Single-Center Experience: immunosuppressive therapy as frontline treatment for 33 children with acquired severe aplastic anemia.
The authors retrospectively analyzed the records of 33 children with acquired severe aplastic anemia (SAA) diagnosed from July 1998 to October 2007 and first treated by immunosuppressive therapy (IST). Serial hematologic parameters, complications, transfusion requirements, and time to response were assessed. Allogeneic hematopoietic stem cell transplantation (HSCT) was attempted in 7 patients after failure of IST (n = 6) or relapse following an initial response to IST (n = 1). One child died of post-transplant lymphoproliferative disorder. Thirty of the 33 patients are alive and well after a median follow-up of 45 months (range, 7-116 months). Overall (transfusion-independent) response to IST was 73% (24/33). The actuarial 5 years survival rate was 89.4%. In this study, all patients with SAA received IST as standard front-line therapy. Approximately three-fourths of patients with SAA have durable recovery and excellent overall survival.
Huang
I-Anne
IA
Chang Gung Memorial Hospital, Chang Gung University, Taoyuan, Taiwan.
Jaing
Tang-Her
TH
Yang
Chao-Ping
CP
Hung
Iou-Jih
IJ
Tsay
Pei-Kwei
PK
Luo
Chih-Cheng
CC
Sun
Chien-Feng
CF
D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007165:Immunosuppression Therapy; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate; D014184:Transplantation, Homologous