OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis

OTHER

no intervention

not applicable, this is an observational retrospective data analysis study; no interventions in the study

Health Care Resource Utilization (HCRU) - 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

Annualized events rate for: Overall outpatient visits

Health Care Resource Utilization (HCRU) - 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

Annualized events rate for: Overall outpatient visits by specialty

Health Care Resource Utilization (HCRU) - 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

Annualized events rate for: Emergency department visits

Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay

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

Annualized events rate for: Hospitalizations (bed days)

Health Care Resource Utilization (HCRU) - Hospitalizations

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

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

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

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

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

Cardiac transplant (Time to event)

All cause mortality

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

Mortality (time-to-event)

Liver transplant

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

Liver transplant (time-to-event)

Heart Failure Hospitalization

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

Heart failure hospitalization (time-to-event)

New ATTR amyloidosis clinical manifestation

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

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)

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

Time to hospitalization (all-cause)

Neuropathy Impairment Score (NIS)

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

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)

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

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)

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

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)

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

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

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

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)

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

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

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

Any other relevant clinical measurement of ATTR amyloidosis functional status

What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.