Rare combined vascular malformation

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ORPHA:458837
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1Active trials8Treatment centers

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Overview

Rare combined vascular malformation is an uncommon condition in which a person is born with two or more types of blood vessel abnormalities occurring together. Vascular malformations are errors in the way blood vessels form during development in the womb. They can involve veins (venous malformations), arteries (arterial malformations), lymph vessels (lymphatic malformations), or tiny capillaries (capillary malformations). When two or more of these types appear together in the same person, the condition is called a combined vascular malformation. These malformations are present at birth, though they may not always be noticed right away. They tend to grow slowly along with the child and do not go away on their own. Symptoms depend on which types of vessels are involved and where in the body they are located. Common problems include visible skin discoloration (such as red, blue, or purple patches), swelling, pain, and sometimes overgrowth of a limb or body part. In some cases, the malformations can affect deeper tissues and organs, leading to bleeding, infection, or problems with blood clotting. Treatment is tailored to each person and may include compression garments, sclerotherapy (injecting a substance to shrink abnormal vessels), laser therapy, or surgery. A team of specialists usually works together to manage the condition. While there is no single cure, many symptoms can be controlled with ongoing care. Research into the genetic causes of these malformations is helping to develop newer targeted therapies.

Key symptoms:

Red, blue, or purple skin patches present from birthSwelling of affected body areasPain or tenderness in the area of the malformationOvergrowth of a limb or body partWarm skin over the affected areaVisible enlarged veins under the skinBleeding from the malformationSkin blisters that may leak clear fluidRecurrent infections in the affected areaBlood clotting problemsDifficulty moving the affected limbDisfigurement or cosmetic concernsFatigue due to chronic pain or blood loss

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 ↗

FDA & Trial Timeline

1 event
Apr 2015Identification of Biomarkers for Patients with Vascular Anomalies

Children's Hospital Medical Center, Cincinnati

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Rare combined vascular malformation.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Identification of Biomarkers for Patients with Vascular Anomalies
Actively Recruiting
PI: Timothy LeCras, PhD (Children's Hospital Medical Center, Cincinnati) · Sites: Boston, Massachusetts; Cincinnati, Ohio

No specialists are currently listed for Rare combined vascular malformation.

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 Rare combined vascular malformation.

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Community

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Caregiver Resources

NORD Caregiver Resources

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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 types of vascular malformations does my child (or do I) have, and where exactly are they located?,Would genetic testing help guide treatment decisions, and if so, what genes should be tested?,What treatment options are available, and what are the risks and benefits of each?,Is a targeted drug therapy like sirolimus or alpelisib appropriate in this case?,How often will imaging and follow-up visits be needed?,Are there any activities or situations I should avoid to prevent complications?,Is there a specialized vascular anomalies center you would recommend for ongoing care?

Common questions about Rare combined vascular malformation

What is Rare combined vascular malformation?

Rare combined vascular malformation is an uncommon condition in which a person is born with two or more types of blood vessel abnormalities occurring together. Vascular malformations are errors in the way blood vessels form during development in the womb. They can involve veins (venous malformations), arteries (arterial malformations), lymph vessels (lymphatic malformations), or tiny capillaries (capillary malformations). When two or more of these types appear together in the same person, the condition is called a combined vascular malformation. These malformations are present at birth, though

At what age does Rare combined vascular malformation typically begin?

Typical onset of Rare combined vascular malformation is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Rare combined vascular malformation?

Yes — 1 recruiting clinical trial is currently listed for Rare combined vascular malformation on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.