Department of Medicine, Center for Liver Disease, Inova Fairfax Hospital, Falls Church, VA, USA.
Betty and Guy Beatty Center for Integrated Research, Claude Moore Health Education and Research Building, Inova Health System, 3300 Gallows Road, Falls Church, VA, 22042, USA.
Dig Dis Sci. 2020 Feb;65(2):416-422. doi: 10.1007/s10620-019-05786-1. Epub 2019 Aug 27.
Primary biliary cholangitis (PBC) is progressive and can cause end-stage liver disease necessitating a liver transplant (LT). PBC patients may be disadvantaged on LT waitlist due to MELD-based priority listing or other factors.
The aim was to assess waitlist duration, waitlist mortality, and post-LT outcomes of PBC patients.
The Scientific Registry of Transplant Recipients data for 1994-2016 was utilized. Adult patients with PBC without hepatocellular carcinoma (HCC) were selected. Their clinico-demographic parameters and waitlist and post-transplant outcomes were compared to those of patients with hepatitis C (HCV) without HCC.
Out of 223,391 listings for LT in 1994-2016, 8133 (3.6%) was for PBC without HCC. Mean age was 55.5 years, 76.9% white, 86.2% female, mean MELD score 21, 6.6% retransplants. There were 52,017 patients with hepatitis C included for comparison. The mean waitlist mortality was 17.9% for PBC and 17.6% for HCV (p > 0.05). The average transplantation rate was 57.7% for PBC and 53.3% for HCV (p < 0.0001), while waitlist dropout (death or removal due to deterioration) rate was 25.0% for PBC and 25.4% for HCV (p > 0.05). There was no significant difference in median waiting duration till transplantation between PBC patients and HCV after 2002 (103 vs. 95 days, p > 0.05). Post-LT mortality and graft loss rates were significantly lower in PBC than in HCV patients (all p < 0.02).
Despite no evidence of impaired waitlist outcomes and favorable post-transplant survival in patients with PBC, there is still a high waitlist dropout rate suggesting the presence of an unmet need for effective treatment.
原发性胆汁性胆管炎（PBC）是一种进行性疾病，可导致终末期肝病，需要进行肝移植（LT）。由于 MELD 为基础的优先排序或其他因素，PBC 患者在 LT 候补名单上可能处于不利地位。
评估 PBC 患者的候补名单持续时间、候补名单死亡率和 LT 后结局。
利用 1994-2016 年 Scientific Registry of Transplant Recipients 数据。选择无肝细胞癌（HCC）的 PBC 成年患者。将他们的临床人口统计学参数以及候补名单和移植后结局与无 HCC 的丙型肝炎（HCV）患者进行比较。
在 1994-2016 年期间，223391 例 LT 登记中，8133 例（3.6%）为无 HCC 的 PBC。平均年龄为 55.5 岁，76.9%为白人，86.2%为女性，平均 MELD 评分为 21，6.6%为再次移植。同时纳入 52017 例丙型肝炎患者进行比较。PBC 的平均候补名单死亡率为 17.9%，HCV 为 17.6%（p>0.05）。PBC 的平均移植率为 57.7%，HCV 为 53.3%（p<0.0001），而候补名单退出率（死亡或因病情恶化而移除）为 25.0%，HCV 为 25.4%（p>0.05）。2002 年后，PBC 患者与 HCV 患者的中位等待移植时间无显著差异（103 天 vs. 95 天，p>0.05）。PBC 患者的 LT 后死亡率和移植物丢失率明显低于 HCV 患者（均 p<0.02）。
尽管 PBC 患者候补名单结果无受损证据且移植后生存率良好，但候补名单退出率仍很高，这表明有效治疗存在未满足的需求。
