of ALT, AST and IgG. These findings contribute to our understanding of treatment options for PBC patients with autoimmune phenomena.