Treatment of acquired transthyretin amyloidosis in domino liver transplantation

Tsamis, Konstantinos I.; Mytilinaios, D.; Heneghan, Michael A.; Gillmore, Julian D.; Gilbertson, Janet A.; Giannopoulos, Sotirios; Sarmas, Ioannis; Konitsiotis, Spyridon

doi:10.1111/ctr.14822

Cited by 3 publications

(3 citation statements)

References 33 publications

(64 reference statements)

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“…18 In some reports, this novel therapeutic drug has been successfully used in de novo FAP among DLT recipients, with good results. 19,20 This trajectory could have interesting implications in the transplantation setting. DLT could be a potential source of more grafts when combining it with this novel drug, thus reducing waitlist time and enable transplantation of more patients.…”

Section: Discussionmentioning

confidence: 99%

“…The agent patisiran has been demonstrated to improve the clinical manifestation of FAP 18 . In some reports, this novel therapeutic drug has been successfully used in de novo FAP among DLT recipients, with good results 19,20 . This trajectory could have interesting implications in the transplantation setting.…”

Section: Discussionmentioning

confidence: 99%

“…2 These studies underline that de novo development of FAP may occur earlier than previously expected; however, it appears not to impact long-term DLT recipient survival rates. 2,7 Given the new therapeutic drugs used in DLT recipients with promising outcomes so far, 19,20 the development of de novo FAP might become better controlled without major risk for the domino recipients in the future. The literature is however lacking large reports regarding the de novo development of metabolic disease.…”

Section: Domino or Deceased Livers: Meta-analysismentioning

confidence: 99%

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Clinical outcomes after transplantation of domino grafts or standard deceased donor livers: a systematic review and meta‐analysis

Parente,

Milana,

Hajibandeh

et al. 2024

J of Gastro and Hepatol

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Background and AimDomino liver transplantation (DLT) utilizes otherwise discarded livers as donor grafts for another recipients. It is unclear whether DLT has less favorable outcomes compared to deceased donor liver transplantation (DDLT). We aimed to assess the outcomes of DLT compared to DDLT.MethodsMEDLINE, Embase, and Web of Science database were searched to identify studies comparing outcomes after DLT with DDLT. Data were pooled using random‐effects modeling, evaluating odds ratios (OR) or mean difference (MD) for outcomes including waiting list time, severe hemorrhage, intensive care unit (ICU), length hospital stay (LOS), rejection, renal, vascular, and biliary events, and recipient survival at 1, 3, 5, and 10 years.ResultsFive studies were identified including 945 patients (DLT = 409, DDLT = 536). The DLT recipients were older compared to the DDLT group (P = 0.04), and both cohorts were comparable regarding lab MELD, hepatocellular carcinoma, and waitlist time. There were no differences in vascular (OR: 1.60, P = 0.39), renal (OR: 0.62, P = 0.24), biliary (OR: 1.51, P = 0.21), severe hemorrhage (OR: 1.09, P = 0.86), rejection (OR: 0.78, P = 0.51), ICU stay (MD: 0.50, P = 0.21), or LOS (MD: 1.68, P = 0.46) between DLT and DDLT. DLT and DDLT were associated with comparable 1‐year (78.9% vs 80.4%; OR: 1.03, P = 0.89), 3‐year (56.2% vs 54.1%; OR: 1.35, P = 0.07), and 10‐year survival (6.5% vs 8.5%; OR: 0.8, P = 0.67) rates. DLT was associated with higher 5‐year survival (41.6% vs 36.4%; OR: 1.70; P = 0.003) compared to DDLT, which was not confirmed at sensitivity analysis.ConclusionThis meta‐analysis of the best available evidence (Level 2a) demonstrated that DLT and DDLT have comparable outcomes. As indications for liver transplantation expand, future high‐quality research is encouraged to increase the DLT numbers in clinical practice, serving the growing waiting list candidates, with the caveat of uncertain de novo disease transmission risks.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Domino or Deceased Livers: Meta-analysismentioning

confidence: 99%

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Clinical outcomes after transplantation of domino grafts or standard deceased donor livers: a systematic review and meta‐analysis

Parente,

Milana,

Hajibandeh

et al. 2024

J of Gastro and Hepatol

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Safety, Tolerability, and Outcomes of Tafamidis for the Treatment of Acquired Amyloid Neuropathy in Domino Liver Transplant Recipients

Nedkova-Hristova,

Donadeu,

Baliellas

et al. 2024

Neurol Ther

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Introduction Acquired amyloid neuropathy is an iatrogenic disease that appears years after a domino liver transplant. The objectives of our study are to analyze the efficacy and tolerability of tafamidis for the treatment of acquired amyloid neuropathy in domino liver transplant recipients. This post-authorization, prospective, longitudinal study included seven domino liver transplant recipients with acquired amyloid neuropathy who received treatment with tafamidis for 18 months. Methods The primary endpoints were the response rate, defined as those patients with an increase of < 2 points on the Neurological Impairment Score (NIS) from baseline, and the change in the NIS score from baseline. Secondary endpoints included the Quantitative Sensory Test, 10-m walk test, quality of life (Norfolk), and disability (Rasch-built Overall Disability Scale). As safety parameters, the evidence of graft rejection, changes in immunosuppressive trough levels and changes in antiviral and allogeneic cellular immunity before and 12 months after tafamidis treatment were also assessed. Results Six patients (85.7%) had responded at 18-months. Compared to baseline, we observed non-statistically significant improvement in mean NIS score at 6 months (− 2.54 points, CI − 5.92 to 0.84), 12 months (− 3.25 points; CI − 6.63 to 0.13), and 18 months (− 2.35 points; CI − 5.74 to 1.02). Changes in the Quantitative Sensory Test, 10-m walk tests and the quality of life and disability questionnaires were not statistically significant. The use of tafamidis did not induce relevant side effects or drug interactions. Also, no acute rejections events nor changes in functional adaptive immunity were observed. Conclusion Our study supports the safety and tolerability of tafamidis for the treatment of acquired amyloid neuropathy in domino liver transplant recipients. Tafamidis shows promise as a useful treatment in the clinical management of these patients. Future randomized placebo-controlled clinical trials with longer follow-up durations are needed. Supplementary Information The online version contains supplementary material available at 10.1007/s40120-024-00621-w.

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Treatment of acquired transthyretin amyloidosis in domino liver transplantation

Tsamis

Mytilinaios²,

Heneghan

et al. 2022

Clinical Transplantation

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Background: Domino liver transplantation (DLT) has been commonly used during the last two decades to partly meet the high need for liver transplants. However, the recipients of grafts from patients with noncirrhotic inherited metabolic disorders may ultimately develop metabolic syndrome, and management is usually intricate, being complicated by the underlying initial disorder, other comorbidities, and post-transplantation conditions. Case:We report here the management and the outcome in a patient with acquired transthyretin amyloidosis after DLT and significant comorbidities. Final treatment with a transthyretin gene silencing agent, patisiran, was well tolerated and resulted in remission of the aggravating neurological deficits in a follow-up period of 2 years. Conclusions:The case presented here supports the concept that patisiran can target the hepatocytes producing the mutated transthyretin in acquired transthyretin amyloidosis, as efficiently as in hereditary transthyretin amyloidosis (hATTR), and can be used to treat patients with transthyretin amyloidosis after DLT.

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Treatment of acquired transthyretin amyloidosis in domino liver transplantation

Cited by 3 publications

References 33 publications

Clinical outcomes after transplantation of domino grafts or standard deceased donor livers: a systematic review and meta‐analysis

Clinical outcomes after transplantation of domino grafts or standard deceased donor livers: a systematic review and meta‐analysis

Safety, Tolerability, and Outcomes of Tafamidis for the Treatment of Acquired Amyloid Neuropathy in Domino Liver Transplant Recipients

Treatment of acquired transthyretin amyloidosis in domino liver transplantation

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