OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
- Conditions
- ATTR Amyloidosis
- Interventions
- Other: no intervention
- Registration Number
- NCT06355934
- Lead Sponsor
- AstraZeneca
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 55000
- Patients aged >18 years at study index date AND
- A reported diagnosis code for amyloidosis OR
- A claim for ATTR-specific treatment OR
- A positive biopsy for amyloidosis and positive immunostaining result of biopsy for ATTR
- Evidence of primary (AL) and secondary (AA) amyloidosis AND/OR
- At least one claim/procedure code for stem cell transplant or at least two claims/procedure codes for chemotherapy and autoimmune disease drugs which may represent AL (primary) or AA (secondary) amyloidosis treatments
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description ATTR with mixed phenotype no intervention Patients with ATTR mixed phenotype ATTR cardiomyopathy no intervention Patients with Transthyretin Amyloidosis Cardiomyopathy ATTR unknown no intervention Patients with ATTR unknown genotype ATTR wild type no intervention Patients with Transthyretin Amyloidosis wild type ATTR polyneuropathy no intervention Patients with Transthyretin Amyloidosis Neuropathy ATTR hereditary no intervention Patients with Transthyretin Hereditary
- Primary Outcome Measures
Name Time Method Neuropathy Impairment Score Lower Limbs (NIS-LL) From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years The NIS-LL score has a range of 0-88 and a greater NIS-LL score indicates poorer function
Neuropathy Impairment Score +7 (NIS+7) From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS+7 score indicates poorer function
Neuropathy Impairment Score modified +7 (mNIS+7) From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years The mNIS+7 Score has a range of -22.32 to 102.32 and a higher mNIS+7 score indicates poorer function
Neuropathy Impairment Score (NIS) From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years The NIS Score has a range of 0 to 244 and a higher NIS score indicates poorer function
Heart Failure Hospitalization 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 Heart failure hospitalization (time-to-event)
Health Care Resource Utilization (HCRU) - 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 Annualized events rate for: Overall outpatient visits
Health Care Resource Utilization (HCRU) - 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 Annualized events rate for: Overall outpatient visits by specialty
Health Care Resource Utilization (HCRU) - 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 Annualized events rate for: Emergency department visits
Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay 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: Hospitalizations (bed days)
Health Care Resource Utilization (HCRU) - Hospitalizations 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: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits
Health Care Resource Utilization (HCRU) - Health care cost From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years 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
Cardiac transplant 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 Cardiac transplant (Time to event)
All cause mortality 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 Mortality (time-to-event)
Liver transplant 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 Liver transplant (time-to-event)
New ATTR amyloidosis clinical manifestation 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 New amyloidosis manifestation (time-to-event); Time to the first development of a new clinical manifestation that the patient did not have pre-index)
Hospitalization (any 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 Time to hospitalization (all-cause)
Neuropathy symptoms and change (NCS) score From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years 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
PND (Polyneuropathy Disability) From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years 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
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 Any other relevant clinical measurement of ATTR amyloidosis functional status
- Secondary Outcome Measures
Name Time Method Health Care Resource Utilization (HCRU) - Outpatient visits From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. Annualized events rate for: Overall outpatient visits
Health Care Resource Utilization (HCRU) - 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. Annualized events rate for: Overall outpatient visits by specialty
Health Care Resource Utilization (HCRU) - Emergency department visits From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. Annualized events rate for: Emergency department visits
Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. Annualized events rate for: Hospitalizations (bed days)
Health Care Resource Utilization (HCRU) - Hospitalizations From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. Annualized events rate for: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits
Health Care Resource Utilization (HCRU) - Health care cost From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years. 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
Trial Locations
- Locations (1)
Research Site
🇬🇧London, United Kingdom