Gorham Stout Disease

What is Gorham-Stout disease?

Children with Gorham-Stout disease experience gradual bone loss (osteolysis) caused by an abnormal overgrowth of lymphatic vessels. These vessels are a normal part of the body’s lymphatic system, which transports a clear fluid containing white blood cells called lymph around the body to help clear toxins and waste. In patients with Gorham-Stout, these thin-walled lymphatic vessels expand (dilate) and multiply, leading to the rapid breakdown of bone (bone resorption).

In children and young adults, bone resorption is a normal process of bone growth, in which new bone replaces old. For those with Gorham-Stout, the overgrown lymphatic vessels may change the balance of bone formation and loss, leading to loss of bone and presence of lymphatic vessels where bone used to be.

Gorham-Stout disease is sometimes called vanishing bone disease or idiopathic or progressive massive osteolysis.

How we care for Gorham-Stout disease

With fewer than 300 known cases of Gorham-Stout, the majority of physicians will never encounter this rare bone disorder. The expert team at Boston Children’s Vascular Anomalies Center has treated or consulted on more than 50 patients from all over the world, giving us extensive experience diagnosing and treating Gorham-Stout at different ages.

Because Gorham-Stout affects surrounding tissues in addition to bone, we often need to treat related complications as well as the disorder itself. Your child will benefit from the expertise of a comprehensive, multidisciplinary team working together to develop a customized treatment plan.

Our areas of innovation for Gorham-Stout disease

While there is no cure for Gorham-Stout, our groundbreaking research with investigational drug therapies has reversed or lessened some of the effects of the disease in many children.

Our patients have access to the latest clinical protocols for investigational drug therapies. We are currently studying the use of immunosuppressant medications to slow and even halt the progression of Gorham-Stout disease.