OverTTuRe: An Observational Multi-Country Study Collecting Real-World Secondary Data on the Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- AstraZeneca
- 入组人数
- 52,121
- 试验地点
- 34
- 主要终点
- Heart Failure Hospitalization
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
ATTR hereditary
Patients with Transthyretin Hereditary
干预措施: no intervention (Other)
ATTR polyneuropathy
Patients with Transthyretin Amyloidosis Neuropathy
干预措施: no intervention (Other)
ATTR unknown
Patients with ATTR unknown genotype
干预措施: no intervention (Other)
ATTR wild type
Patients with Transthyretin Amyloidosis wild type
干预措施: no intervention (Other)
ATTR cardiomyopathy
Patients with Transthyretin Amyloidosis Cardiomyopathy
干预措施: no intervention (Other)
ATTR with mixed phenotype
Patients with ATTR mixed phenotype
干预措施: no intervention (Other)
结局指标
主要结局
Heart Failure Hospitalization
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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)
时间窗: 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 Impairment Score (NIS)
时间窗: 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
Neuropathy Impairment Score Lower Limbs (NIS-LL)
时间窗: 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)
时间窗: 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)
时间窗: 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 symptoms and change (NCS) score
时间窗: 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)
时间窗: 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
时间窗: 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
次要结局
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)