Cold agglutinin disease

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1FDA treatments2Active trials36Specialists8Treatment centers1Financial resources

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Overview

Cold agglutinin disease (CAD), sometimes called cold agglutinin syndrome or cold antibody hemolytic anemia, is a rare blood disorder where the immune system mistakenly attacks red blood cells. Normally, your immune system protects you from germs. In CAD, it produces special proteins called cold agglutinins — antibodies that become active in cooler temperatures. When parts of your body get cold (like your hands, feet, or even your blood as it circulates near the skin), these antibodies stick to red blood cells and signal the body to destroy them. This process is called hemolysis, and it leads to a shortage of red blood cells, known as anemia. The main symptoms of CAD come from this anemia and from reduced blood flow in cold conditions. People often feel very tired, look pale or yellowish (jaundiced), and notice that their fingers, toes, nose, or ears turn blue or purple in the cold — a condition called acrocyanosis or Raynaud's phenomenon. Some people also have dark-colored urine, especially after cold exposure. CAD can be primary (no known underlying cause, often linked to a small abnormal clone of blood cells in the bone marrow) or secondary (triggered by an infection, another blood cancer, or an autoimmune disease). Treatment has improved significantly in recent years. The drug sutimlimab (Enjaymo) was approved by the FDA in 2022 specifically for CAD, making it the first targeted therapy for this condition. Other treatments include rituximab and bendamustine. Keeping warm is also a key part of daily management.

Also known as:

Key symptoms:

Extreme tiredness and low energy (fatigue from anemia)Pale or yellowish skin (pallor or jaundice)Fingers, toes, nose, or ears turning blue or purple in the cold (acrocyanosis)Dark or tea-colored urine, especially after cold exposureShortness of breath, especially with activityRapid or irregular heartbeatDizziness or lightheadednessFeeling cold all the timeSwollen lymph nodes (in some cases)Abdominal discomfort or enlarged spleen

Clinical phenotype terms (14)— hover any for plain English
Abnormal urinary colorHP:0012086
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Feb 2027A Prospective Evaluation of Clinical Outcomes in Acute Ischemic Stroke After Endovascular Treatment w/Doppler

Virginia Commonwealth University — NA

TrialNOT YET RECRUITING
Sep 2026CTCA Prior to Invasive Angiography in Post-Bypass Patients (BYPASS CTCA 2)

Queen Mary University of London — NA

TrialNOT YET RECRUITING
Aug 2026Drug-coatEd Balloon Multicentric Registry in Spain and PORTugal

Portuguese Association of Interventional Cardiology

TrialNOT YET RECRUITING
May 2026Role of Omega-DEK in Childhood Apraxia of Speech

Claudia R. Morris — PHASE2

TrialNOT YET RECRUITING
Apr 2026Autus Valve Continued Access Study (CAS)

Autus Valve Technologies, Inc. — NA

TrialNOT YET RECRUITING
Apr 2026Guideline Adherence in Dyslipidemia With Clinical Decision Support

I.M. Sechenov First Moscow State Medical University

TrialNOT YET RECRUITING
Apr 2026Multivessel Minimally Invasive Coronary Bypass Grafting as HYBRID Revascularization Versus Conventional Off-pump Coronary Artery Bypass Grafting

Catharina Ziekenhuis Eindhoven — NA

TrialNOT YET RECRUITING
Apr 2026Effect of Epilepsy and Antiepileptic Drug Therapy on Gastric Motility and Emptying With Point-Of-Care Gastric Ultrasound

London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

TrialNOT YET RECRUITING
Mar 2026The NAVIGATE-PCI Registry: A New Approach to VIew CT-derived Guidance Ahead of Stenting To Plan Efficient PCI

HeartFlow, Inc.

TrialRECRUITING
Mar 2026Intraoral vs Extraoral Cementation of Zirconia Bridges on Ti-Bases: Microstrain Evaluation

Universitat Internacional de Catalunya — NA

TrialNOT YET RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Enjaymo

SUTIMLIMAB-JOME· Recordati Rare Diseases, Inc.

indicated for the treatment of hemolysis in adults with cold agglutinin disease (CAD)

Clinical Trials

2 recruitingView all trials with filters →
Phase 21 trial
Zanubrutinib in Primary Cold Agglutinin Disease
Phase 2
Active
· Sites: Leuven; Roeselare +8 more · Age: 1899 yrs
Phase 11 trial
Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects With Cold Agglutinin Disease
Phase 1
Actively Recruiting
PI: Jim Bush, MBChB, PhD (Fortrea Clinical Research Unit Ltd.) · Sites: Leeds · Age: 1899 yrs

Specialists

Showing 25 of 36View all specialists →
RM
Ronald G Crystal, MD
NEW YORK, NY
Specialist
PI on 12 active trials
HP
Henrik Birgens, MD, PhD
Specialist
PI on 1 active trial
JP
Jim Bush, MBChB, PhD
Specialist
PI on 2 active trials
MM
Markku Oksman, MD
Specialist
PI on 1 active trial
AM
Alexander Roeth, MD
Specialist
PI on 1 active trial1 Cold agglutinin disease publication
GD
Giuseppe Rossi, Dr.
Specialist
PI on 1 active trial
GP
Geir E Tjonnfjord, MD, PhD
Specialist
PI on 1 active trial
IR
Ignacio Alvarez Rojo
Specialist
PI on 1 active trial
LS
Laurent Sailler
Specialist
PI on 1 active trial
MM
Mary Ann McLaughlin, MD, MPH
Specialist
PI on 2 active trials
SA
Sameh Abdelrahman
Specialist
PI on 1 active trial
SB
Sigbjørn Berentsen
Specialist
12 Cold agglutinin disease publications
AR
Alexander Röth
Specialist
7 Cold agglutinin disease publications
SD
Shirley D'Sa
Specialist
7 Cold agglutinin disease publications
JV
Josephine M I Vos
Specialist
6 Cold agglutinin disease publications
CB
Catherine M Broome
Specialist
6 Cold agglutinin disease publications
MY
Masaki Yamaguchi
Specialist
5 Cold agglutinin disease publications
IW
Ilene C Weitz
LOS ANGELES, CA
Specialist
4 Cold agglutinin disease publications
MW
Marek Wardęcki
Specialist
4 Cold agglutinin disease publications
FS
Frank Shafer
PHILADELPHIA, PA
Specialist
4 Cold agglutinin disease publications
ML
Michelle Lee
Specialist
4 Cold agglutinin disease publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
Enjaymo(SUTIMLIMAB-JOME)Recordati Rare Diseases, Inc.

Travel Grants

No travel grants are currently matched to Cold agglutinin disease.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Cold agglutinin disease

1 articles
NewsRSSApr 22, 2026
Could My CAD Enter Remission?
Cold agglutinin disease (CAD) is a rare blood disorder where the body attacks its own red blood cells in cold temperatures. This article discusses whether CAD c
See all news about Cold agglutinin disease

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.Is my CAD primary or secondary, and does that change my treatment options?,Am I a candidate for sutimlimab (Enjaymo), and how would I receive it?,How cold is too cold for me, and what specific precautions should I take in daily life?,What signs should prompt me to go to the emergency room?,How often will I need blood tests to monitor my condition?,Are there any clinical trials I should consider?,Should I avoid any vaccines, medications, or procedures that could trigger a flare?

Common questions about Cold agglutinin disease

What is Cold agglutinin disease?

Cold agglutinin disease (CAD), sometimes called cold agglutinin syndrome or cold antibody hemolytic anemia, is a rare blood disorder where the immune system mistakenly attacks red blood cells. Normally, your immune system protects you from germs. In CAD, it produces special proteins called cold agglutinins — antibodies that become active in cooler temperatures. When parts of your body get cold (like your hands, feet, or even your blood as it circulates near the skin), these antibodies stick to red blood cells and signal the body to destroy them. This process is called hemolysis, and it leads t

How is Cold agglutinin disease inherited?

Cold agglutinin disease follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Cold agglutinin disease typically begin?

Typical onset of Cold agglutinin disease is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Cold agglutinin disease?

Yes — 2 recruiting clinical trials are currently listed for Cold agglutinin disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Cold agglutinin disease?

25 specialists and care centers treating Cold agglutinin disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.