Children's National researcher leads study on risk factors in pediatric blood and marrow transplant
For Immediate Release
Washington, DC — David A. Jacobsohn, MD, Chief of the Blood and Marrow Transplantation Division at Children’s National Medical Center, is the lead author of the first large-scale retrospective study that clarifies risk factors specific to pediatric patients who have undergone allogeneic hematopoietic stem cell transplantation (HSCT) and have developed chronic graft-versus-host disease (cGVHD).
Published this week in Blood, the peer reviewed journal of the American Society of Hematology, this is the largest study specific to pediatric patients to analyze multiple demographic and clinical factors associated with non-relapse mortality (NRM) after diagnosis of chronic graft-versus-host-disease (cGVHD), the most frequent complication after HSCT.
“Understanding which variables more likely influence outcomes in pediatric patients will help us counsel families and determine the most effective therapeutic approaches,” said Dr. Jacobsohn. “This large study addresses the significant lack of information specific to pediatric patients being treated with blood and/or marrow transplantation for diseases such as leukemia and myelodysplastic syndrome. The results will help us define future clinical trials, and we will be more capable of risk-stratifying patients at diagnosis of cGVHD.”
Blood and marrow transplants are commonly used now to treat a variety of cancers not responding to conventional chemotherapy, as well as a number of non-cancer diagnoses, such as sickle cell disease.
This study involved 1,117 patients with a median age of 12 years. The most common diagnosis among these patients was acute lymphoblastic leukemia (ALL) (49%), followed by acute myeloid leukemia (AML) (27%). Thirteen percent of patients had been transplanted when they were in advanced stages of disease and most patients (97%) had received a myeloablative (complete destruction of marrow function with chemotherapy and/or radiation) pre-treatment regimen. Bone marrow was the most common graft source (71%), followed by peripheral blood (20%), and umbilical cord blood (9%). Dr. Jacobsohn and his team identified that certain risk factors, such as low platelets (among others) at diagnosis of cGVHD led to a significantly worse long-term outcome.
“Parents and even teenagers seek us for transplantation because they have tried all other avenues to treat severe disease,” continued Jacobsohn. “At Children’s National, we have excellent outcomes for pediatric patients with diseases like leukemia and sickle cell disease. This study will help us refine our practices as we continuously search for cures.”
The complete study is available through the journal Blood.
Contact: Paula Darte or Emily Hartman, Children’s Public Relations: 202.476.4500.