Study type

Study type

Non-interventional study

Scope of the study

Safety study (incl. comparative)

Data collection methods

Secondary use of data
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Cohort Driven, Non-interventional observational study
Study drug and medical condition

Name of medicine

TEGSEDI

Additional medical condition(s)

Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN)
Population studied

Short description of the study population

Patients with hATTR-PN in Europe, US, and Canada

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

240
Study design details

Study design

This is a non-interventional, international, prospective cohort safety study of TEGSEDI-exposed and TEGSEDI-unexposed patients. Data will be extracted from the medical record via secondary data collection.

Main study objective

To further characterize the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.

Setting

This study will be conducted in Europe, the US, and Canada at centers which manage patients with hATTR such as specialist centers as well as physicians treating amyloidosis (e.g., neurologists, cardiologists, nephrologists, and hematologists).
All patients from each participating site will be invited to participate in the study in order to minimize selection bias. For eligible patients who do not enroll, reason for non-enrollment will be collected.

Comparators

NA

Outcomes

Primary Outcome Measure:

1) Further characterization of the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.
• Determination of the incidence rate of thrombocytopenia in patients with hATTR-PN treated with TEGSEDI (TEGSEDI-exposed cohort)
• Comparison of the relative rates of thrombocytopenia in hATTR-PN patients treated with TEGSEDI (TEGSEDI exposed) to hATTR-PN patients unexposed to TEGSEDI (TEGSEDI- unexposed)
[Time Frame: 10 years]

Secondary Outcome Measures:

2) Description of the incidence rate of the Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
To describe the incidence rate of the following AESIs:
• severe thrombocytopenia
• serious and non-serious bleeding events
• glomerulonephritis
• composite of stroke and/or cervicocephalic arterial dissection
• central nervous system (CNS) vasculitis
• ocular toxicity due to vitamin A deficiency
[Time Frame: 10 years]

3) Description of the time to onset of Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
To describe the time to onset of the following AESIs:
• severe thrombocytopenia
• serious and non-serious bleeding events
• glomerulonephritis
• composite of stroke and/or cervicocephalic arterial dissection
• central nervous system (CNS) vasculitis
• ocular toxicity due to vitamin A deficiency
[Time Frame: 10 years]

Data analysis plan

Demographic and medical history (including but not limited to disease stage, time since diagnosis, prior and concurrent medications use, history of severe thrombocytopenia, glomerulonephritis, stroke, cervicocephalic arterial dissection, central nervous system vasculitis, and ocular toxicity due to vitamin A deficiency will be summarized using descriptive statistics, with number and percent for categorical variables, and n, mean, standard deviation (SD), standard error (SE) of the mean, median, minimum, and maximum for continuous variables. Demographics and medical history will also be summarized using descriptive statistics on different exposure groups (patients who have already been treated with TEGSEDI versus patients entering this study who are initiating TEGSEDI treatment.