Living-donor liver transplantation for children with tyrosinemia type I.
J Dig Dis
; 21(3): 189-194, 2020 Mar.
Article
em En
| MEDLINE
| ID: mdl-31953907
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Fígado
/
Doadores Vivos
/
Tirosinemias
/
Doença Hepática Terminal
Tipo de estudo:
Evaluation_studies
Aspecto:
Patient_preference
Limite:
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Idioma:
En
Revista:
J Dig Dis
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
China