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Aplastic Anemia Treatment

Aplastic anemia is a blood disorder that affects the bone marrow. Bone marrow is a sponge-like material found in the center of many bones. It contains the stem cells that become blood cells. 

In aplastic anemia, the stem cells in the bone marrow do not make enough new blood cells. Most often, all 3 types of blood cells are affected (called pancytopenia):

  • Red blood cells that carry oxygen
  • White blood cells that fight infections
  • Platelets that help the blood to clot and stop bleeding

In most cases, aplastic anemia occurs in previously healthy children without a clearly identified trigger. It may also occur after certain viral infections. It is not inherited from parents. While in some cases aplastic anemia is linked to cancer or cancer treatments it is not a type of cancer.

Complications of aplastic anemia include serious infections, bleeding, and heart problems. If untreated, this disorder can be life threatening.

Learn more about aplastic anemia on the Together by St. Jude™ online resource.

Treatment of aplastic anemia

Treatment for aplastic anemia may include blood transfusions, medicines that inhibit the immune system and stem cell (bone marrow) transplants.

Aplastic anemia clinical trials

St. Jude offers clinical trials and research studies for children, teens, and young adults with aplastic anemia. 

Open clinical trials

HAPSAA: Partially Matched Related Donor Bone Marrow Transplant for Patients with Aplastic Anemia

Study goal:

The main goal of this study is to learn more about the effects (good and bad) of this treatment in children and young adults with high-risk blood disorders.


Aplastic anemia


21 years old and younger

TransIT3: Immune Therapy vs. Unrelated Donor Transplant for Severe Aplastic Anemia

Study goal:

Determine the optimal, upfront therapy for pediatric SAA in the absence of a matched sibling donor. 


Aplastic Anemia


Up to 25 years old

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.


Leukemia and other blood diseases

Aplastic anemia care at St. Jude

  • St. Jude scientists and doctors work together to learn more about bone marrow failure and blood disorders. These findings can lead to new and better treatments.
  • St. Jude also develops collaborative research partnerships with the NIH and other institutions throughout the world.
  • The St. Jude Bone Marrow Transplant and Cellular Therapy program provides state-of-the-art approaches to provide the best treatments and reduce side effects for pediatric patients. Doctors in the 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


Learn more