How is aplastic anemia treated?
Treatment for children with aplastic anemia is focused on getting the body to make healthy blood cells or supplementing with blood transfusions and other medical treatments.
If the patient has a sibling who is a matched donor, a bone marrow transplant should be considered through our Bone Marrow Transplant Program.
If there is no sibling match or a transplant is not a good option, the patient will be seen by blood specialist who can discuss therapy options with the patient and family. Therapies can also include immunotherapy, which modifies the patient's immune system to protect bone marrow.
Why choose Children's Hospital Colorado for your child's bone marrow failure?
Parents choose us because:
- Pediatric experts in our Blood and Marrow Transplant and Cellular Therapy Program have performed more than 1,000 bone marrow transplants. We've been treating children with bone marrow failure for more than 25 years, making us one of the most experienced pediatric BMT programs in the country.
- At our Center for Cancer and Blood Disorders, we have expert blood doctors who have designed therapy regimens that are non–toxic and successful at increasing blood counts in our patients.
- We provide personalized medical management and word towards accurate diagnosis and curative therapy.
- We partner with genetic counselors to better understand the disease's impact on a family, including predisposition to cancer or other disorders.
- We offer interventional clinical trials and research studies available only at limited institutions around the world. We also have a partnership with the North American Pediatric Aplastic Anemia Consortium (NAPAAC).
Learn more about our nationally-ranked Center for Cancer and Blood Disorders.
Bone marrow failure research at Children's Colorado
Severe aplastic anemia is rare — about 100 cases per year nationally, many with no clear cause. Collaboration, says pediatric hematologist Taizo Nakano, MD, will be key to improving outcomes. He and his team at Children's Colorado have collaborated with Boston Children's Hospital to build a biobank to study the disease.
"Already," he says, "we've identified novel gene mutations that disrupt unstudied pathways in bone marrow failure."
Through the North American Pediatric Aplastic Anemia Consortium — the first organization of its kind — prospective clinical trials are underway, targeting these mutations for potential new therapies. One day, Dr. Nakano hopes, they may lead to better, more personalized options for care.