UMBILICAL-CORD BLOOD INFUSION IN A PATIENT FOR CORRECTION OF WISKOTT-ALDRICH SYNDROME

A 23/4 year old male with thrombocytopenia secondary to Wiskott-Aldric h Syndrome (WAS) and a history of two intracranial hemorrhages as well as hemolytic anemia and neutropenia received a placental blood infusi on from an HLA-identical female sibling born by caesarian section at 3 5 weeks gestation. The patient was prepared with Thiotepa and Cytoxan and received a nucleated cell dose of 3.0 x 10(7)/kg. Cyclosporin A an d Methylprednisolone was given for graft versus host disease (GVHD) pr ophylaxis. An ANC of 0.5 x 10(9)/L and 1.0 x 10(9)/L were achieved on post-transplant days 18 and 28, respectively. Platelet recovery was ra pid with a platelet count greater than or equal to 100 x 10(9)/L on da y (+)39. On posttransplant day (+)11, the patient developed an erythem atous rash consistent with grade I acute GVHD that resolved without th erapy. He was discharged day on (+)60 and has remained free of infecti ons with a normal platelet count off all immunosuppression therapy 30 months post-transplantation. Chimerism studies performed on periphera l blood mononuclear cells by fluorescent in situ hybridization indicat ed that the percentage of donor cells ranged between 55 and 80%. The p henotype and function of peripheral blood lymphocytes are completely n ormal and the patient has responded in vivo with production of antibod ies to both diphtheria and tetanus immunizations. This study demonstra tes the feasibility of collecting placental blood after a multiple bir th delivery and the ability of umbilical cord blood to provide complet e hematopoietic and immunologic reconstitution in a patient with WAS.