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

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Study 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.

Eligibility

Primary outcome measure(s)

• 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)
• 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)
• 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 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
• 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 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

Trial sites (29)

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