SUCCESSFUL ENGRAFTMENT OF UNRELATED CORD-BLOOD STEM-CELLS FOR FAMILIAL ERYTHROPHAGOCYTIC LYMPHOHISTIOCYTOSIS

Familial erythrophagocytic lymphohistiocytosis (FEL) is an autosomal r ecessive disorder that can only be corrected by stem cell transplantat ion. One of our patients with FEL in second complete remission underwe nt successful cord blood stem cell transplantation (CBSCT); the donor was an HLA one-locus mismatched and unrelated individual. The conditio ning regimen consisted of BU/CY/VP-16, The transfused cell dose was 6. 8 x 10(7)/kg, which contained 1.36 x 10(5)/kg of CD34 cells and 3.4 x 10(4)/kg of CFU-GM, After CBSCT, there were no major infectious compli cations. Acute grade I GVHD was well controlled. Neutrophil counts rea ched >0.5 x 10(9)/l by day 20 and platelet counts reached >50 x 10(9)/ l by day 40. Deficient natural killer activity returned to normal afte r the transplant. The patient recovered well more than 7 months after receiving CBSCT, without showing evidence of chronic GVHD. We recommen d CBSCT for FEL patients who have no HLA-matched siblings or unrelated donors.