Retrospective Analysis of Mortality in Tafamidis-Treated Japanese Patients with ATTRwt-CM Using the J-COSSA Registry

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  VYNDAQEL
• Medicinal product name, other:
  VYNMAC®
• Study drug International non-proprietary name (INN) or common name:
  TAFAMIDIS
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX08) tafamidis

  tafamidis
• Additional medical condition(s):
  ATTRwt-CM

Population studied

• Short description of the study population:
  Patients with ATTRwt-CM

Study design details

• Study design:
  Retrospective, observational, longitudinal single-cohort study
• Main study objective:
  Objectives Stage 1: ; - To describe data availability and observed follow-up duration and patterns among patients with ATTRwt-CM treated with tafamidis included in the J-COSSA registry ; - To describe patient demographic and baseline characteristics of the study population across predefined analysis sets ; Objectives Stage 2: ; Primary objective: ; - To describe overall survival among patients with ATTRwt-CM following initiation of tafamidis treatment ; Secondary objectives:; - To describe overall survival among patients with ATTRwt-CM following initiation of tafamidis treatment stratified with established baseline prognostic factors such as New York Heart Association (NYHA) class and National Amyloidosis Centre (NAC) ATTR stage ; Exploratory objectives: ; To explore associations between baseline factors and mortality among patients with ATTRwt-CM following initiation of tafamidis treatment
• Setting:
  The study population is derived from the J-COSSA registry, which comprises patients with systemic amyloidosis across Japan, including patients with ATTRwt-CM.