Gorham-Stout Disease Treatments

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Contact the Vascular Anomalies Center

Our treatment team focuses on giving hope to patients with Gorham-Stout and their families. Our pioneering work with investigational therapies has successfully halted or reversed the course of this progressive bone disease in most patients participating in our clinical studies. 

Your child’s specific treatment plan depends on several factors, including the part of the body affected, severity and progression of the disease and related complications.

Treatment options may include:

  • Medication/drug therapy
  • Surgery
  • Radiation therapy (rarely)

Drug Therapy for Gorham-Stout

Throughout the 20th century, the standard treatment approach for Gorham-Stout was palliative care to relieve the patient’s symptoms. Surgeons often performed orthopedic surgeries to stabilize bone that would further break down months or years later.

Today, anti-vascular and immunosuppressant medications have proven effective for slowing and even reversing the damaging effects of Gorham-Stout. Boston Children’s offers a clinical protocol using these medications, which target the lymphatic vessels that grow abnormally and disrupt the body’s normal bone regeneration process.

Our clinical studies include these medications:

  • Sirolimus: Also known as rapamycin, this oral medication has been found to effectively slow the progression of Gorham-Stout, reduces symptoms and complications and generally stabilizes the disease.  This medication suppresses the immune system and targets lymphatic vessel formation.
  • Interferon alfa-2b: Medication that inhibits the formation of lymphatic vessels and improves symptoms.
  • Bisphosphonates: A class of medications designed for osteoporosis that work to slow bone loss and can help to stabilize Gorham-Stout disease.

Read more about Gorham-Stout research.

Surgery for Gorham-Stout

Surgery alone cannot cure Gorham-Stout. However, your child’s physician may recommend a surgical procedure to stabilize or remove affected bone, or to treat symptoms and complications related to the disease.

At Boston Children’s, we may recommend surgery in conjunction with or after a drug therapy course has successfully stabilized the disease. Surgical outcomes are improved when the disease is under control at the time of surgery.

Surgical recommendations may include:

  • Spinal reconstruction or spinal fusion surgery to stabilize affected vertebrae. We work closely with expert surgeons in our Spinal Program.
  • Bone graft surgery, which in some cases can stimulate the formation of new bone.
  • Surgical removal of affected bone, which may be replaced by the bone graft surgery above or with an artificial, or prosthetic, bone implant.

Radiation therapy for Gorham-Stout

Your child’s physician may recommend radiation to treat the symptoms of Gorham-Stout, such as pain. Radiation is also an effective treatment for complications of the disease, such as chylothorax, a leakage of lymphatic fluid into the chest.  Due to delayed side effects of radiation therapy, we do not recommend radiation as first-line therapy.

Treating Gorham-Stout at Boston Children’s: Our Approach

Our patients benefit from a comprehensive team approach that involves multiple specialists working together to develop customized treatment plans. You and your child can meet with our team during a dedicated clinic day, which allows you to ask as many questions as necessary to fully understand our recommended course of treatment.

As part of the Boston Children’s Vascular Anomalies Center, our experts include:

  • Hematologists who use the most advanced medical therapies today that have reversed the course of the disease in most patients
  • Orthopedic surgeons, including top surgeons from our Spinal Program, to stabilize affected bone through reconstructive procedures.
  • Complete team of medical specialists from neurologists and cardiologists to interventional radiologists who treat the full spectrum of complications related to Gorham-Stout
  • Researchers studying the latest investigational therapies and possible genetic links of the disease
  • Plastic surgeons to repair or improve the appearance of bone and soft tissue deformities
  • Dedicated social worker to help coordinate services to treat the emotional needs of your child and arrange support services.

Meet our Vascular Anomalies team.

Gorham-Stout Consultations for International Patients

We offer second opinions and consultations for patients living across the U.S. and around the world. If travel prohibits you and your child from receiving ongoing care on-site at our Boston hospital, our experts are available to evaluate your child and consult with a local physician to recommend the best treatment course.

Our weekly VAC conference gives you and/or your child’s care team access to our Gorham-Stout experts – without the need to travel to Boston.

To submit a case for review, read more about our VAC conference.

If your child is an international patient, learn more about making an appointment through Boston Children’s International Health Services.

Reviewed by Cameron Trenor, MD, © Boston Children’s Hospital

Boston Children’s is so much more than a hospital—it’s a community of researchers, clinicians, administrators, support staff, innovators, teachers, patients and families, all working together to make the impossible possible. ”
- Sandra L. Fenwick, President and CEO

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