Jagadeesh Menon1, Naresh Shanmugam2, Joseph J Valamparampil1, Abdul Hakeem3, Mukul Vij4, Anil Jalan5, Mettu Srinivas Reddy3, Mohamed Rela3,6. 1. Department of Pediatric Gastroenterology & Hepatology, Dr Rela Institute & Medical Centre, Bharat Institute of Higher Education and Research, Chennai, Tamil Nadu, 600044, India. 2. Department of Pediatric Gastroenterology & Hepatology, Dr Rela Institute & Medical Centre, Bharat Institute of Higher Education and Research, Chennai, Tamil Nadu, 600044, India. drnareshps@gmail.com. 3. Department of Hepatobiliary surgery & liver transplantation, Dr Rela Institute & Medical Centre, Bharat Institute of Higher Education and Research, Chennai, Tamil Nadu, India. 4. Department of Histopathology, Dr Rela Institute & Medical Centre, Bharat Institute of Higher Education and Research, Chennai, Tamil Nadu, India. 5. Department of Pediatric Genetics, NIRMAN, Mumbai, Maharashtra, India. 6. Liver Transplant Unit, Kings College Hospital, London, UK.
Abstract
OBJECTIVES: To report the experience of liver transplantation (LT) for tyrosinemia type 1 (TT-1). METHODS: Clinical data of children with TT-1 who underwent living donor LT between July 2009 and May 2020 were retrospectively analyzed. Data included pre-LT nitisinone therapy, graft type, post-LT complications, HCC incidence, and graft/patient survival. RESULTS: Nine children were diagnosed with TT-1 at a median age of 12 mo (6-54 mo). Nitisinone was started in 6 patients at a median age of 15 mo (6-42 mo), but all had frequent interruption of therapy due to logistics with drug procurement including its cost. Median age at transplantation was 5 y (2-11 y). Explant liver showed HCC in 5 patients (55% of total cohort). The graft and patient survival are 100% with median follow-up of 58 mo (24-84 mo). CONCLUSION: LT is curative for TT-1 and excellent results can be obtained in experienced centers. This is especially favorable in countries with limited resources where the cost of medical therapy is highly prohibitive, with lifelong diet restrictions and unclear long-term risk of HCC.
OBJECTIVES: To report the experience of liver transplantation (LT) for tyrosinemia type 1 (TT-1). METHODS: Clinical data of children with TT-1 who underwent living donor LT between July 2009 and May 2020 were retrospectively analyzed. Data included pre-LT nitisinone therapy, graft type, post-LT complications, HCC incidence, and graft/patient survival. RESULTS: Nine children were diagnosed with TT-1 at a median age of 12 mo (6-54 mo). Nitisinone was started in 6 patients at a median age of 15 mo (6-42 mo), but all had frequent interruption of therapy due to logistics with drug procurement including its cost. Median age at transplantation was 5 y (2-11 y). Explant liver showed HCC in 5 patients (55% of total cohort). The graft and patient survival are 100% with median follow-up of 58 mo (24-84 mo). CONCLUSION: LT is curative for TT-1 and excellent results can be obtained in experienced centers. This is especially favorable in countries with limited resources where the cost of medical therapy is highly prohibitive, with lifelong diet restrictions and unclear long-term risk of HCC.
Authors: C O Esquivel; L Mieles; I R Marino; S Todo; L Makowka; G Ambrosino; P Nakazato; T E Starzl Journal: Transplant Proc Date: 1989-02 Impact factor: 1.066