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

Официальное название

OverTTuRe: An Observational Multi-Country Study Collecting Real-World Secondary Data on the Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis

Обзор

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.

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Health Care Resource Utilization (HCRU) - Outpatient visits [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Health Care Resource Utilization (HCRU) - Outpatient visits by specialty [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Health Care Resource Utilization (HCRU) - Emergency department visits [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Health Care Resource Utilization (HCRU) - Hospitalizations [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Health Care Resource Utilization (HCRU) - Health care cost [Срок оценки: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years]
• Cardiac transplant [Срок оценки: 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]
• All cause mortality [Срок оценки: 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]
• Liver transplant [Срок оценки: 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]
• Heart Failure Hospitalization [Срок оценки: 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]

Вторичные конечные точки (6)

• Health Care Resource Utilization (HCRU) - Outpatient visits [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Испания · 11 центров

• Research Site — A Coruña
• Research Site — Barcelona
• Research Site — Bilbao
• Research Site — El Palmar
• Research Site — Huelva
• Research Site — Las Palmas de Gran Canaria
• Research Site — Madrid
• Research Site — Majadahonda
• … и ещё 3 центра

Китай · 5 центров

• Research Site — Пекин
• Research Site — Чанша
• Research Site — Чэнду
• Research Site — Гуанчжоу
• Research Site — Ухань

Италия · 3 центра

• Research Site — Lecce
• Research Site — Messina
• Research Site — Milan

Португалия · 2 центра

• Research Site — Lisbon
• Research Site — Porto

Швеция · 2 центра

• Research Site — Södertälje
• Research Site — Umeå

США · 1 центр

• Research Site — Eden Prairie

Канада · 1 центр

• Research Site — Calgary

Дания · 1 центр

• Research Site — Horsens

Германия · 1 центр

• Research Site — Berlin

Япония · 1 центр

• Research Site — Tokyo

Великобритания · 1 центр

• Research Site — London

Публикации

• Alexander KM, Kohsaka S, Poulsen SH, Terkelsen AJ, Smith JG, Sundstrom J, Wright J, Jarbrink K, Bhimjiyani AG, Davis L, Matsue Y, Anderson LJ, Pilebro B. Contemporary Description of Clinical Characteristics and Outcomes in Patients with Hereditary ATTR Amyloidosis: Results from the Multicountry OverTTuRe Study. Cardiol Ther. 2026 Jun;15(2):201-229. doi: 10.1007/s40119-026-00450-y. Epub 2026 May 27 PMID 42201638
• Gillmore JD, Hahn K, Smith JG, Conceicao I, Tian Z, Grogan M, Pao C, Wittbrodt E, Jarbrink K, Papas MA, Davis MK. Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care. Cardiol Ther. 2025 Sep;14(3):477-490. doi: 10.1007/s40119-025-00402-y. Epub 2025 Mar 19. PMID 40108078

Идентификаторы

NCT: NCT06355934 · D8450R00004

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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