What is the clinical evidence from this study?

Study design: Review. Population: Transthyretin amyloid cardiomyopathy (ATTR-CM).

What question did this study set out to answer?

To explore the causes, diagnostic challenges, and treatment strategies for transthyretin amyloid cardiomyopathy (ATTR-CM).

March 30, 2026Open Access

Transtiretino Amiloidinė Kardiomiopatija: Etiologija, Diagnostika Ir Gydymas

Key Result

Tafamidis is currently the only approved disease-modifying treatment for transthyretin amyloid cardiomyopathy, while emerging therapies like RNA silencers and gene editing are still being investigated.

Key Points

To explore the causes, diagnostic challenges, and treatment strategies for transthyretin amyloid cardiomyopathy (ATTR-CM).
Review of ATTR-CM etiology including genetic mutation (ATTRv) and age-related changes (ATTRwt).
Assessment of symptoms and diagnostic delays due to non-specific manifestations.
Examination of current treatment options including tafamidis and potential new therapies.
ATTR-CM is linked to unstable transthyretin protein accumulation in the heart.
Diagnosis is often delayed due to symptoms mimicking other heart conditions.
Tafamidis is currently the only approved drug for ATTR-CM, while new therapies are being investigated.

PICO

Population

Transthyretin amyloid cardiomyopathy (ATTR-CM)

Limitations

The true epidemiology of ATTR-CM remains unclear.
Long-term safety and efficacy of emerging alternative therapies are still being investigated.

Abstract

Transtiretino amiloidinė kardiomiopatija (ATTR-CM) yra reta ir pavojinga širdies liga, kurią lemia nestabilaus transtiretino (TTR) baltymo kaupimasis miokardo tarpląstelinėje erdvėje. Ši liga gali būti susijusi su genetine mutacija (ATTRv) arba atsirasti dėl amžinių pokyčių (ATTRwt). Liga dažnai pasireiškia nespecifiniais simptomais, imituojančiais kitas širdies patologijas, todėl diagnozė dažnai nustatoma pavėluotai, atsiradus sunkiai ligos formai. Šiuo metu ATTR-CM terapinis planas apima simptominį ir ligos eigą modifikuojantį gydymą. Nors specifinis transtiretino stabilizatorius tafamidis šiuo metu yra vienintelis ATTR-CM gydymui patvirtintas vaistas, naujos alternatyvios terapijos, tokios kaip TTR koduojančių RNR slopikliai ir genų redagavimo sistemos, turi perspektyvą. Jų ilgalaikis poveikis dar tyrinėjamas.

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Discussion

Authors

Ugnė Janonytė

Gertrūda Dagytė

Laima Kniaževienė

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Transtiretino Amiloidinė Kardiomiopatija: Etiologija, Diagnostika Ir Gydymas

Key Result

Key Points

PICO

Limitations

Abstract

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Authors

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References and Citations

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