Is Liver Transplant Still Used for V30M Amyloidosis?
At a Glance
Liver transplantation was historically the main treatment for ATTRv V30M amyloidosis to stop mutant protein production. Today, surgery has largely been replaced by highly effective, non-invasive medications called RNA silencers and TTR stabilizers that safely manage the disease.
In this answer
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If you are newly diagnosed with ATTRv V30M amyloidosis and have been reading about treatments online, you might feel overwhelmed or anxious. Much of the older literature prominently features liver transplantation as a primary treatment. However, the medical landscape has transformed dramatically over the last decade. Today, while you might still read about liver transplants, they have largely been replaced by highly effective, non-invasive medications.
The History: Why Was a Liver Transplant Used?
To understand why liver transplants were once the standard of care, it helps to understand how V30M amyloidosis works. The liver is the body’s main factory for producing the transthyretin (TTR) protein. In people with the V30M mutation, the liver produces a misfolded (mutant) version of this protein, which then builds up in the nerves, heart, and other organs as amyloid deposits [1][2].
Before the invention of modern drugs, the only way to stop the body from producing this mutant protein was to replace the liver entirely [3][1]. A liver transplant effectively gave the patient a new “factory” that produced normal, healthy TTR protein [2]. For many years, this was an established and vital treatment for early-stage hereditary ATTR amyloidosis, largely because it could slow the disease but could not reverse existing nerve damage [1][4].
The Modern Era: Stabilizers and Silencers
Today, the treatment paradigm has shifted entirely. Starting around 2018, the approval of modern disease-modifying therapies provided powerful, significantly less invasive alternatives to surgery [5][6]. Instead of removing the liver, doctors can now use targeted medications to manage the disease.
These modern therapies generally fall into two categories:
- RNA-Targeted Therapies (Silencers): Medications like patisiran, vutrisiran, inotersen, and eplontersen work by intercepting the genetic instructions in your liver, essentially telling it to stop producing the mutant TTR protein [7][8]. These are typically administered as regular intravenous (IV) infusions or under-the-skin injections. These therapies have been shown to significantly improve nerve function, quality of life, and cardiac symptoms [9][10].
- TTR Stabilizers: Medications like tafamidis bind to the TTR protein in the bloodstream, stabilizing it so that it cannot break apart and form harmful amyloid deposits [11][12]. Stabilizers are typically taken as a daily pill.
Because these medications can effectively slow or halt disease progression without the risks associated with major surgery and lifelong immunosuppression, liver transplantation is now rarely used as a first-line treatment for V30M amyloidosis [5][6]. However, it is important to know that these new medications are not entirely risk-free. They have their own potential side effects, such as vitamin A deficiency or reactions at the injection site, and require regular medical monitoring.
The Limitations of Transplants
Even when liver transplants were common, they were not a perfect cure. Medical research revealed that some patients continued to experience disease progression after their transplant [13][14]. This happened because the healthy TTR protein produced by the new liver could sometimes still get caught on existing amyloid deposits in the body, continuing to build up in the heart, nerves, or eyes over time [15][4]. Today, patients who previously received liver transplants are frequently prescribed modern silencers or stabilizers to manage this ongoing progression [13][16].
When Is Transplantation Still Considered?
While largely obsolete for the average V30M patient, transplantation may still be discussed in extremely rare, specific medical scenarios. For example, in advanced cases where amyloidosis has caused severe, irreversible organ damage, doctors might consider a combined heart and liver transplant [17]. Additionally, in regions where access to modern, high-cost medications is restricted, liver transplantation might still be an established option [1][18]. However, for the vast majority of patients today, treatment focuses on medical therapies rather than surgery.
Common questions in this guide
Why were liver transplants originally used to treat V30M amyloidosis?
How do modern medications replace the need for a liver transplant?
Are liver transplants ever still performed for V30M amyloidosis today?
Why do some patients still experience symptoms after a liver transplant?
Questions to Ask Your Doctor
Curated prompts to bring to your next appointment.
- 1.Based on my current symptoms, am I a better candidate for an RNA silencer or a TTR stabilizer?
- 2.What are the most common side effects of the modern medication you are recommending, and how will we manage them?
- 3.Given that I will be on a modern medication, will I still need regular heart and nerve monitoring?
- 4.Are there any out-of-pocket costs or assistance programs available for the medication you are recommending?
Questions For You
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References
References (18)
- 1
Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges.
Gertz MA
The American journal of managed care 2017; (23(7 Suppl)):S107-S112.
PMID: 28978215 - 2
New Medications in the Treatment of Hereditary Transthyretin Amyloidosis.
Walker S
Hospital pharmacy 2018; (53(4)):236-238 doi:10.1177/0018578718779757.
PMID: 30038442 - 3
The transthyretin amyloidoses: advances in therapy.
Dubrey S, Ackermann E, Gillmore J
Postgraduate medical journal 2015; (91(1078)):439-48 doi:10.1136/postgradmedj-2014-133224.
PMID: 26048914 - 4
Ocular Manifestations and Therapeutic Options in Patients with Familial Amyloid Polyneuropathy: A Systematic Review.
Martins AC, Rosa AM, Costa E, et al.
BioMed research international 2015; (2015()):282405 doi:10.1155/2015/282405.
PMID: 26558262 - 5
Transthyretin familial amyloid polyneuropathy: an update.
Plante-Bordeneuve V
Journal of neurology 2018; (265(4)):976-983 doi:10.1007/s00415-017-8708-4.
PMID: 29249054 - 6
Lipid-nanoparticle-enabled nucleic acid therapeutics for liver disorders.
Arjunan P, Kathirvelu D, Mahalingam G, et al.
Acta pharmaceutica Sinica. B 2024; (14(7)):2885-2900 doi:10.1016/j.apsb.2024.04.015.
PMID: 39027251 - 7
Assessing the effectiveness and safety of Patisiran and Vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review.
Karimi MA, Esmaeilpour Moallem F, Gholami Chahkand MS, et al.
Frontiers in neurology 2024; (15()):1465747 doi:10.3389/fneur.2024.1465747.
PMID: 39286810 - 8
Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease.
Adams D, Koike H, Slama M, Coelho T
Nature reviews. Neurology 2019; (15(7)):387-404 doi:10.1038/s41582-019-0210-4.
PMID: 31209302 - 9
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Adams D, Tournev IL, Taylor MS, et al.
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2023; (30(1)):1-9 doi:10.1080/13506129.2022.2091985.
PMID: 35875890 - 10
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
Adams D, Gonzalez-Duarte A, O'Riordan WD, et al.
The New England journal of medicine 2018; (379(1)):11-21 doi:10.1056/NEJMoa1716153.
PMID: 29972753 - 11
An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy.
Planté-Bordeneuve V, Lin H, Gollob J, et al.
Expert opinion on pharmacotherapy 2019; (20(4)):473-481 doi:10.1080/14656566.2018.1554648.
PMID: 30489166 - 12
Comparison between tafamidis and liver transplantation as first-line therapy for hereditary transthyretin amyloidosis.
Socie P, Benmalek A, Cauquil C, et al.
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2023; (30(3)):303-312 doi:10.1080/13506129.2023.2177986.
PMID: 36795029 - 13
TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients.
Moshe-Lilie O, Dimitrova D, Heitner SB, et al.
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2020; (27(4)):250-253 doi:10.1080/13506129.2020.1784134.
PMID: 32578459 - 14
Clinical improvement after change of therapy from tafamidis to patisiran in progressive TTR amyloidosis post-liver transplantation.
Bulinski C, Discher T, Rutsatz W, et al.
Journal of neurology 2022; (269(7)):3912-3914 doi:10.1007/s00415-022-10978-3.
PMID: 35124750 - 15
A narrative review and expert recommendations on the assessment of the clinical manifestations, follow-up, and management of post-OLT patients with ATTRv amyloidosis.
Casasnovas C, Lladó L, Borrachero C, et al.
Therapeutic advances in neurological disorders 2023; (16()):17562864231191590 doi:10.1177/17562864231191590.
PMID: 37655225 - 16
Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation.
Schmidt HH, Wixner J, Planté-Bordeneuve V, et al.
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons 2022; (22(6)):1646-1657 doi:10.1111/ajt.17009.
PMID: 35213769 - 17
Outcomes of Patients With Familial Transthyretin Amyloidosis After Liver Transplantation.
Banerjee D, Roeker LE, Grogan M, et al.
Progress in transplantation (Aliso Viejo, Calif.) 2017; (27(3)):246-250 doi:10.1177/1526924817715463.
PMID: 29187090 - 18
Long-term outcome of patients with hereditary transthyretin V30M amyloidosis with polyneuropathy after liver transplantation.
Okumura K, Yamashita T, Masuda T, et al.
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2016; (23(1)):39-45 doi:10.3109/13506129.2015.1123149.
PMID: 26763274
This page provides educational information on how treatments for ATTRv V30M amyloidosis have evolved over time. Always consult your healthcare provider to determine the safest and most effective treatment plan for your specific condition.
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