World's smallest combined en bloc liver-pancreas transplantation

Area of Science:

• Pediatric Surgery
• Transplantation Immunology
• Genetics and Rare Diseases

Background:

• Wolf-Hirschhorn Syndrome (WRS) is a rare genetic disorder characterized by infantile insulin-dependent diabetes mellitus, neutropenia, recurrent infections, liver failure susceptibility, bone dysplasia, and developmental delay.
• WRS often leads to fatal outcomes due to fulminant liver and kidney failure, with limited reported cases, predominantly in offspring of consanguineous parents.
• Current literature lacks documented instances of combined en bloc liver and pancreas transplantation in pediatric patients with WRS.

Observation:

• A 2-year-old child diagnosed with WRS presented with severe complications necessitating urgent organ transplantation.
• The child's clinical presentation included features consistent with WRS, highlighting the syndrome's multi-systemic impact.
• The complexity of WRS posed significant challenges for surgical intervention and post-operative management.

Findings:

• A successful combined en bloc liver and pancreas transplant was performed in a pediatric patient with WRS.
• This represents a novel therapeutic approach for managing life-threatening complications associated with WRS in young children.
• The procedure addressed both the metabolic (diabetes) and organ failure (liver) aspects of the syndrome.

Implications:

• Combined en bloc liver and pancreas transplantation may be a viable, albeit complex, treatment option for select pediatric patients with WRS.
• This case expands the surgical armamentarium for rare genetic disorders leading to multi-organ failure.
• Further research and case reporting are crucial to establish the long-term efficacy and safety of this combined transplant strategy in pediatric WRS.