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Liver Transplant for Children With Hepatocellular Carcinoma and Hereditary Tyrosinemia Type 1
Ali Bahador1, Seyed Mohsen Dehghani, Bita Geramizadeh
1Department of Surgery, Transplant Ward, Shiraz University of Medical Sciences, Shiraz, Iran.
Insights
Hepatocellular carcinoma (HCC) and dysplasia prevalence in children with hereditary tyrosinemia type 1 post-liver transplant is lower than previously reported. Patients over 2 years old may benefit from liver transplantation for these conditions.
Area of Science:
- Hepatology
- Pediatric Oncology
- Transplant Surgery
Background:
- Hereditary tyrosinemia type 1 (HT-1) is a rare genetic disorder.
- HT-1 significantly increases the risk of developing hepatocellular carcinoma (HCC) and premalignant liver lesions.
- Liver transplantation is a potential curative treatment for HT-1 and its complications.
Purpose of the Study:
- To determine the prevalence of hepatocellular carcinoma (HCC) and premalignant lesions in children with HT-1.
- To evaluate the outcomes of orthotopic liver transplantation in this patient cohort.
- To assess the impact of age and pretransplant treatment on HCC development.
Main Methods:
- Retrospective review of 36 pediatric patients with HT-1 who underwent liver transplantation.
- Analysis of clinical records, pre- and post-transplant pathology, imaging (ultrasound, CT), and alpha-fetoprotein levels.
- Biopsy of hepatic nodules larger than 10 mm prior to transplantation.
Main Results:
- Hepatocellular carcinoma (HCC) was diagnosed in 5 out of 36 patients (13.9%) upon explanted liver examination.
- All HCC cases occurred in patients older than 2 years of age.
- No HCC was found in patients younger than 2 years; 3 patients had small cell dysplasia.
Conclusions:
- The observed prevalence of HCC and dysplasia in HT-1 pediatric patients post-transplant is lower than previously reported.
- Age appears to be a significant factor, with patients over 2 years old at higher risk.
- Liver transplantation is a viable treatment option, and pretransplant nitisinone treatment was administered to all HCC patients.
Objectives:
This study sought to determine the prevalence of hepatocellular carcinoma and other premalignant lesions in children with hereditary tyrosinemia type 1 who had undergone an orthotopic liver transplant at the Shiraz Transplant Center, in Shiraz, Iran.
Materials And Methods:
Between September 2006, and June 2011, thirty-six patients with hereditary tyrosinemia type 1 received a liver transplant from a deceased (whole or split) or a living-related donor. Clinical records and pathologic specimens, before and after surgery, for each case were reviewed. In addition, ultrasound, abdominal computed tomographic imaging scan findings, and levels of alpha-fetoprotein were recorded.
Results:
Twenty-two patients with hepatic nodules larger than 10 mm underwent a Tru-Cut needle biopsy before their liver transplant. In 2 patients, a diagnosis of hepatocellular carcinoma was made by pathologic examination; in the other 20, cirrhosis was confirmed with no evidence of malignancy. After pathologic examination of the explanted livers, the largest nodules in the 36 patients were 35 mm. Five cases had at least 1 nodule of hepatocellular carcinoma. Three of the other patients had small cell dysplasia in some of nodules. All 5 cases with hepatocellular carcinoma were patients older than 2 years of age (19 patients were older than 2 years of age). All patients with hepatocellular carcinoma received pretransplant nitisinone treatment. All patients with hepatocellular carcinoma after their liver transplant are alive at the time of this writing.
Conclusions:
The prevalence of cell dysplasia and hepatocellular carcinoma in children with hereditary tyrosinemia type 1 in our study is not as high as that reported previously, so it appears that patients older than 2 years of age require a liver transplant.
