Cold Agglutinin Disease Real World Evidence Registry

Study Overview
=================
Brief Summary
-----------------
This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 [NCT02502903,CAD patients], BIVV009-03/EFC16215 [NCT03347396], and BIVV009-04/EFC16216 [NCT03347422]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.

Official Title
-----------------
Cold Agglutinin Disease Real World Evidence Registry

Conditions
-----------------
Cold Agglutinin Disease (CAD), Cold Agglutinin Syndrome (CAS)

Intervention / Treatment
-----------------
* Drug: Sutimlimab


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patient aged ≥18 years Patient able to understand the purpose of the study and who (or whose legally authorized representative) provided signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations Patient with a diagnosis of CAD or CAS as per Investigator judgment based on the diagnosis criteria listed in study protocol Exclusion Criteria: Patient with mixed warm and cold autoimmune hemolytic anemia, or warm autoimmune hemolytic anemia Patient actively participating in a CAD or CAS interventional clinical trial. After a patient completes participation in said trial, he/she may be eligible for enrollment in this registry.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| cold agglutinin disease (CAD)<br>Patient with a diagnosis of CAD as per investigator judgment based on the diagnosis criteria listed in study protocol. In addition, this group includes a cohort of CAD patients treated with sutimlimab | Drug: Sutimlimab<br>* CAD patients in Sutimlimab cohort must be treated according to routine clinical practice<br>|
| cold agglutinin syndrome (CAS)<br>Patient with a diagnosis of CAS as per investigator judgment based on the diagnosis criteria listed in study protocol | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patterns of CAD and CAS disease characteristics | CAD or CAS characteristics at diagnosis, enrollment, and during follow up (e.g., criteria used for diagnosis, symptoms, laboratory results) will be described. | Up to 6 years | 
| Number of participants with CAD or CAS complications | Thromboembolic events (venous, arterial or cerebral), deaths related to CAD or CAS, other CAD/CAS complications (e.g., acute hemolytic crisis, hemolysis-related complications), and other comorbid conditions occurring before enrollment, at enrollment, and during follow up will be described. | Up to 6 years | 
| Patterns of use of CAD and CAS treatments | CAD or CAS treatments received before enrollment, at enrollment, and during the follow up period will be summarized. CAD or CAS symptoms and complications, autoimmune disorders or lymphoproliferative disorders, physician assessment of treatment status, and changes in laboratory results will be described by CAD or CAS treatments. | Up to 6 years | 
| Number of participants with health-resource utilization | Health resource utilization may include hospitalizations (including length of stay), outpatient visits, emergency room visits, specific transfusion visits, nursing home/rehabilitation center stays, and home healthcare visits during registry follow-up period | Up to 6 years | 
| Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ) |  | Up to 6 years | 
| Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) |  | Up to 6 years | 
| 36-Item Short Form Survey Instrument (SF-36) v2 |  | Up to 6 years | 
| Sutimlimab cohort: Sutimlimab treatment patterns | Sutimlimab dosing information, reasons for dose adjustments, reasons for discontinuation will be described. | Up to 6 years | 
| Sutimlimab cohort: Number of participants with adverse events, serious adverse events, adverse events of special interests (AESIs), other events of interests (e.g., Treatment emergent) |  | Up to 6 years | 
| Sutimlimab cohort: change from baseline in hemoglobin |  | From baseline up to 6 years | 
| Sutimlimab cohort: change from baseline in bilirubin |  | From baseline up to 6 years | 
| Sutimlimab cohort: change from baseline in Lactate dehydrogenase (LDH) |  | From baseline up to 6 years | 
| Sutimlimab cohort: number of blood cells transfusions |  | Up to 6 years |