🧬 Genetic Disorder

Blue Rubber Bleb Nevus Syndrome

Also known as: Bean Syndrome, BRBNS

Blue Rubber Bleb Nevus Syndrome (BRBNS) is a rare disorder characterized by the presence of multiple, distinctive, bluish-purple, rubbery, cutaneous (skin) lesions known as blebs, along with vascular malformations that can affect various organs, most commonly the gastrointestinal tract. These lesions can cause pain, bleeding, and anemia. The severity and location of the lesions vary greatly among affected individuals.

👥 <1 / 1 000 000 Prevalence
🔬 3 Active Trials

Imagine you have little blue or purple bumps under your skin, like rubbery blueberries. Sometimes, these bumps can bleed inside your tummy and make you feel tired. Doctors can help by giving you medicine or sometimes removing the bumps.

Signs & Symptoms

  • Bluish-purple rubbery skin lesions (blebs)
  • Gastrointestinal bleeding
  • Iron deficiency anemia
  • Abdominal pain
  • Visceral lesions (e.g., in the small intestine, liver, or central nervous system)
  • Deformed extremities
  • Thrombocytopenia
  • Intussusception
  • Protein losing enteropathy

Treatment Options

MEDICATION FDA Approved

Iron supplementation

SUPPORTIVE
SUPPORTIVE FDA Approved

Blood transfusions

SUPPORTIVE
SURGERY

Endoscopic removal or ablation of lesions

MODERATELY EFFECTIVE
SURGERY

Surgical resection of affected bowel segments

MODERATELY EFFECTIVE
MEDICATION FDA Approved

Sirolimus (Rapamycin)

MODERATELY EFFECTIVE Approved 2009
MEDICATION FDA Approved

Octreotide

MODERATELY EFFECTIVE Approved 1988
MEDICATION FDA Approved

Propranolol

MODERATELY EFFECTIVE Approved 1976

Diagnosis

  • Clinical examination
  • Skin biopsy
  • Endoscopy (colonoscopy, esophagogastroduodenoscopy)
  • Capsule endoscopy
  • Angiography
  • Magnetic Resonance Imaging (MRI)
  • Genetic testing (in familial cases)

History

The first detailed description of Blue Rubber Bleb Nevus Syndrome was provided by William Bean in 1958, who characterized the distinctive cutaneous and gastrointestinal manifestations of the disease.

Recent Breakthroughs

2022

Sirolimus Shows Promise in Reducing Bleeding in BRBNS Patients

A study published in the Journal of Pediatric Gastroenterology and Nutrition demonstrated that sirolimus significantly reduced gastrointestinal bleeding and improved anemia in a cohort of BRBNS patients.

2023

Identification of Novel Somatic Mutations in BRBNS

Research published in Human Genetics identified novel somatic mutations in the TEK gene in a subset of BRBNS patients, providing further insight into the genetic etiology of the disease.