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Intra-arterial Steroid Therapy for Severe Graft Versus Host Disease in Bone Marrow and Stem Cell Transplants Joshua L. Weintraub, MD Associate Professor of Radiology & Surgery Chief, Division of Vascular & Interventional Radiology Mount Sinai Medical Center New York, NY Graft Versus Host Disease (GVHD) • Healthy patients have “killer” cell that attack and destroy foreign invades such as viruses • The transplant patient’s immune system is incapable of mounting an effective immune response • The patient’s tissues are felt to be “foreign” and are damaged by the “killer” cells in the transplant • Damage primarily occurs to the skin, mucous membranes, biliary ducts, and intestine Symptoms • Mild - skin rash • Moderate – skin rash with gastrointestinal symptoms including diarrhea • Severe – skin ulceration, liver dysfunction, severe diarrhea and protein loss associated with a high mortality Graft Versus Host Disease (GVHD) • Occurring in up to 3060% recipients bone marrow transplants from identical sibling stem cell transplants • When the donor is a mismatched sibling or unrelated donor the rate increases to 70-90% Am J Hematol 81:959-962 (2006) Current Treatments • Primary therapy is directed at suppressing the patients immune system with corticosteroids – Reports from 1990’s indicate that 80% of responses incomplete or not sustained • The survival in patients who do not respond to steroids is around 10% Weisdorf, D., et al., Blood, 1990. Intra-arterial Steroid Therapy • Our study evaluates the delivery of high concentration of corticosteroids intra-arterially to the effected organs. • The potential advantage of this treatment lies in the fact that direct intra-arterial administration of steroids may allow significant doses to be delivered to the area most affected, without exposing the patient to the deleterious effects of whole body steroids. Background • 15 patients having undergone bone marrow or stem cell transplantation developed severe GVHD of the gastrointestinal tract and liver • Ages 39 to 71 • Failed conventional medical treatment 60 year old with AML (Acute Myloid Leukemia) • Bone Marrow Transplant from sibling • Severe GVHD • Complete response after single intraarterial treatment with corticosteroids Over 60% of our patients responded • Partial response: 26.7% – Improved symptoms, discharged on intravenous feedings • Complete response: 40 % – Resolution of symptoms, discharged on regular diet Conclusion Intra-arterial infusion of high dose corticosteroids shows promise as an effective and safe treatment for severe complications of bone marrow and stem cell transplantation