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- Title
Outcomes of Pediatric Liver Transplantation in Inherited Metabolic Diseases: A Single-center's Experience.
- Authors
Ersoy, Melike; Ertekin, Vildan; Şık, Güntülü; Çıtak, Agop; Keçeoğlu, Selim; Emiroğlu, Remzi
- Abstract
Objective: To expand on the hitherto limited knowledge of the indications and outcomes of pediatric liver transplantation (LT) for inherited metabolic diseases (IMDs). Methods: Demographic data, pretransplant clinical and laboratory profiles, post-transplant outcomes and survival rates of twelve patients under 18 years who underwent LT for IMDs between January 2015 and June 2021 were analyzed. Results: Twelve (6 female) of 104 (11.5%) patients had a diagnosis of IMD. Four of the patients were diagnosed with primary hyperoxaluria type 1; two had Crigler-Najjar syndrome; and there was one patient each having maple syrup urine disease, propionic acidemia, tyrosinemia type 1, glycogen storage disease type 1a, Wilson's disease, and homozygous familial hypercholesterolemia, respectively. The mean current ages and ages at transplantation of the patients were 8.7 (1-14.2) and 6.5 (0.3-12.8) years, respectively. Their mean follow-up time was 2.7 (0.5-6.1) years. The distribution of LT indications was poor metabolic control (42%), the need for frequent hospitalization due to an acute life-threatening attack (17%), progressive neuromotor retardation (8%), and target organ failure (33%) respectively. The mean time between diagnosis and LT was 2.7 (0.5-6.1) years. No neurological, hematological, or metabolic complications were observed after LT. The biliary stricture developed in two (16.7%) patients, separation of arterial anastomosis in one (8.3%) and ascites infection in one (8.3%) patient. One-year patient and graft survival rates were both 100%. A significant difference was observed between the patients' pre-operative and current height and weight standard deviation scores, respectively (p=0.001 and p=0.006). Conclusion: LT is a good therapeutic option for improving the metabolic control and quality of life of patients with IMDs. Survival rates are excellent compared with other LT indications when appropriate timing and indication is adhered to.
- Subjects
CRIGLER-Najjar syndrome; HOMOZYGOUS familial hypercholesterolemia; SURGERY; PATIENTS; AMINO acid metabolism disorders; SURGICAL complications; TREATMENT effectiveness; MAPLE syrup urine disease; INBORN errors of carbohydrate metabolism; GLYCOGEN storage disease; HOSPITAL care; DESCRIPTIVE statistics; INBORN errors of metabolism; LIVER transplantation; HEPATOLENTICULAR degeneration; TRANSPLANTATION of organs, tissues, etc.; EVALUATION
- Publication
Medical Journal of Bakirkoy, 2022, Vol 18, Issue 1, p94
- ISSN
1305-9319
- Publication type
Article
- DOI
10.4274/BMJ.galenos.2022.2022.1-4