[Treatment of severe aplastic anemia with intensified immunosuppressive therapy and two different regimens with recombinant human granulocyte colony-stimulating factor: a retrospective study based on long-term follow-up].
OBJECTIVE
To compare the efficacy and safety of two different regimens with recombinant human granulocyte colony-stimulating factor (rhG-CSF) combined with intensified immunosuppressive therapy (IIST) in severe aplastic anemia (SAA).


METHODS
Retrospectively analyzed 176 SAA treated with IIST and rhG-CSF in our hospital from March 1994 to December 2007. Regimen A (Group A, n = 96), rhG-CSF 300 µg/d was initiated on day 31 after IIST and subcutaneously administered 1-3 days a week for 3 months. Regimen B (Group B, n = 80), rhG-CSF was initiated at 5 µg·kg(-1)·d(-1) before IIST until hematologic recovery.


RESULTS
(1) The early response rate of Group B (67.5%) was significantly higher than that of Group A (37.5%) (P < 0.01), the interval from IIST to response in Group B was shorter than that in Group A. Moreover, infection-related deaths during first 4 months after IIST were significantly reduced in Group B (6.3%) when compared with Group A (16.7%) (P = 0.034). The cumulative incidence of survival at 4 years in Groups B [(77.7 ± 4.9)%] was also significantly higher than that in Group A [(57.2 ± 5.1)%] (P = 0.006). (2) With regard to 93 refractory patients with no response 4 months after IIST, rhG-CSF therapy was continued in Group B meanwhile stopped in Group A. There were no differences between two groups in terms of survival and the response rates (P = 0.288, 0.066), but there was an increasing risk of evolving into MDS/AML in Group B (22.3%) when compared with Group A (3.71%) (P = 0.023). (3) By multivariate analysis, the severity of disease (P = 0.010, RR = 1.922) and the early response (P < 0.01, RR = 5.749) were associated with the overall survival. Moreover, the number of days of rhG-CSF therapy was the only significant risk factor for SAA evolving into MDS/AML (P = 0.017, RR = 1.004).


CONCLUSIONS
The early initiation of rhG-CSF therapy with proper dose might contribute to a desirable early response and reduced infection-related death rate, but extended administration of rhG-CSF did not improve the long-term outcome of refractory SAA and may further facilitate the progression of SAA into MDS/AML.
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College, Tianjin 300020, China.
Ying-Mei
YM
Li
Xing-Xin
XX
Li
Mei-Li
ML
Ge
Jun
J
Shi
Lin-Sheng
LS
Qian
Jian-Xiang
JX
Wang
Yi-Zhou
YZ
Zheng
D000741:Anemia, Aplastic / Q000628:therapy; D005500:Follow-Up Studies; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D006801:Humans; D007165:Immunosuppression Therapy; D011994:Recombinant Proteins / Q000627:therapeutic use; D012189:Retrospective Studies
D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor