The success in treating pediatric illness is the best example of success through collaboration. In clinical studies, researchers prescribe promising treatment regimens, monitor the patients responses carefully, and compare this treatment to standard treatment. More than half of all children with cancer and blood disorders in the United States receive treatment through clinical trials.
For sickle cell disease, bone marrow transplant is currently the only curative therapy. Beginning with seminal studies led by Fred Hutchinson Cancer Research Center, researchers have documented disease-free survival of around 85 percent and overall survival around 95 percent in hundreds of children with matched sibling donors. Unfortunately, fewer than one in five children with SCD have a matched sibling and unrelated donor transplantation in SCD has met with less success. Outcomes in adults with SCD also have been less successful due to both disease- and transplant-related mortality. SCCA currently has several protocols aimed at improving outcomes for those with SCD.
To find clinical trials for which your child may be eligible, ask your child’s health-care team.