A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany

CHARACTERISTICS, SURVIVAL, HOSPITALIZATIONS AND EPIDEMIOLOGY IN ATTR-CM PATIENTS IN GERMANY: NATION-WIDE RETROSPECTIVE HEALTHCARE CLAIMS DATA ANALYSIS

This observational study aims to describe the characteristics, health outcomes, and epidemiology of individuals diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany.

ATTR-CM is a rare and serious heart disease caused by the buildup of an abnormal protein called transthyretin in the heart muscle. This can lead to heart failure and other cardiovascular complications.

This is a retrospective, non-interventional study based on anonymized statutory health insurance claims data from Germany. The study includes adults with a new diagnosis of ATTR-CM identified between 2020 and 2022. Data from both inpatient and outpatient healthcare settings are analyzed. No participants are recruited, and no medical treatments, procedures, or study visits are assigned as part of this study.

The study will describe overall survival, cardiovascular-related hospitalizations, and the incidence of ATTR-CM in Germany. A predefined subgroup of individuals with at least one documented prescription for tafamidis will be analyzed separately to describe outcomes in this population.

In addition, the study will examine the use of genetic testing after diagnosis and describe patient characteristics, as observed in routine clinical care.

By using nationwide healthcare data, this study aims to provide a comprehensive and recent real-world overview of ATTR-CM in Germany and to support a better understanding of the disease.

Study Overview

• Status: Active, not recruiting
• Conditions: ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
• Intervention / Treatment: Other: tafamidis 61mg (based on prescription data)

• Study Type: Observational
• Enrollment (Estimated): 1

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany, 10117
    • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population consists of adults insured under the German statutory health insurance system who were newly identified with transthyretin amyloid cardiomyopathy (ATTR-CM) between 2020 and 2022.

Description

Inclusion Criteria:

Adults aged 18 years or older at the time of the index date

Individuals with a newly identified transthyretin amyloid cardiomyopathy (ATTR-CM) diagnosis between January 2020 and December 2022, identified using a validated claims-based algorithm

Evidence of amyloidosis, defined as:

At least one inpatient diagnosis or At least two outpatient diagnoses in separate quarters

Evidence of cardiac involvement, defined as at least one diagnosis of cardiomyopathy or heart failure occurring within a defined temporal window before or after the amyloidosis diagnosis

Continuous enrollment in German statutory health insurance for:

At least 8 quarters prior to the index date, and At least 4 consecutive quarters after the index date (or until death)

Exclusion Criteria:

Evidence of light-chain (AL) amyloidosis, including:

AL-specific treatment prescriptions Chemotherapy associated with amyloidosis Diagnosis of multiple myeloma autologous stem cell transplantation

Diagnosis of amyloid angiopathy

Prior amyloidosis diagnosis during the 2-year wash-out period before the index date Missing or implausible information on sex

Additional Criteria for Tafamidis Subgroup At least one prescription of tafamidis 61 mg during follow-up No prescriptions for other disease-modifying therapies for transthyretin amyloidosis with polyneuropathy

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Patients with incident transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany; Adult patients (≥18 years) with incident transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany, identified using a claims-based algorithm in nationwide statutory health insurance data. Incident cases are defined based on a washout period of at least two years without prior amyloidosis diagnosis. Patients are followed from the date of ATTR-CM diagnosis. Subset of the incident ATTR-CM population with at least one documented prescription of tafamidis 61 mg during follow-up will be included.

Other: tafamidis 61mg (based on prescription data); As provided in real world practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall Survival in Individuals With Incident Transthyretin Amyloid Cardiomyopathy (ATTR-CM), Overall and in Those With at Least One Tafamidis Prescription	From incident ATTR-CM diagnosis (Index 1) until death, end of SHI enrollment, or end of data availability (December 31, 2023). For those with at least one tafamidis prescription, additionally from first tafamidis prescription (Index 2).
Cardiovascular-Related Hospitalizations in Individuals With Incident Transthyretin Amyloid Cardiomyopathy (ATTR-CM), Overall and in Those With at Least One Tafamidis Prescription	From incident ATTR-CM diagnosis (Index 1) until death, end of SHI enrollment, or end of data availability (December 31, 2023). For those with at least one tafamidis prescription, additionally from first tafamidis prescription (Index 2).

Secondary Outcome Measures

Outcome Measure	Time Frame
Annual Incidence of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in Germany	Calendar years 2020, 2021, and 2022

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): April 2, 2026
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: March 20, 2026
• First Submitted That Met QC Criteria: March 20, 2026
• First Posted (Actual): March 27, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 19, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: tafamidis
• Other Study ID Numbers: B3461130

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No