Liver transplantation using grafts with rare metabolic disorders

Schielke, Astrid; Conti, Filoména; Goumard, Claire; Perdigao, Fabiano; Calmus, Yvon; Scatton, Olivier

doi:10.1016/j.dld.2014.11.004

Cited by 24 publications

(30 citation statements)

References 83 publications

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“…This may reflect unnecessary anxiety about the use of heterozygous donors for phenotypic correction as opposed to the management of life‐threatening liver disease. Our data provide further support to the existing literature that LDLT, even from obligate heterozygous parents, is successful . Given the shortage of cadaveric donors and the increased use of transplantation for metabolic disease, natural justice suggests that this source should be maximized.…”

Section: Discussionsupporting

confidence: 80%

“…In MSUD, a heterozygous donor may not provide sufficient enzymatic activity to fully prevent metabolic crises . In heterozygotes for A1ATD, hepatic resection theoretically poses increased risks, although adverse consequences have not yet been reported . Another potential factor limiting LDLT may be where there is more than 1 affected sibling in the family.…”

Section: Discussionmentioning

confidence: 99%

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Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States

et al. 2019

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Indications for liver transplantation (LT) in metabolic disease are evolving. We reviewed the US experience with primary LT for metabolic disease in the Scientific Registry for Transplant Recipients (October 1987 to June 2017) to determine the following: temporal changes in indications, longterm outcomes, and factors predicting survival. Patients were grouped by the presence of structural liver disease (SLD) and whether the defect was confined to the liver. There were 5996 patients who underwent LT for metabolic disease, 2354 (39.3%) being children. LT for metabolic disease increased in children but not in adults. Children experienced a 6‐fold increase in LT for metabolic disease without SLD. Indications for LT remained stable in adults. Living donor liver transplantation increased between era 1 and era 3 from 5.6% to 7.6% in children and 0% to 4.5% in adults. Patient and graft survival improved with time. The latest 5‐year patient survival rates were 94.5% and 81.5% in children and adults, respectively. Outcomes were worse in adults and in those with extrahepatic disease (P < 0.01), whereas SLD did not affect outcomes. Survival improved with younger age at LT until age <2 years. On multivariate analysis, diagnostic category, inpatient status, age at LT, and transplant era significantly predicted outcomes in all ages with male sex predicting survival in childhood only. Children without structural disease were less likely to die awaiting LT and had improved post‐LT survival compared with children with chronic liver disease. In conclusion, LT for metabolic disease is increasingly used for phenotypic correction in children; extrahepatic manifestations significantly impact survival at all ages; where indicated, transplantation should not be unnecessarily delayed; and the development of new allocation models may be required.

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Section: Discussionsupporting

confidence: 80%

Section: Discussionmentioning

confidence: 99%

Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States

et al. 2019

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“…General health of potential donors is not only related to donor safety but also the risk of disease transmission. Besides cancer and infectious disease, rare metabolic and inherited diseases can also be transmitted from donor to recipient …”

Section: Discussionmentioning

confidence: 99%

“…Hypercoagulability risk and metabolic disease are the 2 most common medical reasons for donor exclusion. Hemostasis and thrombosis defect are known to increase the risk of intraoperative and postoperative complications, together with the possible transfer of this defect from graft to recipient . Coagulation screening including platelet count, prothrombin time/activated partial thromboplastin time, fibrinogen, and D‐dimer are already included in the standard laboratory screening for potential donors.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of donor workups and exclusions in a single‐center experience of living donor liver transplantation

et al. 2017

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The process of evaluating potential donors in liver transplantation is important to ensure donor safety and provide optimal recipient outcomes. However, there has been no report about donor exclusion rates and reasons for such exclusion in Korea. In this study, we aimed to elucidate the outcomes of potential living liver donor evaluation in a major living donor liver transplantation center. From July 2011 to June 2015, prospectively collected data of 726 potential donors for 588 matched recipients were subsequently evaluated. Among 726 potential donors, 374 potential donors (51.5%) finally reached donation; 352 potential donors (48.5%) were excluded for various reasons. Donor reasons were 29.8%, including medical problems, withdrawal of consent, graft volume issues, and identification of a better suitable donor. Recipient reasons were 20.7%, including recipient death or recovery, allocation to deceased donor, and progressions of hepatocellular carcinoma. A total of 38 (5.2%) potential donors had a fatty liver. Among them, 15 (39.5%) potential donors tried short-term weight reduction and eventually were able to donate. In conclusion, the main reasons for donor exclusion were medical problems and withdrawal of consent. Therefore, thorough medical screening and careful examination for donor voluntarism are important in the donor evaluation process. Liver Transplantation 23 614-624 2017 AASLD.

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“…These procedures are pursued with the prospect that DLT recipients will not develop clinical or subclinical manifestation of enzyme defect associated with the donor liver. However, there are several reports that suggest DLT allografts can transmit the primary disease in the post‐DLT period . Several DLT have been performed using livers from MSUD patients.…”

Section: Introductionmentioning

confidence: 99%

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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Liver transplantation using grafts with rare metabolic disorders

Cited by 24 publications

References 83 publications

Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States

Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States

Evaluation of donor workups and exclusions in a single‐center experience of living donor liver transplantation

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

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