Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine A in selected children with hypoplastic refractory cytopenia

被引:31
作者
Yoshimi, Ayami [1 ]
Baumann, Irith [2 ]
Fuehrer, Monika [3 ]
Bergstraesser, Eva [4 ]
Goebel, Ulrich [5 ]
Sykora, Karl-Walter [6 ]
Klingebiel, Thomas [7 ]
Gross-Wieltsch, Ute [8 ]
van den Heuvel-Eibrink, Marry M. [9 ]
Fischer, Alexandra [1 ]
Noellke, Peter [1 ]
Niemeyer, Charlotte [1 ]
机构
[1] Univ Freiburg, Dept Pediat & Adolescent Med, Div Pediat Hematol & Oncol, Mathildenstr 1, D-79106 Freiburg, Germany
[2] Klinikum Bayreuth, Inst Pathol, Bayreuth, Germany
[3] Ludwig Maximilians Univ Munchen, Children Hosp, Dr von Haunersches Kinderspital, Dept Pediat Hematol & Oncol, Munich, Germany
[4] Univ Zurich, Univ Childrens Hosp, Zurich, Switzerland
[5] Univ Dusseldorf, Dept Pediat Oncol Hematol & Immunol, Dusseldorf, Germany
[6] Hannover Med Sch, Dept Pediat Hematol Oncol, Hannover, Germany
[7] Goethe Univ Frankfurt, Univ Childrens Hosp, Frankfurt, Germany
[8] Olgaspital, Dept Pediat Hematol & Oncol, Stuttgart, Germany
[9] Erasmus MC, Sophia Childrens Hosp, Rotterdam, Netherlands
关键词
myelodysplastic syndrome; refractory cytopenia; immunosuppressive therapy; anti-thymocyte globulin; children;
D O I
10.3324/haematol.10683
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
It is currently unknown whether immunosuppressive therapy or hematopoietic stem cell transplantation is the most appropriate treatment strategy for children with refractory cytopenia and normal karyotype or trisomy 8. We report on 31 children with hypoplastic refractory cytopenia treated with immunosuppressive therapy consisting of antithymocyte globulin and cyclosporine. At 6 months, 22 of 29 eve luable patients had a complete or partial response; a total of ten patients achieved a complete response at varying time points. Six patients subsequently received a transplant because of non-response, progression to advanced myelodysplastic syndrome or evolution of monosomy 7. Overall and failure-free survival rates at 3 years were 88% and 57%, respectively.
引用
收藏
页码:397 / 400
页数:4
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