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Brain & Heart Transforming transthyretin cardiac amyloidosis

tafamidis as a targeted therapy for ATTR-CM represents a 2.3. Eligibility requirements
significant advancement in this field. This drug may offer In accordance with the PRISMA 2020 guidelines, the
improved outcomes in affected patients. This systematic following participant, intervention, and outcome criteria
review underscores the importance of early detection were established for study inclusion:
and timely intervention as well as the need for continued • Participants: Studies involving populations diagnosed
research into additional therapies that can further improve with ATTR-CM, regardless of race, sex, or ethnicity,
the prognosis of patients with this debilitating disease. were included in the analysis. The selection was not
13
The use of tafamidis, coupled with advancements in limited by demographic variables to ensure a broad
non-invasive diagnostic techniques, has transformed representation of the disease impact across diverse
ATTR-CM from a rare, incurable condition into a treatable groups
and increasingly recognized condition, leading to better • Intervention: Studies that specifically administered
patient outcomes and prolonged survival.
tafamidis as the primary intervention for treating
2. Methods the above-mentioned population were included in
the analysis. Trials evaluating tafamidis alone or in
2.1. Objective combination with other therapies were considered if
This systematic review aimed to present comprehensive tafamidis was the main therapeutic focus
evidence regarding tafamidis as a novel therapeutic agent • Outcomes: The primary outcome of interest was the
for ATTR-CM. This review was conducted in accordance efficacy of tafamidis in improving clinical symptoms,
with the Preferred Reporting Items for Systematic Reviews quality of life, and/or disease progression in patients
and Meta-Analyses (PRISMA) 2020 guidelines. 14 with ATTR-CM. The secondary outcomes, such as
reduction in all-cause mortality, hospitalization rates,
2.2. Search strategy and adverse effects associated with tafamidis, were

A comprehensive search was conducted across PubMed also considered to ensure a comprehensive evaluation
and Google Scholar to identify relevant articles. The of treatment impact.
search strategy utilized the terms “Tafamidis,” “Cardiac
amyloidosis,” and “Transthyretin amyloid cardiomyopathy” 2.4. Inclusion and exclusion criteria
using BOOLEAN operators “AND” and “OR” to refine the The following additional inclusion criteria were used:
results. A mesh-based approach was employed to further articles written in English; free full-text articles published
narrow the search, reducing the volume of irrelevant within the past 10 years; article types such as cohort studies,
literature. Table 1 shows the total number of articles retrieved case–control studies, randomized control trials (RCTs),
from each database after applying appropriate filters. non-RCTs, case series, case reports, systematic reviews,
To facilitate a deeper exploration of the impact literature reviews, and meta-analyses. Animal studies were
of tafamidis on ATTR-CM, this systematic review not considered in this systematic review.
was extended into a narrative review, as shown in the 3. Results
Discussion section. This approach allowed for a more
detailed and nuanced analysis of the clinical efficacy, safety, Using the appropriate criteria and search methods,
and broader implications of tafamidis in the management 786 articles from the above-mentioned databases
of this condition. published within the past 10 years were retrieved

Table 1. Databases and search strategies
Database Search strategy Filters No. of
articles
PUBMED Tafamidis OR (“Benzoxazoles/administration and dosage” [Mesh] OR “Benzoxazoles/ Free full-text, studies 755
pharmacokinetics” [Mesh] OR “Benzoxazoles/pharmacology” [Mesh] OR “Benzoxazoles/ published in the past
therapeutic use” [Mesh] ) AND Cardiac amyloidosis OR Transthyretin amyloid 10 years, studies on
cardiomyopathy OR (“Amyloid Neuropathies, Familial/drug therapy” [Mesh] OR humans, and studies
“Amyloid Neuropathies, Familial/etiology” [Mesh] OR “Amyloid Neuropathies, Familial/ written in English
genetics” [Mesh] OR “Amyloid Neuropathies, Familial/pathology” [Mesh] OR “Amyloid
Neuropathies, Familial/physiopathology” [Mesh] OR “Amyloid Neuropathies, Familial/
prevention and control” [Mesh] OR “Amyloid Neuropathies, Familial/therapy” [Mesh])
GOOGLE SCHOLAR Allintitle: Tafamidis AND Transthyretin amyloid cardiomyopathy Studies published in 31
the past 10 years

Volume 2 Issue 4 (2024) 3 doi: 10.36922/bh.4250