Overview
Gorham-Stout disease (GSD) is a very rare bone disorder where one or more bones slowly disappear over time. It is also known as 'vanishing bone disease,' 'phantom bone disease,' or 'massive osteolysis.' In this condition, abnormal blood vessels and lymphatic vessels (tiny channels that carry fluid through the body) grow into bone tissue and gradually replace it. The bone essentially dissolves away, which can cause pain, weakness, and deformity in the affected area. Gorham-Stout disease can affect almost any bone in the body, but it most commonly involves the skull, spine, shoulder, ribs, and pelvis. As the bone breaks down, nearby bones can also become involved. One of the most serious complications happens when the disease affects the spine or chest area, which can put pressure on the spinal cord or cause fluid to build up around the lungs — a condition called chylothorax. There is currently no cure for Gorham-Stout disease. Treatment focuses on slowing the bone loss, managing pain, and preventing complications. Options include radiation therapy, medications such as bisphosphonates (which help protect bone), and drugs that target abnormal blood vessel growth. Surgery may be needed to stabilize bones or repair damage. Because this disease is so rare and unpredictable, care is highly individualized and usually managed by a team of specialists.
Also known as:
Key symptoms:
Bone pain that gets worse over timeSwelling or tenderness near affected bonesBones that break easily or without much forceGradual loss of bone visible on X-rayWeakness or limited movement in the affected areaFluid buildup around the lungs (chylothorax)Difficulty breathing if the chest or spine is affectedSpinal cord problems such as numbness, tingling, or weakness in the limbsDeformity or collapse of affected bonesHeadaches or neurological symptoms if the skull is involvedDifficulty swallowing if bones near the throat are affected
Clinical phenotype terms (36)— hover any for plain English
Sporadic
Usually appears on its own, not inherited from a parent
Variable
Can begin at different ages, from infancy through adulthood
Treatments
No FDA-approved treatments are currently listed for Gorham-Stout disease.
View clinical trials →Clinical Trials
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Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Gorham-Stout disease.
Community
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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.
Questions for your doctor
Bring these to your next appointment
- Q1.Which bones are currently affected, and how quickly does the disease appear to be progressing in my case?,What treatment options do you recommend for me, and what is the goal of each treatment?,Should I be referred to a center with specific experience in Gorham-Stout disease?,What warning signs should prompt me to go to the emergency room immediately?,Are there any clinical trials or experimental treatments I might be eligible for?,How often do I need imaging to monitor the disease, and what type of imaging is best?,What activity restrictions should I follow to reduce my risk of fractures or other complications?
Common questions about Gorham-Stout disease
What is Gorham-Stout disease?
Gorham-Stout disease (GSD) is a very rare bone disorder where one or more bones slowly disappear over time. It is also known as 'vanishing bone disease,' 'phantom bone disease,' or 'massive osteolysis.' In this condition, abnormal blood vessels and lymphatic vessels (tiny channels that carry fluid through the body) grow into bone tissue and gradually replace it. The bone essentially dissolves away, which can cause pain, weakness, and deformity in the affected area. Gorham-Stout disease can affect almost any bone in the body, but it most commonly involves the skull, spine, shoulder, ribs, and
How is Gorham-Stout disease inherited?
Gorham-Stout disease follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Which specialists treat Gorham-Stout disease?
19 specialists and care centers treating Gorham-Stout disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.