A retrospective observational analysis of the realworld care pathway of people with hereditary transthyretin amyloidosis with polyneuropathy in Italy

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Healthcare resource utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Polyneuropathy

  Hereditary neuropathic amyloidosis

Population studied

• Short description of the study population:
  This was a retrospective observational cohort study using administrative healthcare data for descriptive analysis of ~5.5 million inhabitants, which represent ~ 9% of the Italian population, and the age distribution of which is consistent with that obtained by the Italian Institute of Statistics. ; People with ATTRv-PN were identified from 1 January 2021 to 31 December 2021 (accrual period) if at least one specific drug (ATC – Anatomical Therapeutic Chemical codes: N07×X08– tafamidis through its Italian marketing authorization code of the 20 mg box, N07×X12– patisiran, N07×X15– inotersen) was dispensed between 2014 and 2021.
• Age groups:
  • In utero
  • Paediatric Population (< 18 years)

    • Neonate

      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Study design details

• Study design:
  Retrospective observational cohort study. Accrual period 01/01-31/12/2021. Index date: dispensation of specific drug (tafamidis 20 mg, patisiran, inotersen) from 2014 to 2021. ; Baseline characteristics (demographics and comorbidities). 1-year healthcare resource utilization and related direct costs.
• Main study objective:
  This retrospective observational study of Italian administrative healthcare data aimed to identify people with ATTRv-PN in 2021, and describe their demographics, comorbidities, healthcare resource consumption and related direct costs reimbursed by the Italian National Healthcare Service (Servizio Sanitario Nazionale – SSN).
• Setting:
  Italian inhabitants or beneficiaries of the SSN. Overall observation period 2014-2022. Inhospital, local outpatient and primary care settings.
• Summary results:
  From 5.5 million inhabitants of the ReS database in 2021, 36 patients with ATTRv-PN (prevalence: 7.4/1,000,000) were identified (males were 83.3%; patients with ≥2 comorbidities were 61.1%; the mean age was 73 ± 8 years). ; During follow-up, of patients, 91.7% received drugs for ATTRv-PN; >50% received antiepileptics and acid suppressants; 22.2% were admitted to overnight hospitalizations; 30.6% accessed the emergency department; 97.2% received local outpatient specialist care. The per patient mean annual cost was € 122,017; drugs for ATTRv-PN accounted for 94.7% of the total expenditure.