title: Daclizumab for children with corticosteroid refractory graft-versus-host disease.
abstract: Daclizumab, a humanized IL-2 receptor antagonist, has been found to be safe and effective in adults with refractory graft-versus-host disease (GVHD); however, data describing its efficacy for refractory GVHD in children are limited. We report a series of 14 children who were treated with daclizumab for severe acute and/or chronic corticosteroid refractory GVHD. Patients were treated with 2 mg/kg weekly for 8 weeks followed by 1 mg/kg weekly for 4 weeks. Nine of 14 patients responded to daclizumab as measured by improvement of GVHD symptoms, and the ability to substantially wean corticosteroid dose. Five of 11 patients with acute GVHD had complete symptom resolution, and 2/11 had a partial response. Two of four patients with chronic GVHD had complete symptom resolution. In these patients, daclizumab was only effective in treating skin GVHD. Seven of the nine patients who had a complete or partial response eventually had recurrence of GVHD; however, the GVHD was less severe and no longer corticosteroid refractory. There was no infusional toxicity, and no infections that could be attributable to the drug. Daclizumab is a relatively safe and effective medication for corticosteroid refractory GVHD in children and larger studies are needed to evaluate its role in treatment.
authors.affiliation: Division of Oncology in the Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA. teacheyd@email.chop.edu
authors.forename: D T
authors.initials: DT
authors.lastname: Teachey
authors.forename: B
authors.initials: B
authors.lastname: Bickert
authors.forename: N
authors.initials: N
authors.lastname: Bunin
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000911:Antibodies, Monoclonal / Q000008:administration & dosage*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D000077561:Daclizumab; D018572:Disease-Free Survival; D004351:Drug Resistance / Q000187:drug effects; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000401:mortality; D006402:Hematologic Diseases / Q000150:complications / Q000378:metabolism / Q000628:therapy*; D006801:Humans; D007074:Immunoglobulin G / Q000008:administration & dosage*; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D007223:Infant; D008297:Male; D012008:Recurrence; D012074:Remission Induction; D033581:Stem Cell Transplantation; D014184:Transplantation, Homologous
chemical_list: D000305:Adrenal Cortex Hormones; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; D000077561:Daclizumab