Immunosuppressive therapy for aplastic anemia in children:: a more severe disease predicts better survival

被引：139

作者：

Führer, M

Rampf, U

Baumann, I

Faldum, A

Niemeyer, C

Janka-Schaub, G

Friedrich, W

Ebell, W

Borkhardt, A

Bender-Goetze, C

机构：

[1] Univ Munich, Childrens Univ Hosp, Dept Hematol & Oncol, Munich, Germany

[2] Univ Erlangen Nurnberg, Dept Pathol, Erlangen, Germany

[3] Univ Mainz, Inst Med Biometrie Epidemiol & Informat, D-6500 Mainz, Germany

[4] Univ Freiburg, Dept Pediat & Adolescent Med, Div Pediat Hematol & Oncol, Freiburg, Germany

[5] Childrens Univ Hosp, Dept Hematol & Oncol, Hamburg, Germany

[6] Univ Ulm, Dept Pediat, D-7900 Ulm, Germany

[7] Humboldt Univ, Charite, Dept Pediat, Berlin, Germany

来源：

BLOOD | 2005年 / 106卷 / 06期

关键词：

D O I：

10.1182/blood-2005-03-0874

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Autologous T lymphocytes are thought to cause bone marrow failure by immune-mediated excessive apoptosis of stem and progenitor cells. The disease is subclassified into a severe (neutrophil count, > 0.2 x 10(9)/L [>200/mu L]) and a very severe (< 0.2 x 10(9)/L [< 200/mu L]) (vSAA) form. We report the results of a prospective multicenter trial with a combined immunosuppressive regimen of cyclosporin A (CSA), anti-thymocyte globulin (ATG) and, in cases with neutrophil counts fewer than 0.5 x 10(9)/L (< 500/mu L), granulocyte colony-stimulating factor (G-CSF) for treatment of SAA in children. Children with vSAA showed a children with SAA (68% versus 45%; P = .009), as well as better survival (93% versus 81%; P < .001). Thus, in children with SAA a more severe disease stage at diagnosis indicates a favorable outcome with immunosuppressive therapy.

引用

页码：2102 / 2104

页数：3

共 24 条

[11] Risk factors for evolution of acquired aplastic anemia into myelodysplastic syndrome and acute myeloid leukemia after immunosuppressive therapy in children [J].

Kojima, S ;

Ohara, A ;

Tsuchida, M ;

Kudoh, T ;

Hanada, R ;

Okimoto, Y ;

Kaneko, T ;

Takano, T ;

Ikuta, K ;

Tsukimoto, I .

BLOOD, 2002, 100 (03) :786-790

[12]

Kojima S, 2000, BLOOD, V96, P2049

[13]

LOCASCIULLI A, 1990, BONE MARROW TRANSPL, V6, P211

[14]

Maciejewski JP, 2000, AM J HEMATOL, V65, P123, DOI 10.1002/1096-8652(200010)65:2<123::AID-AJH6>3.0.CO

[15]

2-M

[16] Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: A report from the European blood and marrow transplant (EBMT) severe aplastic anaemia working party [J].

Marsh, J ;

Schrezenmeier, H ;

Marin, P ;

Ilhan, O ;

Ljungman, P ;

McCann, S ;

Socie, G ;

Tichelli, A ;

Passweg, J ;

Hows, J ;

Raghavachar, A ;

Locasciulli, A ;

Bacigalupo, A .

BLOOD, 1999, 93 (07) :2191-2195

[17] In-vivo dominant immune responses in aplastic anaemia:: molecular tracking of putatively pathogenetic T-cell clones by TCR β-CDR3 sequencing [J].

Risitano, AM ;

Maciejewski, JP ;

Green, S ;

Plasilova, M ;

Zeng, WH ;

Young, NS .

LANCET, 2004, 364 (9431) :355-364

[18] Antithymocyte globulin and cyclosporine for severe aplastic anemia - Association between hematologic response and long-term outcome [J].

Rosenfeld, S ;

Follmann, D ;

Nunez, O ;

Young, NS .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2003, 289 (09) :1130-1135

[19] Fas-mediated apoptosis is important in regulating cell replication and death in trisomy 8 hematopoietic cells but not in cells with other cytogenetic abnormalities [J].

Sloand, EM ;

Kim, S ;

Fuhrer, M ;

Risitano, AM ;

Nakamura, R ;

Maciejewski, JP ;

Barrett, AJ ;

Young, NS .

BLOOD, 2002, 100 (13) :4427-4432

[20]

Socié G, 2000, SEMIN HEMATOL, V37, P91

← 1 2 3 →