Pediatric Blood & Marrow Transplantation Center
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Severe Aplastic Anemia

University of Minnesota Masonic Children’s Hospital is one of the largest treatment centers for patients with Severe Aplastic Anemia (SAA) undergoing blood and marrow transplant (BMT) in the United States. The care provided is individually tailored to meet the unique needs of each patient and family. Our team of expert health care providers who specialize in bone marrow failure syndromes like SAA, apply leading-edge research developed by University of Minnesota scientists.

This coordinated care, combined with leading research and long-term follow-up, is one reason the university is a top referral center for diagnosing, monitoring and treating children with SAA.

General overview

SAA is a rare condition in which quantities of white blood cells, red blood cells and platelets in the blood and the bone marrow are either very low or absent. As a result of the “empty” bone marrow, patients with SAA can be pale, and may experience fatigue (due to lower than normal red blood cells), infections (due to lower than normal white blood cells) and increased bruising and bleeding (due to lower than normal platelets).

SAA usually arises in a previously healthy child. While some cases of SAA have been linked to infections (e.g., hepatitis viruses), rarely exposure to radiation and chemicals (including some medications), the exact cause is not known in most cases. It is likely that SAA is not one disease but rather a group of conditions, which present in a similar way. In addition, the mechanism whereby the bone marrow cells decrease in number is often the result of an autoimmune disorder where the body’s immune system attacks the blood-forming cells.


Aplastic anemia is rare. In the United States, about 600-900 people are diagnosed each year. Aplastic anemia occurs more frequently in eastern Asian countries. It can affect people of any age, but it is most common in young adults.

At diagnosis

Drug therapy begins with anti-thymocyte globulin and cyclosporine, which suppress the immune attacks. These medications need to be administered for a course of several months. Drug treatment, however, is not always effective in increasing the blood counts. When the drug therapy does not work, bloods and marrow transplant needs to be considered.

Blood and Marrow Transplantation

For patients who are good candidates, BMT is the next line of therapy for children with severe aplastic anemia. BMT from a matched sibling donor should be recommended to all SAA patients who have one, even prior to use of anti-thymocyte globulin and cyclosporine. BMT from an alternative donor, however, should be recommended to all patients who have not responded to immunosuppressive therapy.

Learn more about what to expect during the transplant process.

While our entire blood and marrow transplant team is involved in the care of our patients, the following providers lead the care and treatment plans for patients with SAA and other bone marrow failure syndromes.

Margy MacMillan, MD, M.Sc.

Jakub Tolar, MD, PhD

John Wagner, MD

We continue to explore novel ways to increase cure rates and improve quality of life for patients with SAA. We also collaborate with other researchers throughout the world, keeping our program at the forefront of SAA care worldwide. Click here to see a list of SAA articles and studies our team has published in academic and science journals

Learn more about current research projects.

SAA transplant patients may be eligible for new clinical trials, taking advantage of the latest breakthroughs in therapies and treatments. View a list of open trials.

If you would like to gather more information about Severe Aplastic Anemia, please reference the following recommended sources.

Journey Clinic - Pediatric Blood and Marrow Transplantation Center

2450 Riverside Avenue
Minneapolis, MN 55454
612-365-1000 TTY: 612-672-7300

For information about the Adult Blood and Marrow Transplantation program, click here:

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We have been providing innovative care to children in need of a blood and bone marrow transplant for over 50 years. Call us, we're here to help.

(888) 601-0787 or (612) 273-2800

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