Long-term clinical outcomes and health-related quality of life in patients with isolated methylmalonic acidemia after liver transplantation: experience from the largest cohort study in China.
Yi-Zhou JiangGuang-Peng ZhouLin WeiWei QuZhi-Gui ZengYing LiuYu-Le TanJun WangZhi-Jun ZhuLi-Ying SunPublished in: World journal of pediatrics : WJP (2024)
This large single-center study of the mainland of China showed an overall favorable impact of LT on isolated MMA in terms of long-term survival, metabolic control, and HRQoL in children and families. The potential for persistent neurocognitive impairment and inherent metabolic fragility requires long-term special care. Video Abstract (MP4 153780 KB).