Outcome of adult severe or very severe aplastic anemia treated with immunosuppressive therapy compared with bone marrow transplantation: multicenter trial.
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.
Ahn
Myung-Ju
MJ
Department of Internal Medicine, Hanyang University, Seoul, Korea. ahnmj@hanyang.ac.kr
Choi
Jung-Hye
JH
Lee
Young-Yeul
YY
Choi
Il-Young
IY
Kim
In-Soon
IS
Yoon
Sung-Soo
SS
Park
Sun-Yang
SY
Kim
Byung-Kook
BK
Suh
Cheolwon
C
Son
Hee Jeong
HJ
Jung
Cheol-Won
CW
Lee
Jae Hoon
JH
Sung
Ju-Myung
JM
Im
Suck-Ah
SA
Oh
Doyeun
D
Jung
So-Young
SY
Yoon
Hwi-Joong
HJ
Cho
Kyung-Sam
KS
Lee
Jung-Ae
JA
Yuh
Young-Jin
YJ
Kim
Sung-Rok
SR
Ki
Moran
M
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
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine