Short and Long-Term Outcomes of Liver Transplantation in Pediatric Patients With Inborn Errors of Metabolism: A Single-Center Study
Corresponding Author
Hazel Delal Dara Kar
Department of Pediatrics, Başkent University Faculty of Medicine, Ankara, Turkey
Correspondence:
Hazel Delal Dara Kar ([email protected])
Search for more papers by this authorHalil İbrahim Aydın
Department of Pediatric Metabolic Diseases, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFigen Özçay
Department of Pediatric Gastroenterology and Hepatology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorOya Balcı Sezer
Department of Pediatric Gastroenterology and Hepatology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorEsra Baskın
Department of Pediatric Nephrology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFeride Şahin
Department of Genetics, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorYunus Kasım Terzi
Department of Genetics, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorEmre Karakaya
Department of General Surgery, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMehmet Haberal
Department of General Surgery, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorCorresponding Author
Hazel Delal Dara Kar
Department of Pediatrics, Başkent University Faculty of Medicine, Ankara, Turkey
Correspondence:
Hazel Delal Dara Kar ([email protected])
Search for more papers by this authorHalil İbrahim Aydın
Department of Pediatric Metabolic Diseases, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFigen Özçay
Department of Pediatric Gastroenterology and Hepatology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorOya Balcı Sezer
Department of Pediatric Gastroenterology and Hepatology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorEsra Baskın
Department of Pediatric Nephrology, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFeride Şahin
Department of Genetics, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorYunus Kasım Terzi
Department of Genetics, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorEmre Karakaya
Department of General Surgery, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMehmet Haberal
Department of General Surgery, Başkent University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFunding: This work was supported by Baskent Üniversitesi.
ABSTRACT
Background
Inborn errors of metabolism (IEMs) are inherited diseases causing significant morbidity and mortality, particularly in childhood. Liver transplantation (LT) can be curative or partially effective for these diseases. LT for IEMs has increased, making IEMs the second most common reason for pediatric LT after biliary atresia.
Patients and Methods
Between 2001 and 2023, 50 pediatric patients with IEMs underwent LT at Başkent University, Ankara Hospital. Data collected retrospectively included diagnosis, gender, age of diagnosis, age of LT, LT indication, donor data, graft type, rejection episodes, post-transplant complications, and clinical findings of the IEMs before and after LT. Treatment methods, follow-up duration, and survival time were also recorded.
Results
Of the 332 pediatric LT patients, 50 (15.1%) had IEMs, with three requiring re-transplantations. Diagnoses included glycogen storage diseases (n = 11), tyrosinemia type 1 (n = 10), primary hyperoxaluria (n = 6), urea cycle disorders (n = 6), homozygous familial hypercholesterolemia (n = 4), propionic acidemia (n = 4), deoxyguanosine kinase deficiency (n = 3), maple syrup urine disease (n = 2), methylmalonic acidemia (n = 1), Niemann-Pick disease type B (n = 1), alkaptonuria with unknown neonatal cholestasis (n = 1), and bile acid synthesis disorder (n = 1). The parental consanguinity rate was 74%. Living-related donors provided organs for 48 (90.5%) patients. The mean age at LT was 75.3 ± 8.2 months (range: 5–218), with a follow-up period of 82.1 ± 10.2 months (range:1 day–229 months). Survival rates at 1, 5, 10, and 15 years were 83.7%, 81%, 81%, and 70.9%, respectively.
Conclusion
LT is an effective solution for children with IEM causing chronic organ failure and difficult to manage with medical treatment, showing a good long-term prognosis.
Conflicts of Interest
The authors declare no conflicts of interest.
Open Research
Data Availability Statement
The data that support the findings of this study are available from the corresponding author upon reasonable request. The data are not publicly available due to privacy or ethical restrictions.
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