title: Anti-CD52 antibody-mediated immune ablation with autologous immune recovery for the treatment of refractory juvenile polymyositis.
abstract: Autologous hematopoietic stem cell transplantation (HSCT) has been used for the treatment of both adult and pediatric autoimmune diseases. However, HSCT has significant side effects (neutropenia, thrombocytopenia, infertility, cardiotoxicity) and costs (HSC collection/harvesting, blood product support). In an attempt to avoid the toxicities and costs associated with HSCT, we investigated whether immune ablation similar to that achieved following myeloablative HSCT could be achieved by the intensive administration of an anti-CD52 antibody (Campath-1H antibody). The first patient treated with the treatment regime, who had refractory juvenile polymyositis, achieved immune ablation (the elimination of pre-therapy antigen-specific T lymphocyte immunity) and has had stable clinical improvement for more than 6 years.
authors.affiliation: Division of Rheumatology, Childrens Hospital Los Angeles (CHLA), Los Angeles, CA 90027, USA. areiff@chla.usc.edu
authors.forename: Andreas
authors.initials: A
authors.lastname: Reiff
authors.forename: Bracha
authors.initials: B
authors.lastname: Shaham
authors.forename: Kenneth I
authors.initials: KI
authors.lastname: Weinberg
authors.forename: Gay M
authors.initials: GM
authors.lastname: Crooks
authors.forename: Robertson
authors.initials: R
authors.lastname: Parkman
mesh_terms: D000293:Adolescent; D000074323:Alemtuzumab; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000276:immunology / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use; D000912:Antibodies, Neoplasm / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use; D015703:Antigens, CD / Q000276:immunology*; D000951:Antigens, Neoplasm / Q000276:immunology*; D001327:Autoimmune Diseases / Q000276:immunology / Q000628:therapy; D000074301:CD52 Antigen; D005260:Female; D006023:Glycoproteins / Q000276:immunology*; D006801:Humans; D017285:Polymyositis / Q000276:immunology / Q000628:therapy*
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D015703:Antigens, CD; D000951:Antigens, Neoplasm; D000074301:CD52 Antigen; C000613862:CD52 protein, human; D006023:Glycoproteins; D000074323:Alemtuzumab