Julian's Dinosaur Guild

Dr. Lauri Burroughs was Julian's lead transplant doctor in 2011. Thanks to Dr. Burroughs and her team, Julian continues to enjoy a happy and healthy life. Read on to learn more about aplastic anemia and the clinical research our fundraising helps support.

Acquired severe aplastic anemia is a rare bone marrow failure disorder. Blood cells are all made in a space inside your bones called the bone marrow. There are many types of blood cells, including red blood cells which carry oxygen and give you energy, white blood cells which fight infections, and platelets which help your blood to clot. In aplastic anemia, the bone marrow stops working and thus does not make enough red blood cells, white blood cells, or platelets. When this happens you may be anemic (tired and pale), have fevers or infections, or have increased bruising or bleeding symptoms. These symptoms may be mild initially but if not treated can become life threatening. The cause of acquired aplastic anemia is not fully understood but researchers believe that in many cases the patient’s own immune system attacks or destroys the cells in the bone marrow, causing it to fail.  Treatment options include replacing the bone marrow with cells from a healthy donor (bone marrow transplant), or using strong medications to suppress the immune system and allow the marrow to recover on its own.

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Researchers at the Fred Hutchinson Cancer Research Center were pioneers in the development of bone marrow transplantation for patients with severe aplastic anemia. In order to perform a bone marrow transplant, first an appropriate donor needs to be identified. Bone marrow donors should be HLA matched, meaning that the genes that determine what the body recognizes as ‘self’ are the same. Full siblings, that is brothers or sisters who have the same parents, have a 25% chance of being HLA-matched to each other. Patients with aplastic anemia who undergo transplant with a matched sibling donor have excellent outcomes, with survival greater than 95%. Therefore, bone marrow transplant is now considered the treatment of choice for children who have an HLA matched sibling.

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However, many children may not have a matched sibling, as there is only a 1 in 4 (25%) chance that any brother or sister will be HLA-matched. When a patient does not have a sibling who could serve as a marrow donor, doctors will search a large international listing of volunteers called the National Marrow Donor Registry to try to identify an unrelated donor who might by chance be a match. Because it is rare for 2 people who are not siblings to be HLA-matched, a large number of potential donors need to be screened and this process takes time and expertise and sometimes is unsuccessful. Historically the survival following transplantation using an unrelated donor has not been as good as that seen with a matched sibling donor, though recently outcomes have improved significantly.

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Because unrelated donor transplant has been associated with greater risk and finding a donor can be difficult, patients with aplastic anemia who do not have a matched sibling donor are treated with immune suppressing medications to hopefully stop the attack on their bone marrow cells and allow recovery. While pursuing this course of therapy, doctors will search for an unrelated donor, so that if the immune suppressive treatment is not successful, they can proceed with a bone marrow transplant using an unrelated donor. This was the case for Julian.

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Over the past decade, researchers around the world have been working on improving the transplant process and recently researchers across the United States came together and conducted a prospective bone marrow transplant clinical trial using unrelated donors for patients with severe aplastic anemia. Importantly, the survival on this national clinical trial approached that seen using a fully matched related donor! As a result, researchers have raised the question as to whether or not patients with severe aplastic anemia should go to transplant right away regardless of the donor. Six centers across the United States including the Fred Hutchinson Cancer Research Center and Seattle Children’s Hospital have developed a prospective clinical trial that is comparing upfront bone marrow transplant using an unrelated donor to upfront immune suppression to see which approach is better. This is the first prospective clinical trial in the world that compares these 2 treatment approaches and will help answer vital questions that have remained unanswered including what is the best treatment approach for patients with severe aplastic anemia. Julian would have qualified for this trial if it were open when he was first diagnosed.