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Abstract：
To explore the efficacy and safety of immunosuppressive therapy for the treatment of primary biliary cirrhosis-autoimmune hepatitis (PBC-AIH) overlap syndrome accompanied by decompensated cirrhosis.
A cohort study was performed to evaluate the usefulness of immunosuppressive therapy in this unique group. This cohort study was performed between October 2013 and June 2017 and included 28 biopsy-proven patients diagnosed according to the Paris criteria. The therapies included ursodeoxycholic acid (UDCA) alone (N=14) or in combination with immunosuppression (IS) therapy (N=14). The primary endpoints were biochemical remission, liver-related adverse events, transplant-free survival, and drug side-effects.
The frequency of biochemical remission for the AIH features was significantly higher in the UDCA+IS group than in the UDCA-only group (60.0 versus 9.1%, P=0.024) after 12 months of therapy but not after 3 and 6 months (28.6 versus 0%, P=0.165; 35.7 versus 7.1%, P=0.098). The rates of liver-related adverse events were lower in the combined group (2/14 versus 9/14, P=0.018). The Kaplan-Meier estimate showed that the transplant-free survival was distinct between the two groups (P=0.019). In the UDCA+IS group, mild and transient leukopenia occurred in two patients receiving azathioprine (AZA), and an infection was observed in one patient receiving mycophenolate mofetil (MMF).
PBC-AIH patients with decompensated cirrhosis receiving a combination of UDCA and immunosuppressors presented with higher biochemical remission rates and experienced fewer liver-related adverse events, implying that the combined treatment might be a better therapeutic option for strictly defined decompensated PBC-AIH overlap syndrome.
A cohort study was performed to evaluate the usefulness of immunosuppressive therapy in this unique group. This cohort study was performed between October 2013 and June 2017 and included 28 biopsy-proven patients diagnosed according to the Paris criteria. The therapies included ursodeoxycholic acid (UDCA) alone (N=14) or in combination with immunosuppression (IS) therapy (N=14). The primary endpoints were biochemical remission, liver-related adverse events, transplant-free survival, and drug side-effects.
The frequency of biochemical remission for the AIH features was significantly higher in the UDCA+IS group than in the UDCA-only group (60.0 versus 9.1%, P=0.024) after 12 months of therapy but not after 3 and 6 months (28.6 versus 0%, P=0.165; 35.7 versus 7.1%, P=0.098). The rates of liver-related adverse events were lower in the combined group (2/14 versus 9/14, P=0.018). The Kaplan-Meier estimate showed that the transplant-free survival was distinct between the two groups (P=0.019). In the UDCA+IS group, mild and transient leukopenia occurred in two patients receiving azathioprine (AZA), and an infection was observed in one patient receiving mycophenolate mofetil (MMF).
PBC-AIH patients with decompensated cirrhosis receiving a combination of UDCA and immunosuppressors presented with higher biochemical remission rates and experienced fewer liver-related adverse events, implying that the combined treatment might be a better therapeutic option for strictly defined decompensated PBC-AIH overlap syndrome.
摘要：
探讨免疫抑制治疗原发性胆汁性肝硬化-自身免疫性肝炎(PBC-AIH)重叠综合征合并失代偿期肝硬化的疗效及安全性。
进行了一项队列研究，以评估该独特组中免疫抑制治疗的有效性。该队列研究于2013年10月至2017年6月之间进行，包括根据巴黎标准诊断的28例经活检证实的患者。所述疗法包括单独的熊去氧胆酸(UDCA)(N=14)或与免疫抑制(IS)疗法(N=14)组合。主要终点是生化缓解，肝脏相关不良事件，无移植生存,和药物副作用。
UDCA+IS组AIH特征的生化缓解频率明显高于仅UDCA组（60.0对9.1％，P=0.024）治疗12个月后，但3个月和6个月后没有（28.6对0%，P=0.165；35.7%对7.1%，P=0.098)。联合组的肝脏相关不良事件发生率较低（2/14对9/14，P=0.018）。Kaplan-Meier估计显示两组之间的无移植存活率不同(P=0.019)。在UDCA+IS组中，两名接受硫唑嘌呤（AZA）的患者发生轻度和一过性白细胞减少症，在一名接受霉酚酸酯（MMF）的患者中观察到感染。
接受UDCA和免疫抑制剂联合治疗的失代偿期肝硬化PBC-AIH患者的生化缓解率较高，肝脏相关不良事件较少。这意味着联合治疗可能是严格定义的失代偿性PBC-AIH重叠综合征的更好治疗选择。
进行了一项队列研究，以评估该独特组中免疫抑制治疗的有效性。该队列研究于2013年10月至2017年6月之间进行，包括根据巴黎标准诊断的28例经活检证实的患者。所述疗法包括单独的熊去氧胆酸(UDCA)(N=14)或与免疫抑制(IS)疗法(N=14)组合。主要终点是生化缓解，肝脏相关不良事件，无移植生存,和药物副作用。
UDCA+IS组AIH特征的生化缓解频率明显高于仅UDCA组（60.0对9.1％，P=0.024）治疗12个月后，但3个月和6个月后没有（28.6对0%，P=0.165；35.7%对7.1%，P=0.098)。联合组的肝脏相关不良事件发生率较低（2/14对9/14，P=0.018）。Kaplan-Meier估计显示两组之间的无移植存活率不同(P=0.019)。在UDCA+IS组中，两名接受硫唑嘌呤（AZA）的患者发生轻度和一过性白细胞减少症，在一名接受霉酚酸酯（MMF）的患者中观察到感染。
接受UDCA和免疫抑制剂联合治疗的失代偿期肝硬化PBC-AIH患者的生化缓解率较高，肝脏相关不良事件较少。这意味着联合治疗可能是严格定义的失代偿性PBC-AIH重叠综合征的更好治疗选择。
