EHA Library - The official digital education library of European Hematology Association (EHA)

COLD AGGLUTININ DISEASE (CAD) REAL WORLD EVIDENCE (CADENCE) REGISTRY: DESIGN OF THE FIRST INTERNATIONAL, PROSPECTIVE CAD REGISTRY
Author(s): ,
Alexander Röth
Affiliations:
Department of Hematology,West German Cancer Center, University Hospital Essen, University of Duisburg-Essen,Essen,Germany
,
Ulrich Jäger
Affiliations:
Department of Internal Medicine I,Medical University of Vienna,Vienna,Austria
,
Bernd Jilma
Affiliations:
Department of Clinical Pharmacology,Medical University of Vienna,Vienna,Austria
,
Wilma Barcellini
Affiliations:
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico,Milan,Italy
,
Anita Hill
Affiliations:
Department of Haematology,St. James's University Hospital,Leeds,United Kingdom
,
Quentin Hill
Affiliations:
Department of Haematology,St. James's University Hospital,Leeds,United Kingdom
,
David J. Kuter
Affiliations:
Division of Hematology,Massachusetts General Hospital, Harvard Medical School,Boston, MA,United States
,
Catherine M. Broome
Affiliations:
Division of Hematology,MedStar Georgetown University Hospital,Washington, DC,United States
,
Marc Michel
Affiliations:
Department of Internal Medicine,National Referral Center for Adult Immune Cytopenias, Henri Mondor University Hospital, Assistance Publique Hôpitaux de Paris, Université Paris-Est Créteil,Créteil,France
,
Sacha Sergio Zeerleder
Affiliations:
Department of Hematology and Central Hematology Laboratory (UKHHZL),Inselspital, Bern University Hospital,Bern,Switzerland
,
Yasutaka Ueda
Affiliations:
Department of Hematology and Oncology,Graduate School of Medicine, Osaka University,Osaka,Japan
,
Jaime Morales Arias
Affiliations:
Sanofi,Cambridge, MA,United States
,
Jun Su
Affiliations:
Sanofi,Cambridge, MA,United States
Sigbjørn Berentsen
Affiliations:
Department of Research and Innovation,Haugesund Hospital,Haugesund,Norway
(Abstract release date: 05/14/20) EHA Library. Röth A. 06/12/20; 294102; EP1618
Alexander Röth
Alexander Röth
Contributions
Abstract

Abstract: EP1618

Type: e-Poster

Background
Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia in which hemolysis is mediated via the classical complement pathway. Its estimated incidence rate is 1 per million persons per year, with a prevalence of 16 cases per million persons. Patients with CAD can experience debilitating fatigue and poor quality of life (QoL), and they may have an increased risk of thromboembolism and early mortality. Owing to the rarity of the disease, information regarding the natural history of this condition and its long-term impact and complications is sparse (Jäger U, et al. Blood Rev. 2019).

Aims
To describe the rationale and design of a large, international database of patients with CAD.

Methods
CADENCE is a multinational, multicenter, observational, prospective, longitudinal registry. Patients aged ≥18 years with primary CAD, secondary CAD/cold agglutinin syndrome, and mixed warm and cold autoimmune hemolytic anemia will be included. The main objective of this registry is to better understand various aspects of CAD, including patient and clinical characteristics, patterns of CAD treatment use, healthcare resource utilization (HRU), natural history of disease, long-term clinical outcomes, and impact on patient QoL. The enrollment period is expected to last ≥2 years, and patients will be followed for 3 years after enrollment. Data will be collected and analyzed at regional and global levels; sites will complete electronic case report forms, and patients will complete patient-reported outcome (PRO) questionnaires (36-Item Short Form Health Survey [version 2] and Functional Assessment of Chronic Illness Therapy – Fatigue scale) at enrollment and at intervals of about every 6 months during follow-up. 

Results
The CADENCE Registry was launched in December 2019, with last patient last visit expected at the end of 2024. It is expected to enroll approximately 725 patients with CAD across 121 sites in multiple countries, including the United States, Canada, France, United Kingdom, Italy, Germany, Austria, Norway, Japan, Australia, and Spain (Figure). Data collected will include clinical characteristics, disease complications (eg, thromboembolism), comorbidities, mortality, treatment regimens, HRU, and PROs. Relevant biomarkers will be assessed. Interim analyses will be performed after 100 patients, 250 patients, and 500 patients are enrolled in the study. Final analyses will be performed at the end of the study both at regional and global levels.

Conclusion
As the first ever global registry for patients with CAD, CADENCE will help advance the understanding of the natural history and disease process of this ultra-rare disease in which the ability to conduct prospective trials with sufficient numbers of patients is challenging. This registry will provide clinicians with a data resource that may help improve treatment practices for patients with CAD.

Session topic: 31. Transfusion medicine

Keyword(s): Autoimmune hemolytic anemia (AIHA), Complement, Population, Thromboembolic events

Abstract: EP1618

Type: e-Poster

Background
Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia in which hemolysis is mediated via the classical complement pathway. Its estimated incidence rate is 1 per million persons per year, with a prevalence of 16 cases per million persons. Patients with CAD can experience debilitating fatigue and poor quality of life (QoL), and they may have an increased risk of thromboembolism and early mortality. Owing to the rarity of the disease, information regarding the natural history of this condition and its long-term impact and complications is sparse (Jäger U, et al. Blood Rev. 2019).

Aims
To describe the rationale and design of a large, international database of patients with CAD.

Methods
CADENCE is a multinational, multicenter, observational, prospective, longitudinal registry. Patients aged ≥18 years with primary CAD, secondary CAD/cold agglutinin syndrome, and mixed warm and cold autoimmune hemolytic anemia will be included. The main objective of this registry is to better understand various aspects of CAD, including patient and clinical characteristics, patterns of CAD treatment use, healthcare resource utilization (HRU), natural history of disease, long-term clinical outcomes, and impact on patient QoL. The enrollment period is expected to last ≥2 years, and patients will be followed for 3 years after enrollment. Data will be collected and analyzed at regional and global levels; sites will complete electronic case report forms, and patients will complete patient-reported outcome (PRO) questionnaires (36-Item Short Form Health Survey [version 2] and Functional Assessment of Chronic Illness Therapy – Fatigue scale) at enrollment and at intervals of about every 6 months during follow-up. 

Results
The CADENCE Registry was launched in December 2019, with last patient last visit expected at the end of 2024. It is expected to enroll approximately 725 patients with CAD across 121 sites in multiple countries, including the United States, Canada, France, United Kingdom, Italy, Germany, Austria, Norway, Japan, Australia, and Spain (Figure). Data collected will include clinical characteristics, disease complications (eg, thromboembolism), comorbidities, mortality, treatment regimens, HRU, and PROs. Relevant biomarkers will be assessed. Interim analyses will be performed after 100 patients, 250 patients, and 500 patients are enrolled in the study. Final analyses will be performed at the end of the study both at regional and global levels.

Conclusion
As the first ever global registry for patients with CAD, CADENCE will help advance the understanding of the natural history and disease process of this ultra-rare disease in which the ability to conduct prospective trials with sufficient numbers of patients is challenging. This registry will provide clinicians with a data resource that may help improve treatment practices for patients with CAD.

Session topic: 31. Transfusion medicine

Keyword(s): Autoimmune hemolytic anemia (AIHA), Complement, Population, Thromboembolic events

By clicking “Accept Terms & all Cookies” or by continuing to browse, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies