title: Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children.
abstract: Patients with severe aplastic anemia (SAA) and an absolute neutrophil count (ANC) of 0 typically have fatal outcomes. We defined fulminant AA (FAA) as ANC = 0 for at least 2 weeks prior to and after immunosuppressive therapy (IST). We analyzed the outcomes of 35 children with FAA among 288 children who enrolled in a prospective study for AA (AA-97 study). AA was classified as FAA (n = 35), very SAA (vSAA; n = 129), or SAA (n = 124). All of the children received the IST with horse anti-thymocyte globulin (ATG) and cyclosporine (CsA). A significantly lower response rate at 6 months was seen in children with FAA when compared to those with vSAA or SAA (40.0, 63.6, and 63.7 %, respectively; p = 0.027). Of 20 nonresponder patients in the FAA group, 11 were rescued by alternative donor transplantation, and 5 patients showed a late response after 6 months. Consequently, no significant difference was noted in overall survival when comparing the FAA, vSAA, and SAA groups (88.5, 95.8, and 96.8 %). These findings indicate that IST with ATG and CsA is justified as a first-line treatment for children with FAA who lack a human leukocyte antigen-matched sibling donor.
authors: Department of Pediatrics, School of Medicine, Nihon University, 30-1 Ohyaguchi-Kamicho, Itabashi-ku, 173-8610, Tokyo, Japan, yagasaki.hiroshi@nihon-u.ac.jp.
authors: Hiroshi
authors: H
authors: Yagasaki
authors: Hiroyuki
authors: H
authors: Shichino
authors: Akira
authors: A
authors: Ohara
authors: Ryoji
authors: R
authors: Kobayashi
authors: Hiromasa
authors: H
authors: Yabe
authors: Shouichi
authors: S
authors: Ohga
authors: Kazuko
authors: K
authors: Hamamoto
authors: Yoshitoshi
authors: Y
authors: Ohtsuka
authors: Hiroyuki
authors: H
authors: Shimada
authors: Masami
authors: M
authors: Inoue
authors: Hideki
authors: H
authors: Muramatsu
authors: Yoshiyuki
authors: Y
authors: Takahashi
authors: Seiji
authors: S
authors: Kojima
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000741:Anemia, Aplastic / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D000818:Animals; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006736:Horses; D006801:Humans; D007165:Immunosuppression Therapy; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D009504:Neutrophils / Q000187:drug effects / Q000276:immunology / Q000473:pathology; D011446:Prospective Studies; D012720:Severity of Illness Index; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine