Myelodysplastic Syndrome

Myelodysplastic syndrome (MDS) is a rare disease of the blood, only occurring in four out of every 1 million children. This rare disease keeps the body from properly producing blood cells and producing enough of them. MDS develops in the bone marrow, the soft, spongy center of the long bones that produces the three major blood cells. With this disease, the blood cells lose their ability to mature and function properly.

How Dana-Farber/Boston Children's approaches myelodysplastic syndrome
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center has one of the nation’s most experienced, multidisciplinary teams at diagnosing and treating pediatric MDS. Dana-Farber/Boston Children’s is the only large pediatric center in the U.S. that has been awarded the MDS Centers of Excellence award by the MDS Foundation.

At Dana-Farber/Boston Children’s, children with myelodysplastic syndrome are treated through our Myelodysplastic Syndrome Specialty Program. We offer specialized diagnostic and treatment options, and our Pediatric Stem Cell Transplant Center is one of the largest and most experienced pediatric stem cell transplant programs in the world.

In almost all instances, MDS in children can be cured only through a bone marrow transplant, also known as a hematopoietic stem cell transplant (HSCT). HSCT uses high doses of chemotherapy or radiation therapy to destroy all the cells in the bone marrow, healthy and diseased ones. Healthy cells from the bone marrow of another person—either a relative (usually a sibling) or an unrelated individual—are given through an infusion to the patient to restore the bone marrow that was previously destroyed by the chemotherapy and/or radiation therapy.

Learn more
Find more in-depth information about myelodysplastic syndrome (MDS) on the Dana-Farber/Boston Children’s website, including answers to:

  • What causes MDS?
  • What are the symptoms of MDS?
  • How do you diagnose MDS?
  • How do you treat MDS in children?
  • What is the latest research on MDS?
  • What is the long-term outlook for children with MDS?

Clinical Trials
For many children with rare or hard-to-treat conditions, clinical trials provide new options.