Blue rubber bleb nevus

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ORPHA:1059OMIM:112200Q27.8
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2Active trials9Specialists8Treatment centers1Financial resources

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

Blue rubber bleb nevus syndrome (BRBNS), also known as Bean syndrome, is a rare vascular disorder characterized by multiple venous malformations that primarily affect the skin and gastrointestinal (GI) tract, though they can occur in virtually any organ. The condition is named for the distinctive blue, soft, compressible, rubbery skin lesions that can range in size from a few millimeters to several centimeters. These lesions are present at birth or appear during early childhood and tend to increase in number and size over time. The gastrointestinal venous malformations are the most clinically significant feature, as they are prone to chronic bleeding that can lead to iron-deficiency anemia, which may be severe and require blood transfusions. The small intestine is most commonly affected, but lesions can occur throughout the entire GI tract. Venous malformations may also involve the liver, spleen, central nervous system, musculoskeletal system, lungs, and other organs. Skin lesions may be painful, particularly with changes in temperature or pressure, and can cause localized sweating. Some patients experience thrombosis within the malformations, which can lead to pain and, in rare cases, consumptive coagulopathy. Treatment of BRBNS is primarily supportive and symptom-directed. Iron supplementation and blood transfusions are frequently needed for chronic anemia. Endoscopic interventions, including sclerotherapy and laser photocoagulation, can be used to manage GI bleeding. Surgical resection of affected bowel segments may be necessary in severe cases. Pharmacological agents such as sirolimus (an mTOR inhibitor) have shown promising results in reducing the size and number of lesions and controlling GI bleeding in some patients. The condition is associated with somatic mutations in the TEK gene (encoding the TIE2 receptor), which plays a role in vascular development.

Also known as:

Clinical phenotype terms— hover any for plain English:

Cavernous hemangiomaHP:0001048Microcytic anemiaHP:0001935VolvulusHP:0002580Intestinal bleedingHP:0002584Abnormality of the vasculatureHP:0002597Prolonged bleeding timeHP:0003010Gastrointestinal infarctionsHP:0005244Arteriovenous malformationHP:0100026Visceral angiomatosisHP:0100761
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

3 events
Sep 2024Safety of the Sonablate HIFU System for the Ablation of Incompetent Veins of the Periphery

Sonablate — NA

TrialACTIVE NOT RECRUITING
Apr 2024Metabolic Availability of Methionine From Mung Beans in Healthy Adults

The Hospital for Sick Children — NA

TrialACTIVE NOT RECRUITING
Jun 2013Lymphatic Anomalies Registry for the Assessment of Outcome Data

Boston Children's Hospital

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Blue rubber bleb nevus.

2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

2 recruitingView all trials with filters →
N/A1 trial
Safety of the Sonablate HIFU System for the Ablation of Incompetent Veins of the Periphery
N/A
Active
PI: Naiem Nassiri, MD, FSVS, RPVI (The Vascular Care Group) · Sites: Darien, Connecticut · Age: 1899 yrs
Other1 trial
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Actively Recruiting
PI: Melisa Ruiz-Gutierrez, M.D. (Boston Children's Hospital) · Sites: Boston, Massachusetts

Specialists

9 foundView all specialists →
EP
Emily M Garland, PhD
ATLANTA, GA
Specialist
PI on 3 active trials
NR
Naiem Nassiri, MD, FSVS, RPVI
DARIEN, CT
Specialist
PI on 1 active trial
MM
Melisa Ruiz-Gutierrez, M.D.
BOSTON, MA
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

Financial Resources

1 resources

SULFAMYLON

Rising Pharma Holdings, Inc.

Burn

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copay card
Copay CardPatient Assistance
Accepting applications

Travel Grants

No travel grants are currently matched to Blue rubber bleb nevus.

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Community

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Latest news about Blue rubber bleb nevus

Disease timeline:

New recruiting trial: Lymphatic Anomalies Registry for the Assessment of Outcome Data

A new clinical trial is recruiting patients for Blue rubber bleb nevus

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.

Common questions about Blue rubber bleb nevus

What is Blue rubber bleb nevus?

Blue rubber bleb nevus syndrome (BRBNS), also known as Bean syndrome, is a rare vascular disorder characterized by multiple venous malformations that primarily affect the skin and gastrointestinal (GI) tract, though they can occur in virtually any organ. The condition is named for the distinctive blue, soft, compressible, rubbery skin lesions that can range in size from a few millimeters to several centimeters. These lesions are present at birth or appear during early childhood and tend to increase in number and size over time. The gastrointestinal venous malformations are the most clinically

How is Blue rubber bleb nevus inherited?

Blue rubber bleb nevus follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Blue rubber bleb nevus?

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

Which specialists treat Blue rubber bleb nevus?

9 specialists and care centers treating Blue rubber bleb nevus are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Blue rubber bleb nevus?

1 patient support program are currently tracked on UniteRare for Blue rubber bleb nevus. See the treatments and support programs sections for copay assistance, eligibility, and contact details.