PortalPublikationenLong-term follow-up of children conditioned with Treosulfan:...

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

Standard

Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience. / Beier, R; Schulz, A; Hönig, M; Eyrich, M; Schlegel, P-G; Holter, W; Stachel, K D; Ehlert, K; Greil, J; Nürnberger, W; Wössmann, W; Bader, P; Urban, C; Müller, I; Suttorp, M; Sauer, M; Gruhn, B; Meisel, R; Zimmermann, M; Sykora, K-W.

in: BONE MARROW TRANSPL, Jahrgang 48, Nr. 4, 01.04.2013, S. 491-501.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Beier, R, Schulz, A, Hönig, M, Eyrich, M, Schlegel, P-G, Holter, W, Stachel, KD, Ehlert, K, Greil, J, Nürnberger, W, Wössmann, W, Bader, P, Urban, C, Müller, I, Suttorp, M, Sauer, M, Gruhn, B, Meisel, R, Zimmermann, M & Sykora, K-W 2013, 'Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience', BONE MARROW TRANSPL, Jg. 48, Nr. 4, S. 491-501. https://doi.org/10.1038/bmt.2012.188

APA

Beier, R., Schulz, A., Hönig, M., Eyrich, M., Schlegel, P-G., Holter, W., Stachel, K. D., Ehlert, K., Greil, J., Nürnberger, W., Wössmann, W., Bader, P., Urban, C., Müller, I., Suttorp, M., Sauer, M., Gruhn, B., Meisel, R., Zimmermann, M., & Sykora, K-W. (2013). Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience. BONE MARROW TRANSPL, 48(4), 491-501. https://doi.org/10.1038/bmt.2012.188

Vancouver

Beier R, Schulz A, Hönig M, Eyrich M, Schlegel P-G, Holter W et al. Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience. BONE MARROW TRANSPL. 2013 Apr 1;48(4):491-501. https://doi.org/10.1038/bmt.2012.188

Bibtex

@article{aa58ef2759e241089db99a6ee70addff,
title = "Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience",
abstract = "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.",
keywords = "Adolescent, Adult, Antineoplastic Agents, Alkylating, Austria, Bone Marrow Transplantation, Busulfan, Child, Child, Preschool, Common Variable Immunodeficiency, Disease-Free Survival, Female, Follow-Up Studies, Germany, Humans, Infant, Male, Metabolism, Inborn Errors, Myeloablative Agonists, Neoplasms, Registries, Risk Factors, Survival Rate, Transplantation Conditioning",
author = "R Beier and A Schulz and M H{\"o}nig and M Eyrich and P-G Schlegel and W Holter and Stachel, {K D} and K Ehlert and J Greil and W N{\"u}rnberger and W W{\"o}ssmann and P Bader and C Urban and I M{\"u}ller and M Suttorp and M Sauer and B Gruhn and R Meisel and M Zimmermann and K-W Sykora",
year = "2013",
month = apr,
day = "1",
doi = "10.1038/bmt.2012.188",
language = "English",
volume = "48",
pages = "491--501",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "4",

}

RIS

TY - JOUR

T1 - Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience

AU - Beier, R

AU - Schulz, A

AU - Hönig, M

AU - Eyrich, M

AU - Schlegel, P-G

AU - Holter, W

AU - Stachel, K D

AU - Ehlert, K

AU - Greil, J

AU - Nürnberger, W

AU - Wössmann, W

AU - Bader, P

AU - Urban, C

AU - Müller, I

AU - Suttorp, M

AU - Sauer, M

AU - Gruhn, B

AU - Meisel, R

AU - Zimmermann, M

AU - Sykora, K-W

PY - 2013/4/1

Y1 - 2013/4/1

N2 - 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.

AB - 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.

KW - Adolescent

KW - Adult

KW - Antineoplastic Agents, Alkylating

KW - Austria

KW - Bone Marrow Transplantation

KW - Busulfan

KW - Child

KW - Child, Preschool

KW - Common Variable Immunodeficiency

KW - Disease-Free Survival

KW - Female

KW - Follow-Up Studies

KW - Germany

KW - Humans

KW - Infant

KW - Male

KW - Metabolism, Inborn Errors

KW - Myeloablative Agonists

KW - Neoplasms

KW - Registries

KW - Risk Factors

KW - Survival Rate

KW - Transplantation Conditioning

U2 - 10.1038/bmt.2012.188

DO - 10.1038/bmt.2012.188

M3 - SCORING: Journal article

C2 - 23085832

VL - 48

SP - 491

EP - 501

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 4

ER -