Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience

被引：35

作者：

Beier, R. ^{[1
]}

Schulz, A. ^{[2
]}

Hoenig, M. ^{[2
]}

Eyrich, M. ^{[3
]}

Schlegel, P-G ^{[3
]}

Holter, W. ^{[4
]}

Stachel, K. D. ^{[5
]}

Ehlert, K. ^{[6
]}

Greil, J. ^{[7
]}

Nuernberger, W. ^{[8
]}

Woessmann, W. ^{[8
]}

Bader, P. ^{[9
]}

Urban, C. ^{[10
]}

Mueller, I. ^{[11
]}

Suttorp, M. ^{[12
]}

Sauer, M. ^{[1
]}

Gruhn, B. ^{[13
]}

Meisel, R. ^{[14
]}

Zimmermann, M. ^{[1
]}

Sykora, K-W ^{[1
]}

机构：

[1] Hannover Med Sch, Dept Pediat Hematol & Oncol, D-30625 Hannover, Germany

[2] Univ Ulm, Sect Stem Cell Transplantat, Dept Pediat & Adolescent Med, D-89069 Ulm, Germany

[3] Univ Wurzburg, Childrens Hosp, Pediat Stem Cell Transplantat Program, D-97070 Wurzburg, Germany

[4] St Anna Childrens Hosp, Vienna, Austria

[5] Univ Hosp Erlangen, Childrens & Adolescents Hosp, Sect Oncol Haematol & Cell Therapy, Erlangen, Germany

[6] Univ Childrens Hosp Munster, Dept Paediat Haematol Oncol, Munster, Germany

[7] Univ Heidelberg Hosp, Dept Oncol Haematol Immunol & Pneumonol, Ctr Child & Adolescent Med, Heidelberg, Germany

[8] Univ Hosp Giessen & Marburg, Childrens Hosp Giessen, Dept Paediat Haematol Oncol, Giessen, Germany

[9] Goethe Univ Frankfurt Main, Ctr Child & Adolescent Med 3, Sect Stem Cell Transplantat, Frankurt Main, Germany

[10] Graz Med Univ, Clin Dept Pediat Haematol Oncol, Graz, Austria

[11] Univ Childrens Hosp Tubingen, Dept Gen Pediat Hematol & Oncol, Tubingen, Germany

[12] Univ Hosp Carl Gustav Carus, Div Haematol & Oncol, Dept Childrens & Adolescent Med, Dresden, Germany

[13] Univ Jena, Dept Pediat, Jena Univ Hosp, Jena, Germany

[14] Univ Dusseldorf, Ctr Child & Adolescent Hlth, Dept Pediat Oncol Hematol & Immunol, D-40225 Dusseldorf, Germany

来源：

BONE MARROW TRANSPLANTATION | 2013年 / 48卷 / 04期

关键词：

treosulfan; haematopoietic SCT; children; inborn errors of metabolism; leukaemia; long-term follow-up; STEM-CELL TRANSPLANTATION; HIGH-DOSE TREOSULFAN; FLUDARABINE; REGIMEN; DISEASE; CYCLOPHOSPHAMIDE; DIAGNOSIS; BUSULFAN;

D O I：

10.1038/bmt.2012.188

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

We report the long-term follow-up of children transplanted with Treosulfan (TREO)-based conditioning in Germany and Austria. Nine centres reported a total of 109 transplantations. Patients were stratified according to the paediatric TRM risk score derived from the paediatric BMT registry (PRST) and compared with the historical transplant population of this registry. Underlying diseases were malignancies, immunodeficiencies, and haematologic and metabolic disorders. TREO total dose ranged from 21-42 g/m(2). Additional conditioning drugs included fludarabine, thiotepa, melphalan, CY and/or TBI. EFS at 3 years for non-malignant and malignant diseases was 88% and 49%, respectively. Leukaemia patients in remission had a survival of 51% at 3 years; nonremission patients relapsed and died within 18 months. TRM and OS in the low-risk groups 0 and 1 were similar to PRST controls. TRM in the high-risk groups 2 and 3 was markedly lower (9% vs 28% and 13% vs 53%, respectively) than in the PRST group, but OS was similar. In conclusion, TREO-based conditioning regimens in children resulted in excellent engraftment and long-term survival in nonmalignant disease. In high-risk malignancy, low acute toxicity was followed by low TRM but it did not translate into increased survival. Bone Marrow Transplantation (2013) 48, 491-501; doi: 10.1038/bmt.2012.188; published online 22 October 2012

引用

页码：491 / 501

页数：11

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