Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ATTR cardiomyopathy | Patients with Transthyretin Amyloidosis Cardiomyopathy |
| |
| ATTR polyneuropathy | Patients with Transthyretin Amyloidosis Neuropathy |
| |
| ATTR unknown | Patients with ATTR unknown genotype |
| |
| ATTR with mixed phenotype | Patients with ATTR mixed phenotype |
| |
| ATTR wild type | Patients with Transthyretin Amyloidosis wild type |
| |
| ATTR hereditary | Patients with Transthyretin Hereditary |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| no intervention | Other | not applicable, this is an observational retrospective data analysis study; no interventions in the study |
|
| Measure | Description | Time Frame |
|---|---|---|
| Health Care Resource Utilization (HCRU) - Outpatient visits | Annualized events rate for: Overall outpatient visits | 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 | 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 | 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) | 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 | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Health Care Resource Utilization (HCRU) - Outpatient visits | Annualized events rate for: Overall outpatient visits | 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 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
This study will include data from patients aged > 18 years with a diagnosis of ATTR amyloidosis or other evidence confirming ATTR amyloidosis. Patients will be followed until exit from the database (loss to follow-up), death, or end of database period (data collection). Baseline data will apply a lookback period of 12 months. Patient identification will be from 2014-01-01 onwards
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| AstraZeneca Clinical Study Information Center | Contact | 1-877-240-9479 | information.center@astrazeneca.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Completed | Eden Prairie | Minnesota | 55344 | United States | |
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42201638 | Derived | Alexander KM, Kohsaka S, Poulsen SH, Terkelsen AJ, Smith JG, Sundstrom J, Wright J, Jarbrink K, Bhimjiyani AG, Davis L, Matsue Y, Anderson LJ, Pilebro B. Contemporary Description of Clinical Characteristics and Outcomes in Patients with Hereditary ATTR Amyloidosis: Results from the Multicountry OverTTuRe Study. Cardiol Ther. 2026 Jun;15(2):201-229. doi: 10.1007/s40119-026-00450-y. Epub 2026 May 27. | |
| 40108078 |
Not provided
Not provided
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Not provided
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org.
Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Not provided
Not provided
Not provided
Not provided
| 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) | 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) | 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) | 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) | 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) | 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) | 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 | 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 | 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 | 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 | 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 | 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 | 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 | From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years |
Annualized events rate for: Overall outpatient visits by specialty |
| 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 | 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) | 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 | 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 | From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. |
| Recruiting |
| Calgary |
| Alberta |
| T2P 4K7 |
| Canada |
| Research Site | Completed | Beijing | China |
| Research Site | Completed | Changsha | China |
| Research Site | Completed | Chengdu | China |
| Research Site | Completed | Guangzhou | China |
| Research Site | Completed | Wuhan | China |
| Research Site | Completed | Horsens | Denmark |
| Research Site | Completed | Berlin | Germany |
| Research Site | Completed | Tokyo | Japan |
| Research Site | Recruiting | Lisbon | Portugal |
| Research Site | Not yet recruiting | Porto | Portugal |
| Research Site | Completed | A Coruña | Spain |
| Research Site | Completed | Barcelona | Spain |
| Research Site | Completed | Bilbao | Spain |
| Research Site | Completed | El Palmar | Spain |
| Research Site | Completed | Huelva | Spain |
| Research Site | Completed | Las Palmas de Gran Canaria | Spain |
| Research Site | Completed | Madrid | Spain |
| Research Site | Completed | Majadahonda | Spain |
| Research Site | Completed | Palma de Mallorca | Spain |
| Research Site | Completed | Salamanca | Spain |
| Research Site | Completed | Valencia | Spain |
| Research Site | Completed | Södertälje | Sweden |
| Research Site | Completed | Umeå | Sweden |
| Research Site | Completed | London | United Kingdom |
| Derived |
| Gillmore JD, Hahn K, Smith JG, Conceicao I, Tian Z, Grogan M, Pao C, Wittbrodt E, Jarbrink K, Papas MA, Davis MK. Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care. Cardiol Ther. 2025 Sep;14(3):477-490. doi: 10.1007/s40119-025-00402-y. Epub 2025 Mar 19. |