03024nas a2200349   4500000000100000008004100001260001300042653001800055653001500073653003200088653001000120653002100130653002000151653002600171653001100197653002900208653001100237653000900248653002400257653001500281653001600296100001200312700001200324700001800336700001800354700001600372245012900388300000800517490000800525520212700533022001402660       1999                            d  c1999 Apr10aAcute disease10aAdolescent10aBone Marrow Transplantation10aChild10aChild, Preschool10aChronic Disease10aGraft vs Host Disease10aHumans10aImmunosuppressive Agents10aInfant10aMale10aRemission Induction10aTeratogens10aThalidomide1 aMehta P1 aKedar A1 aGraham-Pole J1 aSkoda-Smith S1 aWingard J R00aThalidomide in children undergoing bone marrow transplantation: series at a single institution and review of the literature.  ae440 v1033 a<p>Thalidomide has one of the most notorious drug histories because of its teratogenicity. Its widespread use in the 1960s led to a worldwide epidemic of phocomelia in inborns; this in turn led to its complete ban in most of the world. However, it has now been licensed for selected indications including graft-versus-host-disease (GVHD) after bone marrow transplantation, wasting associated with tuberculosis and human immunodeficiency virus infection, and leprosy. Little is known, however, about its use in children in these settings. Therefore, we report our experience and review the literature on thalidomide in children for GVHD after bone marrow transplantation. We studied 6 patients, 2 with chronic GVHD, 2 with acute GVHD, and 2 with acute GVHD progressing into chronic disease. One patient with chronic GVHD had a complete response, whereas the other had a partial response. Side effects consisted primarily of sedation and constipation, which are reported previously and well known side effects. None had neuropathy. One patient had rash, eosinophilia, and early pancreatitis that began shortly after initiation of thalidomide, persisted, and resolved only after discontinuation of thalidomide. Eosinophilia and pancreatitis are both previously unreported side effects or associated findings of thalidomide treatment. Review of the literature reveals three major studies of thalidomide in GVHD; of these two included children and adults together, and one in which age range of patients was not mentioned. In addition, four series of children receiving only thalidomide are reported. These series contained 1 to 14 patients each. Results show efficacy in at least 50% of children with chronic GVHD and little or no efficacy in children with exclusively acute GVHD. Side effects are similar to those reported in adults and consisted mostly of sedation and constipation, both of which subsided over time and resolved after discontinuing the drug. We speculate on the reasons for which thalidomide is more effective in chronic, compared with acute, GVHD in children, and make recommendations for future study.</p>  a1098-4275