Observatory of ATTR amyloidosis and of patients treated with Vyndaqel® (tafamidis) OBSAMYL study

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation

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

Non-interventional study

• Non-interventional study design:
  Cross-sectional

  Other
• Non-interventional study design, other:
  Restrospective, secondary data collection study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  TAFAMIDIS
• Medical condition to be studied:
  Hereditary neuropathic amyloidosis

  Acquired ATTR amyloidosis

  Cardiac amyloidosis

Population studied

• Short description of the study population:
  All centres having the ability to treat patients for this disease have been invited to participate on behalf of the scientific committee. They include the national reference center for amyloidosis, and several regional competence centres; Patients Eligibility for the Census Part:; - Patient ≥18 years old;; - With a diagnosis of hereditary or wild-type ATTR amyloidosis or a pathogenic TTR mutation in participating centres and alive on 1st June 2017;; - Who does not oppose to his/her data collection; No Exclusion criteria; Patients Eligibility for tafamidis part:; Inclusion criteria; - Patient ≥ 18 years old;; - With at least one documented prescription of tafamidis (Vyndaqel®) outside of a clinical trial since its launch in France through an Early Access Programme (EAP); Exclusion criteria; - Patient who participated and received tafamidis only within an interventional study evaluating the efficacy of tafamidis at time of data collection
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  1300

Study design details

• Main study objective:
  1) To estimate the number of alive patients diagnosed with TTR-related amyloidosis (hereditary and senile) in France on 1st June 2017, globally, by stage of the disease, and type of mutation if applicable 2) To evaluate, in a real-life setting, modalities of tafamidis use, its safety and effectiveness profile for patients who have received tafamidis in France whether or not they are still alive
• Outcomes:
  - Estimation of all cases of TTR-related amyloidosis (hereditary and senile) patients (census population) : defined in the study as the total number of patients with TTR-related amyloidosis, alive on 1st June 2017 and diagnosed in participating centers providing care for TTR-related amyloidosis. - Description of modalities of tafamidis use, and its safety and effectiveness profile, - Description of the patient's family potentially carrying a pathogenic TTR mutation (census population except for senile amyloidosis) - Time between first symptom(s) and diagnosis - Estimation of the number of patients with pathogenic TTR mutations
• Data analysis plan:
  Statistical analyses will be explorative and descriptive nature. The analysis will be conducted using SAS (version 9.4 or higher, SAS Institute, North Carolina USA). All CRF parameters and evaluation criteria will be summarized using descriptive statistics: −Sample size, mean, standard deviation, minimum, median, Q1, Q3, maximum, 95% CI and number of missing data for continuous variables, −Frequency and percentage for each category, 95% CI and number of missing data for discrete variables.

Documents

Study results

Summary_results_V6.6 rapport_20201014