Constitutional dyserythropoietic anemia

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Overview

Constitutional dyserythropoietic anemia (CDA) is a group of rare inherited blood disorders that affect the way red blood cells are produced in the bone marrow. In healthy people, the bone marrow makes red blood cells through a well-organized process. In CDA, this process goes wrong, leading to abnormal red blood cell development (called dyserythropoiesis). This means the body cannot make enough healthy red blood cells, resulting in anemia — a condition where the blood cannot carry enough oxygen to the body's tissues. The main symptoms of CDA include fatigue, pale skin, weakness, and sometimes jaundice (yellowing of the skin and eyes). Because the body breaks down the abnormal red blood cells faster than normal, iron can build up in the body over time, which can damage organs like the heart and liver. Some patients may also develop an enlarged spleen. The severity of CDA varies widely — some people have mild anemia that barely affects daily life, while others need regular blood transfusions. There are several types of CDA (Type I, II, III, and others), each caused by different genetic changes. Treatment depends on the type and severity and may include blood transfusions, iron chelation therapy to remove excess iron, splenectomy (removal of the spleen) in certain cases, and supportive care. In severe cases, bone marrow transplantation may be considered. Research is ongoing to better understand these conditions and develop more targeted treatments.

Key symptoms:

Fatigue and tirednessPale skinWeaknessJaundice (yellowing of skin and eyes)Enlarged spleenEnlarged liverIron overload in the bodyDark-colored urineGallstonesShortness of breath during activitySlow growth in childrenBone changes (in severe cases)Heart problems from iron buildupLeg ulcers (in some types)

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Constitutional dyserythropoietic anemia.

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No actively recruiting trials found for Constitutional dyserythropoietic anemia at this time.

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Search ClinicalTrials.gov ↗Join the Constitutional dyserythropoietic anemia community →

No specialists are currently listed for Constitutional dyserythropoietic anemia.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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

Travel Grants

No travel grants are currently matched to Constitutional dyserythropoietic anemia.

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Community

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

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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.What specific type of CDA do I (or my child) have, and what gene is involved?,How severe is the anemia, and will blood transfusions be needed?,What is my current iron level, and do I need iron chelation therapy?,Should I consider genetic counseling for family planning purposes?,Are there any clinical trials or new treatments available for my type of CDA?,How often should I have blood tests and organ function monitoring?,Is bone marrow transplantation an option in my case, and what are the risks and benefits?

Common questions about Constitutional dyserythropoietic anemia

What is Constitutional dyserythropoietic anemia?

Constitutional dyserythropoietic anemia (CDA) is a group of rare inherited blood disorders that affect the way red blood cells are produced in the bone marrow. In healthy people, the bone marrow makes red blood cells through a well-organized process. In CDA, this process goes wrong, leading to abnormal red blood cell development (called dyserythropoiesis). This means the body cannot make enough healthy red blood cells, resulting in anemia — a condition where the blood cannot carry enough oxygen to the body's tissues. The main symptoms of CDA include fatigue, pale skin, weakness, and sometimes