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Bone Marrow Transplantation and Immune Deficiency

Bone Marrow Transplantation and Immune Deficiency

 

Significant Accomplishments

Treatment of HLH

Our Bone Marrow Transplant Program is a leader in diagnosis and treatment of HLH, with children travelling to Cincinnati from across the US and other nations for therapy.  This year Rebecca Marsh, MD, and Lisa Filipovich, MD, published data in the journal Blood about the outcomes of two transplant strategies and identified reduced intensity transplantation as the optimal approach. 

Fanconi Anemia Comprehensive Care Clinic

The Fanconi Anemia Comprehensive Care Clinic continues to expand.  We offer life-long care to more than 150 children and adults with this rare disorder. Most children with Fanconi anemia eventually develop marrow failure and require transplantation.  We have completed an important study of endocrinological abnormalities in this population, led by Susan Rose, MD, Division of Endocrinology.  We also have explored the mechanism of endocrinopathy in a mouse model study lead by Qishen Pang, PhD, Division of Experimental Hematology.  This study showed that oxidant stress causes tissue damage in Fanconi anemia.  Parinda Mehta, MD, has used these data to develop a novel clinical trial using an anti-oxidant food supplement.

Prospective proteomic study of TMA

Transplant associated thrombotic microangiopathy (TMA) is a severe complication of transplantation, commonly leading to organ failure and death.  Sonata Jodele, MD,  in collaboration with the Division of Nephrology, has launched a study of proteomic biomarkers of TMA, identifying possible markers that can predict occurrence of the disorder 10 to 14 days before clinical presentation.  We expect these data to generate new treatment options for this disorder.

Intensive care and bone marrow transplant

Bone marrow transplant is associated with significant morbidity and mortality, and a significant proportion of children will require intensive care.  We have collaborated with Ranjit Chima, MD, and Derek Wheeler, MD, FAAP, to analyze intensive care outcomes in this high-risk population. The data show that 82 percent of children admitted after stem cell transplant survive and leave the ICU.  These outstanding results were presented at the annual meeting of the American Society of Bone Marrow Transplant and the Society for Critical Care Medicine.
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