Aplastic anemia
Disease Information
Overview
We're excited to have a program that brings together the clinical and supportive expertise necessary to provide the best individualized care for patients with aplastic anemia and their families. In addition, we're expanding research on this disease by using novel techniques to convert skin cells into blood-forming cells, with the goal of providing new stem cell therapies.
Colin Sieff, MB.BCh., Director, Bone Marrow Failure Program
Normally, bone marrow produces all of the blood cells your child’s body needs. Aplastic anemia is a blood disorder that occurs when the bone marrow fails to make enough of all types of blood cells: red cells, white cells and platelets.
Aplastic anemia was once fatal in many cases. However, treatment for children with the condition has improved significantly due to innovative new therapies including stem cell transplantation and effective supportive care. Here are some important things you should know about this condition:
- It affects three in every million people in the United States each year.
- Aplastic anemia in children can occur sporadically with no known reason.
- It can also result from a previous illness or existing disorder.
- Aplastic anemia is typically diagnosed using blood tests.
- The disease is most often treated using drugs that suppress the immune system.
- A hematopoietic stem cell transplant is currently the only cure for aplastic anemia.
How Boston Children's Hospital approaches aplastic anemia
At Children’s Hospital Boston, we’ll approach your child’s treatment with sensitivity and support—for your child and your whole family. It will be our constant goal to maximize your child’s health and quality of life.
Our Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic provides comprehensive treatment for children aplastic anemia. We are a national pediatric hematology oncology referral center, with one of the nation’s most experienced, multidisciplinary teams in the diagnosis and treatment of aplastic anemia.
- We offer specialized diagnostic and treatment options, including hematopoietic stem cell transplantation.
- We offer direct referral to Dana-Farber/Children’s Hospital Cancer Center’s Pediatric Stem Cell Transplant Program, one of the nation’s oldest and most experienced pediatric stem cell transplant programs.
- Our pediatric hematology experts provide your child with on-going, comprehensive follow-up care to manage the symptoms of aplastic anemia.
| Children’s Back to School program |
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At age 11, Ronald (R.J.) Agostinelli was diagnosed with acute lymphoblastic leukemia, a cancer of the white blood cells. He missed seven months of elementary school while having chemotherapy. Here, R.J. talks about what it was like returning to his class after a long absence. |
Reviewed by Colin Sieff, MB.BCh.,
© Children’s Hospital Boston, 2010

