title: Outcome of adult severe or very severe aplastic anemia treated with immunosuppressive therapy compared with bone marrow transplantation: multicenter trial.
abstract: To compare survival rates and long-term complications after bone marrow transplantation (BMT) or treatment with immunosuppressive agents (ISA) in the management of adult aplastic anemia (AA) and to identify prognostic factors associated with improved survival, we evaluated 229 adult AA patients treated with ISA from 1990 to 2001 and compared the results with those for 64 BMT recipients. Of 156 patients with severe aplastic anemia (SAA) or very severe AA treated with ISA (antithymocyte globulin [ATG] or ATG plus cyclosporine), 46.8% showed complete or partial response and 7.1% had relapses. After long-term follow-up, 1 case each of acute leukemia, myelodysplastic syndrome, and paroxysmal nocturnal hemoglobinuria developed. The 6-year survival rate was 69%. Response to ISA, disease severity, and low absolute neutrophil count (ANC) (< or = 200/mm3) were associated with poor survival. Patient age, sex, initial platelet count, etiology, or treatment regimen did not significantly affect survival. Cox regression analysis showed low ANC to be the only pretreatment variable significantly associated with poor survival (P = .000). Of 64 BMT recipients, 82.8% had sustained engraftment, and 12.5% experienced graft failure. Twenty (31.3%) of the patients developed grade II to IV acute graft-versus-host disease (GVHD), and 12 (18.8%) of the patients developed chronic GVHD. The 6-year survival rate was 79%. Patient age and sex, disease severity, etiology, ANC, initial platelet count, and treatment regimen did not affect survival. Survival of 83 AA patients, aged 14 to 40 years, treated with ISA was not statistically significant from that of 61 adult AA patients who underwent BMT (6-year survival rate, 65% and 79%, respectively). However, BMT in adult AA achieved long-term engraftment and a lower relapse rate than ISA. These results suggest that ISA can achieve a high response rate and long-term survival among patients with adult AA, regardless of disease severity. Further studies with larger numbers of patients and long-term follow-up are needed.
authors.affiliation: Department of Internal Medicine, Hanyang University, Seoul, Korea. ahnmj@hanyang.ac.kr
authors.forename: Myung-Ju
authors.initials: MJ
authors.lastname: Ahn
authors.forename: Jung-Hye
authors.initials: JH
authors.lastname: Choi
authors.forename: Young-Yeul
authors.initials: YY
authors.lastname: Lee
authors.forename: Il-Young
authors.initials: IY
authors.lastname: Choi
authors.forename: In-Soon
authors.initials: IS
authors.lastname: Kim
authors.forename: Sung-Soo
authors.initials: SS
authors.lastname: Yoon
authors.forename: Sun-Yang
authors.initials: SY
authors.lastname: Park
authors.forename: Byung-Kook
authors.initials: BK
authors.lastname: Kim
authors.forename: Cheolwon
authors.initials: C
authors.lastname: Suh
authors.forename: Hee Jeong
authors.initials: HJ
authors.lastname: Son
authors.forename: Cheol-Won
authors.initials: CW
authors.lastname: Jung
authors.forename: Jae Hoon
authors.initials: JH
authors.lastname: Lee
authors.forename: Ju-Myung
authors.initials: JM
authors.lastname: Sung
authors.forename: Suck-Ah
authors.initials: SA
authors.lastname: Im
authors.forename: Doyeun
authors.initials: D
authors.lastname: Oh
authors.forename: So-Young
authors.initials: SY
authors.lastname: Jung
authors.forename: Hwi-Joong
authors.initials: HJ
authors.lastname: Yoon
authors.forename: Kyung-Sam
authors.initials: KS
authors.lastname: Cho
authors.forename: Jung-Ae
authors.initials: JA
authors.lastname: Lee
authors.forename: Young-Jin
authors.initials: YJ
authors.lastname: Yuh
authors.forename: Sung-Rok
authors.initials: SR
authors.lastname: Kim
authors.forename: Moran
authors.initials: M
authors.lastname: Ki
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000401:mortality; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D016026:Bone Marrow Transplantation; D016572:Cyclosporine / Q000008:administration & dosage*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D015999:Multivariate Analysis; D011379:Prognosis; D012008:Recurrence; D012720:Severity of Illness Index; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine