Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis

Wang, Jie; Chen, Hongyu; Tang, Zihuan; Zhang, Jinquan; Xu, Yuanwei; Wan, Ke; Hussain, Kifah; Gkoutos, Georgios V.; Han, Yuchi; Chen, Yucheng

doi:10.1016/j.eclinm.2023.102172

Cited by 6 publications

(5 citation statements)

References 37 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Although tafamidis cannot stop the progression of CA, tafamidis is able to slow it in some cases, particularly in the early-stage cases, and it is associated with reduced deterioration in the left ventricular ejection fraction (LVEF), especially in those with wtATTR. Additionally, studies have highlighted that tafamidis delays both structural and functional changes in the left ventricle [5,6], which is also supported by a very recent meta-analysis, indicating no significant LVEF decrease after treatment with tafamidis [7]. Lastly, long-term data suggest that tafamidis improves survival and quality of life, with significant benefits in terms of functional capacity [8].…”

Section: Introductionmentioning

confidence: 74%

“…However, he still complained of paresthesia in both the upper (especially at the left fingers) and lower limbs (more at the left ankle), although the neurological examination remained within the normal limits. The HDRS score was borderline (7), whereas MoCA improved to a normal value (28). The patient was recently re-visited: he was still on tafamidis and appeared to be cardiologically stable, with improved effort tolerance, although symptoms of peripheral nervous system dysfunction persisted.…”

Section: Case Reportmentioning

confidence: 99%

See 1 more Smart Citation

Interdisciplinary Multidimensional Assessment of Transthyretin Amyloidosis before and after Tafamidis

Pennisi,

Lanza,

Vitello

et al. 2023

Life

View full text Add to dashboard Cite

Background: Clinically, there is considerable heterogeneity in the presentation of transthyretin amyloidosis (ATTR), which ranges from primarily cardiac and primarily neurologic to mixed disease, among other manifestations. Because of this complex presentation, the diagnosis and management of patients with ATTR are often challenging and should be performed in interdisciplinary centers specialized in amyloidosis. Here, we aimed to increase awareness of ATTR detection and pathophysiology through a multidimensional multiorgan approach. Case report: We reported on a 60-year-old man with wild-type ATTR who underwent a number of both basic and advanced cardiological and neurological investigations at baseline and after a treatment period with the TTR tetramer stabilizer, tafamidis. Several findings are provided here, some of which might be considered instrumental correlates of the patient’s clinical improvement after therapy. Conclusions: Adequate awareness and prompt recognition of ATTR support early diagnosis and faster access to therapies, thereby slowing the progression and improving the prognosis. The need for a multidisciplinary alliance between specialists and the opportunity to perform, at least in selected cases, a set of specific examinations for a detailed assessment of ATTR patients can also provide valuable insights into the physiopathology and response to therapy of a disease as complex and intriguing as ATTR.

show abstract

Section: Introductionmentioning

confidence: 74%

Section: Case Reportmentioning

confidence: 99%

Interdisciplinary Multidimensional Assessment of Transthyretin Amyloidosis before and after Tafamidis

Pennisi,

Lanza,

Vitello

et al. 2023

Life

View full text Add to dashboard Cite

show abstract

“…Results from ATTR‐ACT and a recent meta‐analysis, incorporating outcome data from all suitable studies conducted to date, demonstrate that tafamidis prolongs survival and reduces cardiovascular hospitalizations in ATTR‐CA. 9 , 12 We herewith add to these data by reporting overall and MACE‐free survival among one of the largest and the first German real‐world cohort of patients with ATTR‐CA.…”

Section: Discussionmentioning

confidence: 99%

Outcomes in Cardiac Transthyretin Amyloidosis and Association With New York Heart Association Class: Real‐World Data

Müller,

Latinova,

Brand

et al. 2024

JAHA

View full text Add to dashboard Cite

Background Results from ATTR‐ACT (Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy) indicate that tafamidis prolongs survival and reduces cardiovascular hospitalizations in cardiac transthyretin amyloidosis (ATTR‐CA). However, real‐world data supporting these findings are scarce. Thus, we sought to characterize the clinical outcome of patients with ATTR‐CA treated with tafamidis in a real‐world setting and assess the prognostic role of the New York Heart Association (NYHA) classification. Methods and Results We conducted a retrospective observational study, enrolling a consecutive sample of patients with ATTR‐CA (wild‐type or variant) treated with tafamidis. Clinical outcome was tracked through follow‐up visits or phone calls. Primary outcomes were death and major adverse cardiac events (MACE), a composite end point of death and hospitalizations for acute cardiac decompensation, myocardial infarction, severe arrythmias, or stroke. Kaplan‐Meier analysis estimated overall and MACE‐free survival including NYHA subgroups (NYHA I/II versus NYHA III). One hundred sixty‐seven patients with ATTR‐CA (94.6% wild‐type) were enrolled and followed for a median of 539 [323–869] days. Median overall survival was not reached. Estimated 1‐year, 2‐year, and 5‐year overall survival among the whole cohort was 93.5%, 85.9%, and 70.2%, respectively. Overall survival was higher in the NYHA I/II subgroup ( P =0.002). Median MACE‐free survival time was 1082 (95% CI, 962–1202) days. MACE‐free survival was higher in the NYHA I/II subgroup ( P <0.001). With respective hazard ratios of 5.85 (95% CI, 1.48–23.18; P =0.012) and 3.95 (95% CI, 1.99–7.84; P <0.001), NYHA III was an independent predictor of death and MACE. Conclusions Treatment of ATTR‐CA with tafamidis led to substantial improvements of clinical outcome. NYHA classification at treatment initiation is a reliable tool to provide patients with individualized prognostic information.

show abstract

“…While the impact of Tafamidis on all-cause mortality and cardiovascular-related hospitalization is remarkable little is known about the autonomic effect, post-therapeutic quality of life, and other cardiovascular parameters, and lack of evidence of the degree of stabilization to which approved doses exhibit fullest potential [ 13 ]. Most real-world studies lack large sample sets, sufficient follow-up periods, and equal representation of the subtypes precluding the availability of evidence regarding long-term efficacy and generalizability associated with Tafamidis treatment [ 20 ]. A noteworthy lack of real-world studies evaluating changes in a comprehensive set of echocardiographic parameters after Tafamidis treatment at follow-up exists in the literature.…”

Section: Main Textmentioning

confidence: 99%

“…A meta-analysis of 15 studies comprising 2765 patients revealed a significant Tafamidis-associated reduction in the composite endpoint of all-cause death, heart transplant, heart assist device implantation, heart failure exacerbations, and hospitalization in both ATTR types [ 20 ].…”

Section: Real-world Evidence (Table 2 )mentioning

confidence: 99%

Tafamidis therapy in transthyretin amyloid cardiomyopathy: a narrative review from clinical trials and real-world evidence

Ogieuhi,

Ugiomoh,

Muzofa

et al. 2024

Egypt Heart J

View full text Add to dashboard Cite

Background Amyloidosis is a heterogeneous group of disorders caused by the extracellular deposition of insoluble misfolded proteins, leading to end-organ damage. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a subtype in which a protein known as transthyretin accumulates within the heart tissue, progressively resulting in restrictive cardiomyopathy and heart failure. Due to the progressive nature of ATTR-CM, clinical management requires efficacious regimens to manage the debilitating condition and Tafamidis shows promising results in this regard. Main body ATTR-CM poses a significant challenge due to its nature and limited therapeutic options. Tafamidis is a novel therapy designed to stabilize the transthyretin tetramers, inhibiting the formation of amyloid fibrils. It has emerged as a promising treatment and the only FDA-approved drug for ATTR-CM. Tafamidis' role in slowing disease progression and improving outcomes in patients with ATTR-CM has been demonstrated in the major randomized control trial ATTR-ACT with promising open-label extension studies, some still ongoing. Additionally, real-world evidence supports its use in clinical practice, showing its role in reducing morbidity and mortality associated with this condition. Clinical evidence shows its efficacy in improving symptoms and cardiac function in patients. Case studies also reveal significant benefits to patients like reducing myocardial damage, reversal of atrial fibrillation, and resolution of heart failure symptoms. Real-world outcomes and clinical trials show a consistent reduction in amyloid deposition, cardiovascular-related hospitalizations, and all-cause mortality with Tafamidis therapy. Conclusion Tafamidis is an essential component of the treatment of ATTR-CM and this narrative review synthesizes the current evidence regarding safety, efficacy, and utilization in real practice. While it shows promising effects, its effectiveness may also vary and high cost precludes real-world large-scale studies. Overall, Tafamidis emerges as a valuable therapeutic option for managing ATTR-CM.

show abstract

Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis

Cited by 6 publications

References 37 publications

Interdisciplinary Multidimensional Assessment of Transthyretin Amyloidosis before and after Tafamidis

Interdisciplinary Multidimensional Assessment of Transthyretin Amyloidosis before and after Tafamidis

Outcomes in Cardiac Transthyretin Amyloidosis and Association With New York Heart Association Class: Real‐World Data

Tafamidis therapy in transthyretin amyloid cardiomyopathy: a narrative review from clinical trials and real-world evidence

Contact Info

Product

Resources

About