The overall aim of this observational study is to generate real-world evidence on the pre- and post-diagnosis disease journeys, including baseline characteristics, treatment patterns and selected clinical, economic, and humanistic outcomes (for example Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments) in patients with ATTR amyloidosis, and to better understand how the disease is presented.
This retrospective, observational, longitudinal, multi-country cohort study aims to describe characteristics, treatment patterns, and outcomes in ATTR amyloidosis patients. Primary objectives: Describe patient characteristics (for example demographics, family history of ATTR, key comorbidities and humanistic outcomes (e.g. Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments), treatment patterns, and disease outcomes. Characterize and quantify the healthcare resource utilization (HCRU) in ATTR amyloidosis patients who will be followed post-index until the end of follow-up. Secondary objectives: Describe demographics, clinical characteristics and HCRU in ATTR amyloidosis patients prior to diagnosis.
Study Type
OBSERVATIONAL
Enrollment
55,000
not applicable, this is an observational retrospective data analysis study; no interventions in the study
Research Site
Eden Prairie, Minnesota, United States
COMPLETEDHealth Care Resource Utilization (HCRU) - Outpatient visits
Annualized events rate for: Overall outpatient visits
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Outpatient visits by specialty
Annualized events rate for: Overall outpatient visits by specialty
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Emergency department visits
Annualized events rate for: Emergency department visits
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay
Annualized events rate for: Hospitalizations (bed days)
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Hospitalizations
Annualized events rate for: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Health care cost
This variable will describe all-cause direct medical and pharmacy costs only, utilizing the amount charged, where available in the data. The direct medical costs will include costs incurred from inpatient stays, outpatient visits, emergency room visits, procedures, and laboratory tests. The inclusion of pharmacy costs is subject to data availability and will include all pharmacy costs per patient separated on pharmacy dispensed and in-hospital dispensed when possible. When feasible, costs will be divided on all-cause, CV, and other specialties
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Research Site
Calgary, Alberta, Canada
RECRUITINGResearch Site
Beijing, China
COMPLETEDResearch Site
Changsha, China
COMPLETEDResearch Site
Chengdu, China
COMPLETEDResearch Site
Guangzhou, China
COMPLETEDResearch Site
Wuhan, China
COMPLETEDResearch Site
Horsens, Denmark
COMPLETEDResearch Site
Berlin, Germany
COMPLETEDResearch Site
Tokyo, Japan
COMPLETED...and 16 more locations
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Cardiac transplant
Cardiac transplant (Time to event)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of first cardiac transplant, assessed throughout the study until end of follow-up, up to a maximum of 12 years
All cause mortality
Mortality (time-to-event)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of death due to any cause, assessed throughout the study until end of follow-up, up to a maximum of 12 years
Liver transplant
Liver transplant (time-to-event)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of first liver transplant, assessed throughout the study until end of follow-up, up to a maximum of 12 years
Heart Failure Hospitalization
Heart failure hospitalization (time-to-event)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of first hospitalization for heart failure, assessed throughout the study until end of follow-up, up to a maximum of 12 years
New ATTR amyloidosis clinical manifestation
New amyloidosis manifestation (time-to-event); Time to the first development of a new clinical manifestation that the patient did not have pre-index)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of first diagnosis of new ATTR amyloidosis clinical manifestation, assessed throughout the study until end of follow-up, up to a maximum of 12 years
Hospitalization (any cause)
Time to hospitalization (all-cause)
Time frame: From diagnosis of ATTR amyloidosis (index date) until date of first hospitalization for any reason, assessed throughout the study until end of follow-up, up to a maximum of 12 years
Neuropathy Impairment Score (NIS)
The NIS Score has a range of 0 to 244 and a higher NIS score indicates poorer function
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Neuropathy Impairment Score Lower Limbs (NIS-LL)
The NIS-LL score has a range of 0-88 and a greater NIS-LL score indicates poorer function
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Neuropathy Impairment Score +7 (NIS+7)
The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS+7 score indicates poorer function
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Neuropathy Impairment Score modified +7 (mNIS+7)
The mNIS+7 Score has a range of -22.32 to 102.32 and a higher mNIS+7 score indicates poorer function
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Neuropathy symptoms and change (NCS) score
NSC score is a questionnaire composed of 38 questions that assess the presence and severity of these neuropathy symptoms. The NSC score ranges from -114 to 114 for males and -108 to 108 for females. Greater scores indicate worse symptom severity; a negative value indicates an improvement in symptom severity from baseline
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
PND (Polyneuropathy Disability)
The PND is a 6-stage scoring system: Stage 0: no impairment; Stage 1: sensory disturbances but preserved walking capabilities; Stage 2: impaired walking capacity, but ability to walk without a stick or crutches; Stage 3A/B: walking with help of 1 or 2 sticks or crutches; Stage 4: confined to wheel chair or bedridden. A greater stage indicates greater impairment
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Other relevant clinical measurement of ATTR amyloidosis functional status
Any other relevant clinical measurement of ATTR amyloidosis functional status
Time frame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Health Care Resource Utilization (HCRU) - Outpatient visits
Annualized events rate for: Overall outpatient visits
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Health Care Resource Utilization (HCRU) - Outpatient visits by specialty
Annualized events rate for: Overall outpatient visits by specialty
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Health Care Resource Utilization (HCRU) - Emergency department visits
Annualized events rate for: Emergency department visits
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay
Annualized events rate for: Hospitalizations (bed days)
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Health Care Resource Utilization (HCRU) - Hospitalizations
Annualized events rate for: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Health Care Resource Utilization (HCRU) - Health care cost
This variable will describe all-cause direct medical and pharmacy costs only, utilizing the amount charged, where available in the data. The direct medical costs will include costs incurred from inpatient stays, outpatient visits, emergency room visits, procedures, and laboratory tests. The inclusion of pharmacy costs is subject to data availability and will include all pharmacy costs per patient separated on pharmacy dispensed and in-hospital dispensed when possible. When feasible, costs will be divided on all-cause, CV, and other specialties
Time frame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.