🔴 Effect of Tafamidis on Renal Function in Patients With Transthyretin Amyloid Cardiomyopathy in ATTR-ACT #openaccess JACC Journals

sciencedirect.com/science/articl…
#cardiology #CardioTwitter #CardioEd
#meded #cardiotwitter #MedEd #medstudent #paramedic #Cardiology #CardioEd

A targeted therapy towards cardiac amyloidosis? This landmark trial showed tafamidis (a transthyretin binder) to reduce mortality and hospitalization in transthyretin cardiac amyloidosis, prompting earlier screening for this life-threatening disorder. ATTR-ACT Trial, NEJM 2018 ♥️

Wild-type Transthyretin Amyloid Cardiomyopathy

•Age-related, life-threatening disease
•Misfolded transthyretin deposition in myocardium
•Tafamidis: FDA-approved treatment. Other ongoing trials (i.e AG10)
🔑Need for more alternative therapies, esp. for late-stage diagnoses

Effect of Tafamidis on Renal Function in Patients With Transthyretin Amyloid Cardiomyopathy in ATTR-ACT

tafamidis treatment was associated with a reduction in this deterioration, and a higher incidence of improved eGFR and CKD

sciencedirect.com/science/articl…

Effect of long-term tafamidis treatment on health-related quality of life in patients with transthyretin amyloid cardiomyopathy

Tafamidis reduced HRQoL decline in patients with ATTR-CM

onlinelibrary.wiley.com/doi/full/10.10…

In ATTR-ACT, we found that renal function deteriorates over time in patients with ATTR-CM, and tafamidis treatment was associated with a reduction in this deterioration, and a higher incidence of improved eGFR and CKD staging over 30 months vs placebo.

sciencedirect.com/science/articl…

Prof Jeffrey Kelly, giving Cardiology grand Rounds at the UPMC Cardiac Amyloidosis Center on January 24. An inspiring story of scientific discovery, development of the first-in-class drug tafamidis, and entrepreneurship, enthralling an audience of 120 faculty, fellows and staff.

Thank you John Dodson for the opportunity to debate tafamidis in older adults with Mathew Maurer

Most patients never meet the scientist behind life-saving drugs. Now, one patient personally thanked Prof. Jeff Kelly for creating tafamidis. Join our next Front Row lecture as Kelly shares insights on his pioneering research. Register now: ow.ly/LUBq50RsZ8y

$BBIO

Acoramidis produces near-complete TTR stabilization in blood samples from patients with variant transthyretin amyloidosis that is greater than that achieved with tafamidis European Society of Cardiology

ACC 2024. Post hoc analysis of ATTR-ACT. Patients on Tafamidis 80 vs placebo had a higher likelihood of improvement in a range of disease measures after 30m

Dr. Tsai presents early findings on monitoring treatment effects of Tafamidis for ATTR-CM. GLS and parameters of diastolic dysfunction are seen to be stable on echo post-treatment. #ACC23 #WCC #NOLA #ACCFIT #scrippscardiology 📉💊

⚠️Improved long-term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe HF symptoms

👉⤵️all-cause ☠️ with continuous tafamidis treatment vs delayed tafamidis treatment (placebo then tafamidis) in patients with NYHA
class III…

Came across this sobering analysis from Cantor on $BBIO and their arguments look quite convincing. At the end of the day is this another case of spending 7-8 years and hundreds of millions to get a similar clinical profile to that of a marketed drug (tafamidis)?

#HFpEF における非典型的な原因として必ず考慮し、できる限り除外すべきトランスサイレチン型(ATTR)心アミロイドーシス

TTR合成から心筋浸潤までの過程とともに考える #Tafamidis だけではないこの心アミロイドーシスへの治療の現在と未来

現在も色々臨床試験が進行中、益々 #早期診断 が重要に！

🔴Tto sintomático: TMO para IC (mal tolerada), arritmia/bloqueo (control del ritmo, Marcapasos, TRC, ICD),
🔴Tto modificador: estabilizador de TTR (Tafamidis, diflunisal), silenciador Gen (inotersen, Patisiran).
🔴Futuro: terapias de degradación (Acs. Monoclonales contra TTR).

Results:
Acoramidis better than placebo for all secondary endpoints. All-cause mortality close (not powered for this). Mortality curves separate late like with tafamidis.

Almost 50% reduction in CV hospitalization.

Acoramidis also has more TTR stabilization than tafamidis.

$BBIO MGT will provide
Acoramidis updates at ESCHF (5/11)& ISA (5/26), anticipate data that
show differentiation vs. Tafamidis BMO survey indicates ~90% of physicians
view Acoramidis as similar or incrementally better vs. Tafamidis

#HFpEF の鑑別で重要なATTRアミロイド心筋症に対する #acoramidis (tafamidisよりTTR四量体を安定化)🔥

死亡、心血管関連入院、NTproBNP、6分間歩行距離を含む主要アウトカムをプラセボよりも有意に改善！有害事象は両群で同様

tafamidisからの切替も全体で17％(試験開始1年は併用無)

#ATTRibute_CM

Transtiretin amiloid kardiyomiyopati (ATTR-KM) Tanısı ve Tafamidis Tedavisi konulu uluslararası toplantımız gerçekleşti.

Moderatörlüğünü Başakşehir Çam ve Sakura Şehir Hastanesi Prof. Dr. Burcu Esen Akkaş yaptığı etkinlikte, İtalya Policlinico S.Orsola Dr. Rachele Bonfiglioli,