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Multivisceral transplantation for megacystis microcolon intestinal hypoperistalsis syndrome
M Masetti1, M M Rodriguez, J F Thompson
1Department of Surgery, University of Miami School of Medicine, Florida 33136, USA.
Background:
Megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS) is a rare autosomal recessive disorder causing a functional neonatal bowel obstruction. Its etiopathogenesis is not fully understood. The prognosis is poor in the majority of cases; most patients die before the age of 6 months. In this report, we describe our experience with three patients with MMIHS in whom multivisceral transplantation was performed.
Methods:
Three patients with MMIHS underwent multivisceral transplantation. All patients were females with a history of long-term total parenteral nutrition (TPN) with TPN-related cholestatic liver disease.
Results:
Patient 1 died 17 months after transplantation because of aspiration after revision of her feeding gastrostomy. At the time of death, the graft was functioning and the patient was completely off TPN. Patient 2 is alive 17 months after transplant. She is a fully functional, active 2-year-old and has also recently begun oral feeding after intensive rehabilitation. Patient 3 died on day 44 of multisystem failure.
Conclusions:
This is the first report in the literature of multivisceral transplantation for MMIHS. Although one of the three patients died 44 days after surgery from multiorgan system failure, the other two patients had long-term survival after transplant and both grew well on enteral feeding alone. One patient died 17 months from a non-transplant-related complication, while the other is living at home off of TPN, with almost complete dietary rehabilitation 17 months after transplant. Our case reports suggest that multivisceral transplantation is a valuable therapeutic option for patients affected by MMIHS with TPN-induced liver failure.
Insights
Multivisceral transplantation offers a potential life-saving option for infants with Megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS). This approach demonstrated long-term survival and improved feeding in two out of three MMIHS patients.
Area of Science:
- Gastroenterology
- Pediatric Surgery
- Transplantation Medicine
Background:
- Megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS) is a rare, severe neonatal bowel obstruction with poor prognosis.
- The exact causes of MMIHS remain unclear, necessitating exploration of advanced treatment modalities.
- MMIHS patients often require long-term total parenteral nutrition (TPN), leading to complications like liver disease.
Purpose of the Study:
- To evaluate the efficacy and outcomes of multivisceral transplantation in patients with MMIHS.
- To assess the potential for long-term survival and improved quality of life post-transplantation for MMIHS.
- To present the initial experience with this surgical intervention for a rare pediatric condition.
Main Methods:
- Three female patients diagnosed with MMIHS underwent multivisceral transplantation.
- All patients had a history of prolonged TPN and associated cholestatic liver disease.
- Outcomes were assessed based on survival, graft function, TPN dependence, and nutritional rehabilitation.
Main Results:
- One patient survived 17 months post-transplant, achieving independence from TPN and significant oral feeding, but died from an unrelated complication.
- A second patient remains alive at 17 months, is functionally active, and has begun oral feeding after intensive rehabilitation.
- The third patient experienced multisystem failure and died 44 days after the transplant procedure.
Conclusions:
- Multivisceral transplantation represents a novel therapeutic approach for MMIHS, offering a chance for long-term survival.
- Successful transplantation can lead to independence from TPN and substantial nutritional rehabilitation in select MMIHS patients.
- While challenging, the positive outcomes in two patients suggest MMIHS transplantation is a viable option for those with TPN-induced liver failure.