Congenital secondary polycythemia

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ORPHA:238536
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16Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Congenital secondary polycythemia is a rare blood condition that is present from birth. In this disease, the body makes too many red blood cells, but unlike primary polycythemia (where the problem starts in the bone marrow itself), secondary polycythemia happens because something outside the bone marrow signals it to overproduce red blood cells. In the congenital form, this is caused by inherited genetic changes that affect how the body senses or responds to oxygen levels. The most common mechanism involves mutations that increase the production of erythropoietin (EPO), a hormone made mainly by the kidneys that tells the bone marrow to make more red blood cells, or mutations that make the body act as though oxygen levels are low even when they are normal. Key symptoms include a ruddy or reddish complexion, headaches, dizziness, fatigue, and an increased risk of blood clots due to thickened blood. Some patients may experience high blood pressure, blurred vision, or itching. The severity can vary widely depending on the specific genetic cause. Treatment typically focuses on reducing the number of red blood cells through periodic blood removal (phlebotomy) to lower the risk of dangerous clots. In some cases, low-dose aspirin may be recommended. Identifying the exact genetic cause is important because it can guide treatment decisions and help predict the disease course. This condition is also sometimes referred to as congenital secondary erythrocytosis.

Also known as:

Key symptoms:

Reddish or ruddy skin color, especially on the faceHeadachesDizziness or lightheadednessFatigue or tirednessBlurred or double visionItching, especially after a warm bathHigh blood pressureBlood clots in veins or arteriesShortness of breathTingling or numbness in hands or feetNosebleedsFeeling of fullness in the headStroke or mini-stroke symptoms in severe cases

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Congenital secondary polycythemia.

View clinical trials →

No actively recruiting trials found for Congenital secondary polycythemia at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Congenital secondary polycythemia community →

Specialists

16 foundView all specialists →
NM
Neal Flomenberg, MD
PHILADELPHIA, PA
Specialist
PI on 5 active trials
DC
Dolores Grosso, DNP, CRNP
Specialist
PI on 4 active trials
RN
Ryotaro Nakamura
Duarte, California
Specialist

Rare Disease Specialist

PI on 6 active trials
JM
Jeffrey J. Raizer, MD
CHICAGO, IL
Specialist
PI on 7 active trials
MB
Merav Bar
SEATTLE, WA
Specialist
PI on 1 active trial2 Congenital secondary polycythemia publications
SP
Suzanne C. Danhauer, PhD
WINSTON SALEM, NC
Specialist
PI on 5 active trials
AW
Ann Woolfrey
SEATTLE, WA
Specialist
PI on 5 active trials
TD
Tamila Kindwall-Keller, DO
Specialist
PI on 1 active trial19 Congenital secondary polycythemia publications
PM
Paul Armistead, MD
CHAPEL HILL, NC
Specialist
PI on 1 active trial

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 Congenital secondary polycythemia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Congenital secondary polycythemia

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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 genetic mutation is causing my polycythemia, and how does that affect my treatment plan?,How often will I need phlebotomy, and what is my target hematocrit level?,What are the warning signs of a blood clot that I should watch for?,Should I take low-dose aspirin or any other medication to prevent clots?,Are there any activities, environments, or medications I should avoid?,Should my family members be tested for this condition?,Are there any clinical trials or new treatments being studied for my condition?

Common questions about Congenital secondary polycythemia

What is Congenital secondary polycythemia?

Congenital secondary polycythemia is a rare blood condition that is present from birth. In this disease, the body makes too many red blood cells, but unlike primary polycythemia (where the problem starts in the bone marrow itself), secondary polycythemia happens because something outside the bone marrow signals it to overproduce red blood cells. In the congenital form, this is caused by inherited genetic changes that affect how the body senses or responds to oxygen levels. The most common mechanism involves mutations that increase the production of erythropoietin (EPO), a hormone made mainly b

At what age does Congenital secondary polycythemia typically begin?

Typical onset of Congenital secondary polycythemia is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Congenital secondary polycythemia?

16 specialists and care centers treating Congenital secondary polycythemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.