Etanercept as a salvage treatment for refractory aplastic anemia.
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.
Dufour
Carlo
C
Haematology Unit, G.Gaslini Children's Hospital, Genova, Italy. carlodufour@ospedale-gaslini.ge.it
Giacchino
Raffaella
R
Ghezzi
Pietro
P
Tonelli
Rossella
R
Ferretti
Elisa
E
Pitto
A
A
Pistoia
Vito
V
Lanza
Tiziana
T
Svahn
Johanna
J
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*
D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; D018124:Receptors, Tumor Necrosis Factor; D000068800:Etanercept