Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children

• Published in: Annals of hematology Vol. 93; no. 5; pp. 747 - 752
• Main Authors: Yagasaki, Hiroshi, Shichino, Hiroyuki, Ohara, Akira, Kobayashi, Ryoji, Yabe, Hiromasa, Ohga, Shouichi, Hamamoto, Kazuko, Ohtsuka, Yoshitoshi, Shimada, Hiroyuki, Inoue, Masami, Muramatsu, Hideki, Takahashi, Yoshiyuki, Kojima, Seiji
• Format: Journal Article
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.05.2014; Springer Nature B.V
• Subjects: Acute Disease; Adolescent; Anemia, Aplastic - immunology; Anemia, Aplastic - mortality; Anemia, Aplastic - pathology; Anemia, Aplastic - therapy; Animals; Antilymphocyte Serum - therapeutic use; Child; Child, Preschool; Cyclosporine - therapeutic use; Female; Hematology; Hematopoietic Stem Cell Transplantation; Histocompatibility Testing; Horses; Humans; Immunosuppression; Immunosuppressive Agents - therapeutic use; Male; Medicine; Medicine & Public Health; Neutrophils - drug effects; Neutrophils - immunology; Neutrophils - pathology; Oncology; Original Article; Prospective Studies; Severity of Illness Index; Survival Analysis; Treatment Outcome; Fulminant aplastic anemia; Immunosuppressive therapy; Children; Cyclosporine; Horse anti-thymocyte globulin
• ISSN: 0939-5555; 1432-0584
• DOI: 10.1007/s00277-013-1984-x

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.