Living‐donor liver transplantation for children with tyrosinemia type I

Abstract

Objective To evaluate the efficacy of living‐donor liver transplantation (LDLT) in children with tyrosinemia type I. Methods Altogether 10 patients diagnosed with tyrosinemia type I underwent LDLT between June 2013 and April 2019. Cirrhosis was the indication for LDLT in all 10 patients, and hepatocellular carcinoma (HCC) was suspected in nine. Patients' outcomes, including liver function, restoration of metabolism, quality of life and physical development, were analyzed after LDLT. Results All recipients were alive with a normal liver function after a median follow‐up period of 49 months. Pathological examinations detected HCC in one patient, dysplasia in five and cirrhosis in all. Nine patients were found to have elevated alpha‐fetoprotein level, and their median alpha‐fetoprotein level dropped from 2520 ng/mL to a normal level after LDLT, with no recurrence of HCC detected during the follow‐up. Tyrosine metabolism was restored to its normal level with normalized plasma tyrosine and succinylacetone concentrations. Moreover, urinary succinylacetone excretion decreased significantly during the follow up. LDLT improved patients’ renal tubular function, as evidenced by the normalized plasma phosphate concentration and improved glomerular filtration rate. Severe rickets symptoms, including spontaneous fractures and bone pain, were also ameliorated. Improved motor function was reported by all patients' parents during the follow‐up. Dietary restriction was no longer required, which was associated with a favorable catch‐up in growth and improved quality of life. Complete resolution of hypertrophic cardiomyopathy was observed one year after LDLT in one patient. Conclusion LDLT is an effective treatment for patients with end‐stage liver disease resulting from tyrosinemia type I.

Liver Transplantation for Pediatric Hepatocellular Carcinoma: A Systematic Review

“…After screening titles and abstracts, 563 articles were retrieved for full-text evaluation. Ultimately, 67 non-overlapping studies [ 11 , 12 , 22 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 55 , 56 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 , 65 , 66 , 67 , 68 , 69 , 70 , 71 , 72 , 73 ,…”

Hereditary Tyrosinemia Type 1 in Jordan: A Retrospective Study

“…Nitisinone introduction improve survival and reduce the admission rates for the patients [ 17 ]. Liver transplantation is the only definitive therapy for the disease [ 18 – 20 ].…”

Pediatric Liver Transplantation For Hereditary Metabolic Disorders With Structural Liver Damage

“…Currently, renal impairment and indication of combined liver-kidney transplantation are not that much significant compared to the era before introduction of NTBC. Patients show normal liver function and normal growth on unrestricted diet with no metabolic crises after LT with patient survival rate higher than 85% (24)(25)(26)(27). In our experience, one chronic form TT-1 patient was transplanted because of dysplastic nodules and high AFP levels from a parental donor.…”

“…Tyrosinemia type-1 is an autosomal recessive metabolic disorder present in 2 forms as acute form in neonatal period or chronic form with development of HCC risk beside toxic effects of tyrosine and phenylalanine metabolites (1,(3)(4)(5)(24)(25)(26)(27). Hepatocellular carcinoma risk is very high in chronic form.…”