Surgical gene therapy by domino auxiliary liver transplantation

D’Antiga, Lorenzo; Colledan, M.

doi:10.1002/lt.24326

Cited by 10 publications

(5 citation statements)

References 10 publications

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“…Matsunami et al and Badell et al reported successful results and in our experience one PA, two CN‐1 and one carbamoyl phosphate synthatase deficiency patient received MSUD livers with metabolically normal post‐transplantation follow‐up period. Rare cases using APOLT (auxiliary partial orthotopic liver transplantation) technique have been reported as well . Govil et al have discussed the idea of swap domino auxiliary liver transplantation for liver‐based metabolic disorder pairs to perform donor‐less transplantation .…”

Section: Discussionmentioning

confidence: 99%

Technique and outcome of domino liver transplantation from patients with maple syrup urine disease: Expanding the donor pool for live donor liver transplantation

Çelik

Kelly

Soltys

et al. 2019

Clinical Transplantation

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Aim/Background: Domino liver transplantation (DLT) using liver allografts from patients with metabolic disorders enhances organ utilization. Short-and long-term course and outcome of these patients can impact the decision to offer this procedure to patients, especially those with diseases that can potentially be cured with liver transplant. We reviewed the outcomes of DLT from maple syrup urine disease (MSUD) patients in our large academic pediatric and adult transplant program. Methods: All patients receiving DLT were analyzed retrospectively with a minimum of one-year follow-up period for patient and donor characteristics, early and late postoperative complications and patient and graft survival with their MSUD donors in terms of age, weight, MELD/PELD scores, cold ischemia time, postoperative leucine levels, and peak ALT (alanine aminotransferase) levels during the first 48 postoperative hours.

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Section: Discussionmentioning

confidence: 99%

Technique and outcome of domino liver transplantation from patients with maple syrup urine disease: Expanding the donor pool for live donor liver transplantation

Çelik

Kelly

Soltys

et al. 2019

Clinical Transplantation

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“…Matsunami et al reported successful short‐term results from transplanting MSUD liver to a patient with pyruvate carboxylase deficiency, and this study confirms normal metabolic balance >1‐year posttransplant. Additional case reports using domino auxiliary partial orthotopic liver transplantation technique have been described . In the current series, CN‐1 patients achieved dramatic correction of their hyperbilirubinemia with uneventful posttransplant course.…”

Section: Discussionmentioning

confidence: 57%

“…Additional case reports using domino auxiliary partial orthotopic liver transplantation technique have been described. 24,25 In the current series, CN-1 patients achieved dramatic correction of their hyperbilirubinemia with uneventful posttransplant course. Significant metabolic improvement and clinical correction was also seen in our PA and CPSD-1 patients.…”

Section: Discussionmentioning

confidence: 59%

Domino liver transplantation for select metabolic disorders: Expanding the living donor pool

et al. 2019

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Domino liver transplantation (DLT) involves transplanting liver from a patient with metabolic disease into a patient with end‐stage liver disease with the expectation that the recipient will not develop the metabolic syndrome or the recurrent syndrome will have minimal affect. The domino donor gets a deceased donor or a segment of live‐donor liver through the deceased donor organ allocation system. Waitlist mortality for the domino recipient exceeds morbidity associated with getting the donor disease. Between 2015 and 2017, four patients with three metabolic disorders at UPMC Children's Hospital of Pittsburgh underwent DLT with domino allografts from maple syrup urine disease (MSUD) patients. These included patients with propionic acidemia (PA) (n = 1), Crigler‐Najjar (CN) syndrome type‐1 (n = 2), and carbamoyl phosphate synthetase deficiency (CPSD) (n = 1). Mean follow‐up was 1.6 years (range 1.1‐2.1 years). Total bilirubin levels normalized postoperatively in both CN patients and they maintain normal allograft function. The PA patient had normal to minimal elevations of isoleucine and leucine, and no other abnormalities on low protein diet supplemented with a low methionine and valine free formula. No metabolic crises have occurred. The patient with CPSD takes normal baby food. No elevation in ammonia levels have been observed in any of the patients. DLT for a select group of metabolic diseases alleviated the recipients of their metabolic defect with minimal evidence of transferrable‐branched chain amino acid elevations or clinical MSUD despite increased protein intake. DLT using allografts with MSUD expands the live donor liver pool and should be considered for select metabolic diseases that may have a different enzymatic deficiency.

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“…The explanted liver of a child affected by propionic acidemia (receiving living related donor organ) has been used as an auxiliary graft in a child with Crigler‐Najjar, with effective clearance of hyperbilirubinemia and no manifestations of PA seen in the recipient 116 . This may only be feasible in larger, well‐resourced centers with a larger number of patients with non‐cirrhotic metabolic liver disease awaiting liver transplant 117 . Long‐term safety of an implanted graft with a metabolic defect needs to be considered.…”

Section: Future Directionsmentioning

confidence: 99%

“…116 This may only be feasible in larger, well-resourced centers with a larger number of patients with non-cirrhotic metabolic liver disease awaiting liver transplant. 117 Long-term safety of an implanted graft with a metabolic defect needs to be considered.…”

Section: Domino Auxiliary Partial Orthotopic Liver Transplantationmentioning

confidence: 99%

Current status and future directions of liver transplantation for metabolic liver disease in children

Eldredge,

Hardikar

2023

Pediatric Transplantation

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Orthotopic liver transplantation (OLT) in the care of children with inborn errors of metabolism (IEM) is well established and represent the second most common indication for pediatric liver transplantation in most centers worldwide, behind biliary atresia. OLT offers cure of disease when a metabolic defect is confined to the liver, but may still be transformative on a patient's quality of life reducing the chance of metabolic crises causing neurological damage in children be with extrahepatic involvement and no “functional cure.” Outcomes post‐OLT for inborn errors of metabolism are generally excellent. However, this benefit must be balanced with consideration of a composite risk of morbidity, and commitment to a lifetime of post‐transplant chronic disease management. An increasing number of transplant referrals for children with IEM has contributed to strain on graft access in many parts of the world. Pragmatic evaluation of IEM referrals is essential, particularly pertinent in cases where progression of extra‐hepatic disease is anticipated, with long‐term outcome expected to be poor. Decision to proceed with liver transplantation is highly individualized based on the child's dynamic risk‐benefit profile, their family unit, and their treating multidisciplinary team. Also to be considered is the chance of future treatments, such as gene therapies, emerging in the medium term.

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Surgical gene therapy by domino auxiliary liver transplantation

Cited by 10 publications

References 10 publications

Technique and outcome of domino liver transplantation from patients with maple syrup urine disease: Expanding the donor pool for live donor liver transplantation

Technique and outcome of domino liver transplantation from patients with maple syrup urine disease: Expanding the donor pool for live donor liver transplantation

Domino liver transplantation for select metabolic disorders: Expanding the living donor pool

Current status and future directions of liver transplantation for metabolic liver disease in children

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