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Myelodysplastic Syndromes Treatment

Myelodysplastic syndromes (MDS) are a group of disorders in which the bone marrow does not make enough healthy blood cells. Bone marrow is the spongy material inside bones. It contains stem cells that become red blood cells, white blood cells, and platelets.

MDS signs and symptoms are related to a decrease in healthy blood cells. These include:

  • Too few red blood cells (anemia) reduces the ability to carry oxygen to the body’s tissues.
  • Too few white blood cells (leukopenia) increases the risk of infection.
  • Low platelets (thrombocytopenia) causes easy bruising and bleeding.

Causes of MDS can include previous aplastic anemia or a history of chemotherapy or radiation treatment for other cancers. MDS may also be linked to genetic conditions, such as inherited bone marrow failure syndromes and MDS predisposition syndromes. Often, the cause of MDS in children is not known.

Pediatric MDS is rare. About 4 in every 1 million children are diagnosed with MDS each year. There are 2 main types of MDS in children: refractory cytopenia of childhood (RCC) and MDS with excess blasts (MDS-EB).

Learn more about MDS on the Together by St. Jude online resource.

Treatment of myelodysplastic syndrome

Children with some types of MDS, such as RCC, may not need treatment other than close monitoring. RCC can worsen or progress to MDS-EB. In some cases, MDS can develop into acute myeloid leukemia (AML).

The most effective treatment for most types of MDS is an allogeneic bone marrow (stem cell) transplant. Chemotherapy may be used to treat patients with MDS, MDS-EB, or patients who have developed AML.

Other treatments for MDS include medicines to help the bone marrow make new blood cells and antibiotics to prevent or treat infections. Red blood cell and platelet transfusions can help with symptoms of anemia and bleeding.

Myelodysplastic syndrome  clinical trials

St. Jude offers clinical trials and cancer research studies for children, teens, and young adults for myelodysplastic syndromes.

Learn more about clinical research at St. Jude

10-CBA: Access and Distribution of Unlicensed Cryopreserved Cord Blood Units

Study goal:

The primary objective of this study is to examine the incidence of neutrophil recovery after cord transplantation.

In participants receiving a non-licensed CBU: 1) to determine the frequency of graft rejection; 2) to determine the frequency of infection; 3) to determine the frequency of serious infusion reactio; 4) to determine the 1 year survival rate.

NCBP01: Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

Study goal:

The primary purpose of this study is to examine the safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products in a multi-institution setting.

CPXSMN: CPX-351 in Pediatric Patients with Secondary Myeloid Neoplasms

Study goal:

This study will help us understand the effects of CPX-351 treatment in patients with MDS and AML.


1-22 years old

DIRECT70: CAR T–Cell Therapy for Children with Blood Malignancies

Study goal:

The purpose of this study is to find the highest dose of CD70+ CAR cells that is safe to give to patients with CD70+ blood cancers.


Up to 21 years old

REF2HCT: Haploidentical Bone Marrow Transplant for Relapsed or Refractory Leukemia, Lymphoma, or Myelodysplastic Syndrome After an Earlier Transplant

Study goal:

The main goal of this study is to learn about the good and bad effects of transplanting blood cells donated by a family member to children and young adults with cancer that has come back or did not improve after a previous bone marrow transplant.


21 years old and younger

Myelodysplastic syndrome care at St. Jude

St. Jude provides the highest quality of care for patients with MDS:

  • St. Jude scientists and doctors work together to learn more about bone marrow failure disorders and blood disorders. These findings can lead to new and better treatments.
  • St. Jude doctors and faculty members are engaged in the latest research studies. Patients who qualify for clinical trials may choose to take part. By doing these studies, we hope to better understand and treat blood disorders.
  • The St. Jude Bone Marrow Transplant and Cellular Therapy program uses state-of-the-art approaches to provide the best treatments and reduce side effects for pediatric patients.
  • Doctors in the St. Jude Transplant Program work closely with scientists to rapidly move discoveries from the lab to the clinic.
A statue of children running and holding hands

Seeking treatment at St. Jude

Patients accepted to St. Jude must have a disease we treat and must be referred by a physician or other qualified medical professional. We accept most patients based on their ability to enroll in an open clinical trial.

How to seek treatment

Contact the Physician / Patient Referral Office

Call: 1-888-226-4343 (toll-free) or 901-595-4055 (local)  | Fax: 901-595-4011 | Email: | 24-hour pager: 1-800-349-4334


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