Complete Response to Tacrolimus in a Child With Severe Aplastic Anemia Resistant to Cyclosporin A

Aplastic anemia (AA) is a rare disorder in children, usually treated with immunosuppressive therapy (IST) including antithymocyte globulin (ATG) and cyclosporin A. There are no current widely used alternative therapies with comparable efficacy. We describe a child with severe aplastic anemia (SAA), who developed severe gingival hyperplasia secondary to cyclosporin A, unresponsive to intensive dental intervention. When IST was changed to tacrolimus there was a significant improvement in the gingival hyperplasia, but equally important, he achieved complete response of his AA within several months. The use of tacrolimus in children with AA may he a potential modality of treatment. Pediatr Blood Cancel 2009;52:525-527. (c) 2008 Wiley-Liss, Inc.