Chinese Medical E-ournals Database

Chinese Journal of Obstetrics & Gynecology and Pediatrics(Electronic Edition) ›› 2009, Vol. 05 ›› Issue (04): 338 -343. doi: 10.3877/cma.j.issn.1673-5250.2009.04.102

Original Article

Clinical Analysis of the Efficacy of Intensive Immunosuppressive Therapy in 68 Children With Severe Aplastic Anemia

Chun CHEN, Ying-fei LI, Jian-pei FANG, Hong-man XUE, Dun-hua ZHOU, Ke HUANG, Hong-gui XU, Hai-xia GUO, Shao-liang HUANG   

  1. Department of Pediatrics, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou 510120, China
  • Published:2009-08-01
  • Supported by:
    * Project No. 30772367, supported by the National Natural Science Foundation of China
Objective

To analyze the therapeutic efficacy of intensive immunosuppressive therapy(IST) in children with severe aplastic anemia(SAA) and compared it with only cyclosporine A(CSA) therapy, and evaluate the relation between granulocyte colony stimulating factor (G-CSF) reaction and therapeutic efficacy before intensive immunosuppressive therapy.

Methods

The study cohort consisted of 68 consecutive patients diagnosed as severe aplastic anemia in the Department of Pediatrics, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University between January 2000 and November 2007. Fifty-five cases of 68 patients accepted intensive immunosuppressive group (IST group), while other 13 cases received only cyclosporine A (CSA group) because of poor economy. The different therapeutic efficacy of these two regimens was analyzed. The procedure of this study was consistent with ethical standard established by the committee of investigation in human beings of Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, and it was approved by this committee. Informed consent was obtained from all participates.24 cases of severe aplastic anemia received granulocyte colony stimulating factor as experimental treatment before intensive immunosuppressive therapy according to the degree of peripheral blood count's response to granulocyte colony stimulating factor. These 24 cases were divided into reaction sub-IST group (n=13)and non-reaction sub-IST group.(n=11). The differences of therapeutic efficacy at the end of 6 months after treatment and long-term effects were observed.

Results

The average effective time of IST group and CSA group after treatment were 3.4 months(1~13 months) and 4.5 months (1~15 months), respectively. The efficacy rates of IST group and CSA group at the end of 1st, 3rd, 6th, 9th and 12th months after treatment were 70.9%, 58.2%, 79.5%, 87.2% and 89.7% vs. 46.2%, 38.5%, 46.2%, 61.5% and 61.5%, respectively. More than 1 year follow-up of 39 patients in IST group, the total efficacy rate was 89.7%(35/39). More than 1 year follow-up of 13 patients in CSA group, the total efficacy rate was 61.5%(8/13). There had statistically significant difference between two groups(χ2=5.419, P<0.05). The efficacy rates of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor at the end of 6th months after treatment were 76.9% and 45.6%, respectively.There had no significant difference between two groups (P>0.05). The total efficacy rates of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor were 92.3%, (12/13)and 54.5%(6/11), respectively.The former was significant higher than that of the latter. There was statistically significant difference in these two groups(P<0.05). The efficacy of intensive immunosuppressive therapy was better than that of single-use cyclosporine A. The average effective time of IST group and CSA group after treatment were 3.4 months and 4.5 months, respectively. The efficacy of IST group and CSA group at 1 year after treatment were 89.7% and 61.5%, respectively. The total efficacy rate of reaction sub-IST group and non-reaction sub-IST group to granulocyte colony stimulating factor were 92.3% and 54.5%, respectively.

Conclusion

The therapeutic efficacy of intensive immunosuppressive therapy in children with severe aplastic anemia is better than that of cyclosporine A therapy only. Children with severe aplastic anemia who responded well to granulocyte colony stimulating factor had a better prognosis in the intensive immunosuppressive therapy.

1 Bacigalupo A, Hows J, Gordon–Smith EC, et al. Bone marrow transplantation for severe aplastic anemia from donors other than HLA identical siblings: A report of the BMT Working Party. Bone Marrow Transplant,1988,3(6):531–535.
2 Camitta BM, Thomas ED, Nathan DG, et al. Severe aplastic anemia: A prospective study of the effect of early marrow transplantion on acute mortality.Blood,1976,48(1):63–70.
3 Chen C, Fang JP, Huang SL,et al.The clinical analysis of the efficacy of immunosuppressive therapy in 54 children patients with severe aplastic anemia. Chin J Pediatr.2006,44(11):841–844.
4 Stary J,Locatelli F,Niemeyer CM,et al.Stem cell transplantation for aplastic anemia and myelodysplastic syndrome.Bone Marrow Transplant, 2005,35(suppl 1):S13–S16.
5 Further M,Rampf U,Baumann I,et al.Immunosuppressive therapy for aplasic anemia in children:A more severe disease predicts better survival.Blood,2005,106(6):2102–2104.
6 Jeng MR,Naidu PE,Rieman MD,et al.Granulocyte–macrophage colony stimulating factor and immunosuppression in the treatment of pediatric aquired severe aplastic anemia. Pediatr Blood Cancer, 2005, 45(2): 170–175.
7 Killick SB,Marsh JC,Gordon–Smith EC,et al.Long–term outcome of aquired aplastic anemia in children:Comparison between immunosuppressive therapy and bone marrow transplantation.Br J Haematol, 2000, 111: 321–328.
8 Goldenberg NA,Graham DK,Liang X,et al.Successful treatment of severe aplastic anemia in children using standardized immunosuppressive therapy with antithymocyte globulin and cyclosporine A.Pediatr Blood Cancer, 2004,43(7):718–722.
9 Kojima S,Hibi S,Kosaka Y,et al.Immunosuppressive therapy using antithymocyte globulin,cyclosporine, and danazol with or without human granulocyte colony–stimulating factor in children with acquired apastic anemia.Blood,2000,96(6):2049–2054.
10 Scheinberg P, Wu CO, Nunez O, et al.Long–term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine.J Pediatr,2008,153(6):814–819.
11 Lu J,Chai YH, Cao YF,et al. The clinical prophase observation of the therapeutic effect of antilymphocyte globulin combining cyclosporin A in treatment of pediatric severe aplastic anemia.China Pediatr Blood,2005, 10(3):103–105.
12 Futher M,Burdach S,Ebell W,et al.Relapse and clonal disease in chidren with aplastic anemia after immunosuppressive therapy(IST):The SAA 94 experience.German/Austrian Pediatric Aplastic Anemia Working Group. Klin Pediatr,1998,210(4):173–179.
13 De–Medeiros CR,Ribeiro RC,Bittencourt MA.Long–term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin.Braz J Med Biol Res,2000,33(5):553–558.
14 Socie G, Mary JY, Schrezenmeier H, et al. Granulocyte–stimulating factor and severe aplastic anemia: A survey by the European Group for Blood and Marrow Transplantation (EBMT).Blood,2007 109(7): 2794–2796.
15 Shichino H,Mugishima H,Takamura M,et al.Treatment of aplastic anemia with antithymocyte globulin,cyclosporin A,methylprednisolone, danazol and recombinant human grannlocyte–colony stimulating factor. Acta Paediatr Jpn,1996,38(6):644–647.
16 Huang YL, Huang SL.Progress of immune–mediated pathogenesis and treatment in children patients with aplastic anemia. Chin J Pediatr,2006,44(2):153–155.
17 Yong NS, Maciejewski JP. Aplastic anemia. In: Ronald H. Hematology–basic principles and practice.3rd ed.Beijing :Harcourt Asia Churchill Livingstone,2001,297–331.
18 Mourad G, Garrigue V, Squifflet JP, et al. Induction versus moninduction in renal transplant recipients with tacrolimus–based immunosuppression. Transplantation,2001,72(6):1050–1055.
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