title: Etanercept as a salvage treatment for refractory aplastic anemia.
abstract: About 10-15% of patients with acquired aplastic anemia (AAA) have resistant/recurrent disease not eligible for standard treatment like hematopoietic stem cell transplantation and/or combined immunosuppression. We report a 17-year-old male with an 11 years history of AAA who, after two courses of immunosuppression, was red cell transfusion-dependent, severely thrombocytopenic, refractory to platelet transfusion, had iron overload and post-transfusion HCV infection. This patient achieved transfusion independence from platelets and normalized Hb after treatment with the anti-TNF agent Etanercept. Over a 12 months follow-up he experienced only transient increase of liver transaminases.
authors.affiliation: Haematology Unit, G.Gaslini Children's Hospital, Genova, Italy. carlodufour@ospedale-gaslini.ge.it
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
authors.forename: Raffaella
authors.initials: R
authors.lastname: Giacchino
authors.forename: Pietro
authors.initials: P
authors.lastname: Ghezzi
authors.forename: Rossella
authors.initials: R
authors.lastname: Tonelli
authors.forename: Elisa
authors.initials: E
authors.lastname: Ferretti
authors.forename: A
authors.initials: A
authors.lastname: Pitto
authors.forename: Vito
authors.initials: V
authors.lastname: Pistoia
authors.forename: Tiziana
authors.initials: T
authors.lastname: Lanza
authors.forename: Johanna
authors.initials: J
authors.lastname: Svahn
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000503:physiopathology; D000753:Anemia, Refractory / Q000188:drug therapy* / Q000503:physiopathology; D002986:Clinical Trials as Topic; D000068800:Etanercept; D006526:Hepatitis C / Q000150:complications; D006801:Humans; D007074:Immunoglobulin G / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D018124:Receptors, Tumor Necrosis Factor / Q000627:therapeutic use*; D016879:Salvage Therapy / Q000379:methods*
chemical_list: D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; D018124:Receptors, Tumor Necrosis Factor; D000068800:Etanercept