Amyloidosis, Immunoglobulin Light-chain
Conditions
Brief summary
This registry is a observational, multi-center study designed to collect data and analyze it retrospectively on patients with cardiac amyloidosis who have been evaluated and treated at major amyloid centers across the US and internationally between 1997 and 2025.
Detailed description
Amyloidosis is a rare, multisystem disorder in which an insoluble protein is deposited in tissues, leading to organ dysfunction. Several proteins have been identified to potentially lead to amyloid cardiomyopathy (AC). Given the rare nature of these diseases, a large, multi-center effort to describe the characteristics of these patients and their outcomes with novel treatment modalities has not been established. (TBD) academic medical centers from the US and internationally will compile demographic, hemodynamic and organ-involvement data, as well as treatment strategies for AL and TTR amyloidosis. This registry is an observational, multi-center study designed to collect data and analyze it retrospectively on patients with cardiac amyloidosis who have been evaluated and treated at major amyloid centers across the US and internationally between 1997 and 2025. It is expected that the total patient population will be approximately 5000 patients.
Interventions
OTHERRegistry
Observational, data collection
Sponsors
Cedars-Sinai Medical Center
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Established diagnosis of AL or TTR cardiomyopathy identified or treated within the timeframe * Specific to CSMC: If the individual provided consent while they were alive, and if sufficient information exists in their chart, their data will be included. * Information on deceased individuals may be included, but only with the appropriate approval from the external site IRB and/or according to the federal regulations for the protection of human subjects.
Exclusion criteria
* At Cedars-Sinai, records that specifically state not to be used in research will not be accessed. Patients who have enacted a No Research Flag or are noted as Break the Glass will not be included. For external sites, records that indicate No Research Flag or are noted as Break the Glass may be included based on institutional policies and appropriate approvals, as applicable.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Quantify disease severity at diagnosis, progression and survival in patients with cardiac amyloidosis | 1997 - 2025 | Clinical Outcomes: Disease severity at presentation, progression, and survival \[time frame 3 years\]. Severity and progression determined by change in NYHA Class, NT-ProBNP and troponin. |
| Quantify incidence of complications from cardiac amyloidosis | 1997 - 2025 | Determine incidence of arrhythmias (atrial fibrillation; ventricular arrhythmias) after diagnosis, renal dysfunction (rise in creatinine and development of end-stage renal disease), stroke, bleeding complications \[time frame 3 years\] |
Countries
Canada, United States
Outcome results
None listed