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Solid organ transplantation in primary mitochondrial disease: Proceed with caution
Sumit Parikh1, Amel Karaa2, Amy Goldstein3
1Neurogenetics & Mitochondrial Disease, Center for Pediatric Neurology, Cleveland Clinic, Cleveland, OH, United States.
Solid organ transplants in patients with mitochondrial disease generally have good outcomes, except for those with POLG-related conditions. Careful consideration is advised for transplant eligibility, especially for POLG-related mitochondrial diseases.
Area of Science:
- Transplantation Medicine
- Genetics
- Rare Diseases
Background:
- Solid organ transplantation is infrequently performed in patients with primary mitochondrial disease.
- Existing data on outcomes are limited to case reports and small series.
- The impact of specific genetic mitochondrial diseases on transplant outcomes is not well understood.
Purpose of the Study:
- To evaluate post-transplant outcomes in adult and pediatric patients with primary mitochondrial disease.
- To determine if the underlying genetic cause of mitochondrial disease affects transplant morbidity and mortality.
- To assess the safety and tolerability of solid organ transplantation in this patient population.
Main Methods:
- Retrospective data collection from 35 patients across 17 transplant centers.
- Analysis of outcomes following liver, kidney, or heart transplantation.
- Comparison of outcomes between patients with and without POLG-related mitochondrial disease.
Main Results:
- Excluding POLG-related disease, post-transplant survival was comparable to non-mitochondrial disease patients.
- Most patients (majority) did not experience worsening of their mitochondrial disease within 90 days post-transplant.
- Post-transplant complications, including organ rejection, were infrequent and manageable.
Conclusions:
- Patients with mitochondrial disease generally tolerate solid organ transplantation well.
- Exclusion from transplantation solely based on mitochondrial disease diagnosis may not be warranted.
- POLG-related mitochondrial disease requires additional caution; transplant teams should consider mitochondrial disease in unexplained organ failure.
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