Liver transplant for inherited metabolic disease among siblings

Abstract

Liver transplantation (LT) is a very successful therapy for many forms of inherited metabolic disease (IMD). 1 Most of these disorders have autosomal recessive inheritance and hence more than one sibling may be affected. This situation is likely to result in unique stresses and challenges for affected families. Despite this, there is little published experience describing outcomes when multiple siblings undergo LT for the same genetic disorder. Indications for LT in IMD fall into two broad groups. Where the defect causes structural liver disease (SLD), LT is utilized to treat complications of end-stage liver disease. In those without SLD, LT is used to correct the metabolic phenotype, hence preventing systemic complications and improving quality of life. The indications for LT in those with SLD are usually clear and consistent, whereas indications to correct the metabolic phenotype are more subjective and still evolving. 1,2 These latter will likely reflect the experience of treating and referring teams, family expectations, and cultural factors. There is no published information as to how families make decisions on timing of LT or order of LT among siblings. It seems likely that decisions on LT for subsequent siblings will be impacted by the experience and outcome of earlier cases. We report our experience from a single pediatric transplant program of siblings who have undergone LT for IMD in order to document indications for LT, if there were differences in LT timing and

Outcomes After Living Donor Liver Transplantation in Pediatric Patients with Inherited Metabolic Diseases

“…In addition, genetic counseling will be required for the parents and recipients in the future. However, if siblings undergo LT for IMDs, it has been reported that later siblings should be listed and transplanted at a significantly younger age [ 31 ].…”