Diseases that can be treated with a marrow or umbilical cord blood transplant (also called hematopoietic cell transplant) in children can be quite different from diseases that primarily affect adults. Transplant-related research is uncovering the best treatment options for children who need a transplant.
Because a transplant can also affect a child’s future growth and development, these issues are of particular concern to researchers dedicated to improving the post-transplant quality of life in young patients.
A recent study focused on the optimal transplant timing for children with severe aplastic anemia.
Transplantation preferred in pediatric aplastic anemia
For children with severe aplastic anemia, the two standard treatment options are immunosuppressive therapy (IST) or a transplant. Children with severe aplastic anemia whose disease does not respond to an initial course of IST have significantly higher survival if they then undergo allogeneic transplantation rather than receive a second course of IST. (Allogeneic transplants use cells from a donor, either related or unrelated.)
Results from a long-term study in Japan show that five-year survival was 83.9% in 31 patients who received a transplant and 9.5% in 21 patients who received a second course of IST. [1]
Research is shaping treatment
Research results from studies such as these are being applied every day in the clinic and hospital transplant units to improve outcomes for young patients.
One way your funding helps
Be The Match Foundation® understands investing in cutting-edge research is critical to serving more patients in need of a transplant. Funding clinical and observational studies is costly. But the data gleaned from in-depth patient tracking is so vital to refining therapies, improving survival rates and discovering the next generation of transplant care that your funds are needed here.
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Information for patients and families
See our information for patients and family members about:
Updates on current transplant research
References
- Kosaka Y, Yagasaki H, Sano K, et al. Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia. Blood. 2008; 111(3):1054-1059.
http://bloodjournal.hematologylibrary.org/cgi/content/full/111/3/1054