Perioperative characteristics and management of liver transplantation for isolated methylmalonic acidemia-the largest experience in China
Perioperative characteristics and management of liver transplantation for isolated methylmalonic acidemia-the largest experience in China
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DOI：
10.21037/hbsn.2019.03.04
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发表时间：
2019-10-01
影响因子：
8
通讯作者：
Chen, Xiao-Jie
中科院分区：
文献类型：
--
作者：
Jiang, Yi-Zhou;Sun, Li-Ying;Chen, Xiao-Jie
背景资料：关于甲基丙二酸血症（MMA）患者肝移植（LT）围手术期临床特征的详细共识和指南很少。方法：选取2013年6月至2017年12月在北京友谊医院行肝移植的7例MMA患者，收集患者的临床资料（临床特征、实验室检查、尿MMA水平的时间变化、治疗等），并对患者的临床表现、治疗方案进行回顾性分析。回顾性分析围手术期发生的并发症。结果：7例患者均确诊为孤立性MMA，其中3例接受活体肝移植（LDLT），4例接受死亡供体肝移植（DDLT）。术后几天内和术后2周内代谢紊乱，平均血碱过剩值（BE-B）恢复正常，而血浆碳酸氢盐（HCO_3 ~-）虽经间歇性碳酸氢钠校正仍低于正常值。丙酰肉毒碱（C3）和C3/C2明显降低，尿MMA平均值降低81.7%（P <0.01），高于正常值上限。代谢纠正药物的给药方式与之前相同。1例1：1“（血肌酐升高）前肾功能不全的患者，观察期间通过调整免疫抑制方案，肾功能维持稳定。所有患者存活至今。结论：LT是一种有效的治疗，以防止代谢危机，但MMA患者往往是代谢脆弱，即使手术后。围手术期应密切监测酸中毒发作，实施碳酸氢钠纠正。仍然需要纠正代谢的药物。特殊的免疫抑制方案是维持肾功能的有效方法。
Background: There are few detailed consensus and guidelines on perioperative clinical characteristics of liver transplantation (LT) in patients with methylmalonic acidemia (MMA). This retrospective study investigated details of the clinical course and individualized treatment plan of the center with largest experience in China.Methods: A total of 7 MMA patients undergoing LT in Beijing Friendship Hospital from June 2013 to December 2017 were enrolled in the study, whose clinical data (clinical characteristics, laboratory findings, chronological changes in urine MMA levels, treatment, etc.) during perioperative period were analyzed retrospectively. All the patients received strict postoperative management.Results: All the 7 cases were confirmed to have isolated MMA, among which, 3 cases received living donor liver transplantation (LDLT), 4 cases received deceased donor liver transplantation (DDLT). A wild fluctuate of metabolic condition was observed within the first few days after surgery and two weeks after LT, the mean base excess of blood value (BE-B) restored to normal whereas plasma bicarbonate (HCO3-) was still below normal value even with intermittent sodium bicarbonate correction. it also showed marked reduction in propionylcarnitine (C3) and C3/C2 level and the mean urine MMA by gas chromatography-mass spectrometry (GC-MS) was reduced by 81.7% (P72x higher than upper limit of normal. The metabolism-correcting medications were administered as before. The renal function of one case with renal insufficiency before 1:1' (serum creatinine rising) maintained stable by adjusting the immunosuppressive regimen during the observation period. All patients survive to date.Conclusions: LT is an effective treatment to prevent metabolic crisis, but patients with MMA tend to be metabolically fragile even after surgery. During perioperative period, close monitoring should be given for acidosis episodes so as to implement sodium bicarbonate correction. Metabolism-correcting medications are still needed. Special immunosuppressive regimen is an effective way of maintaining renal function for those with kidney dysfunction.