Posted: Apr 14, 2011
Inherited bone marrow failure syndromes impact a patient’s red blood cells, white blood cells and platelets and include conditions such as Fanconi anemia, Diamond Blackfan anemia and Kostmann syndrome.(1, 2)
For patients with inherited bone marrow syndromes, donor (allogeneic) transplantation is the only curative treatment option. In the past, bone marrow has been the source of stem cells used in these procedures, but increasingly cord blood is being used.(3)
In fact, a recent study concluded that related cord blood transplantation provides excellent outcomes for children with inherited bone marrow failure syndromes. Researchers found that the patients who received a related cord blood transplant showed a very high survival rate of 95% and lower incidence of complications like graft versus host disease, compared with patients who received an unrelated cord blood transplant.(2)
The authors concluded that cord blood transplantation is an excellent treatment option for patients with an available matched sibling donor. Additionally, they believe their findings further support storing cord blood in a related or family banking setting, considering the absence of risk to the donor and the outcomes previously reported for metabolic disorders and immune deficiencies.
Many bone marrow failure syndromes are eligible for medical need based programs, such as CBR’s Designated Treatment Program®, that provide free cord blood banking services to obstetric patients with a qualifying family member diagnosed with a condition treatable by stem cell transplant. For more information on the Designated Treatment Program, please visit cordblood.com.
1. Bizzetto R, Bonfim C, Rocha V, et al. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica. Jan;96(1):134-141.
2. Besa E. Bone Marrow Failure. 2008. http://emedicine.medscape.com/article/199003. Accessed February 15, 2011.
3. MacMillan ML, Walters MC, Gluckman E. Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies. Semin Hematol. Jan 2010;47(1):37-45.