Genetic complex vascular malformation with associated anomalies

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6Specialists8Treatment centers

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Overview

Genetic complex vascular malformation with associated anomalies is a rare condition in which a person is born with abnormal blood vessels — including veins, arteries, capillaries, or lymphatic vessels — that develop incorrectly during early growth in the womb. Unlike simple vascular malformations that involve only one type of vessel, complex vascular malformations involve a combination of vessel types and are accompanied by other body abnormalities (anomalies). These associated anomalies can affect the bones, soft tissues, skin, and internal organs, and may include overgrowth of a limb, asymmetric body growth, skin discoloration (such as port-wine stains or other birthmarks), and problems with blood clotting. Symptoms vary widely depending on which blood vessels are affected and where in the body the malformations occur. Some people have mild symptoms, while others face serious complications such as chronic pain, bleeding, infections, or organ dysfunction. The malformations are present at birth but may become more noticeable as a child grows. Because these conditions are caused by genetic changes, they are not the result of anything that happened during pregnancy. Treatment is tailored to each individual and is usually managed by a multidisciplinary team. Options may include compression garments, sclerotherapy (injecting a substance to shrink abnormal vessels), laser therapy, surgery, and medications such as sirolimus that target the pathways involved in abnormal vessel growth. There is currently no cure, and management focuses on controlling symptoms, preventing complications, and improving quality of life.

Also known as:

Key symptoms:

Visible birthmarks or skin discoloration such as port-wine stainsSwelling or enlargement of a limb or body partAsymmetric body growth (one side larger than the other)Chronic pain in the affected areaWarm or tender lumps under the skinBleeding from abnormal blood vesselsBlood clotting problemsSkin ulcers or wounds that heal slowlyBone overgrowth or undergrowthSoft tissue massesLymphatic fluid leakage or swelling (lymphedema)Recurrent infections in affected areasFatigue due to chronic blood loss or painDifficulty moving the affected limb

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 Genetic complex vascular malformation with associated anomalies.

View clinical trials →

No actively recruiting trials found for Genetic complex vascular malformation with associated anomalies at this time.

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Specialists

6 foundView all specialists →
PM
Peter A Merkel, MD, MPH
Tampa, Florida
Specialist

Rare Disease Specialist

PI on 7 active trials
PM
Paul Martin
Specialist
PI on 3 active trials73 Genetic complex vascular malformation with associated anomalies publications
SM
Shivaani Kummar, MD
PORTLAND, OR
Specialist
PI on 5 active trials

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 Genetic complex vascular malformation with associated anomalies.

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Community

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Latest news about Genetic complex vascular malformation with associated anomalies

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Caregiver Resources

NORD Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What type of vascular malformation does my child have, and which blood vessels are involved?,Has genetic testing been done, and if so, what mutation was found and what does it mean for treatment?,What are the treatment options, and what are the risks and benefits of each?,Is my child a candidate for targeted therapy such as sirolimus or alpelisib?,How often will imaging and follow-up visits be needed?,What warning signs should I watch for that would require emergency care?,Are there any clinical trials available for this condition?

Common questions about Genetic complex vascular malformation with associated anomalies

What is Genetic complex vascular malformation with associated anomalies?

Genetic complex vascular malformation with associated anomalies is a rare condition in which a person is born with abnormal blood vessels — including veins, arteries, capillaries, or lymphatic vessels — that develop incorrectly during early growth in the womb. Unlike simple vascular malformations that involve only one type of vessel, complex vascular malformations involve a combination of vessel types and are accompanied by other body abnormalities (anomalies). These associated anomalies can affect the bones, soft tissues, skin, and internal organs, and may include overgrowth of a limb, asymme

At what age does Genetic complex vascular malformation with associated anomalies typically begin?

Typical onset of Genetic complex vascular malformation with associated anomalies is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Genetic complex vascular malformation with associated anomalies?

6 specialists and care centers treating Genetic complex vascular malformation with associated anomalies are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.