Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT)

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Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). / Locasciulli, Anna; Oneto, Rosi; Bacigalupo, Andrea; Socie, Gerard; Korthof, Elisabeth; Békássy, Albert; Schrezenmeier, Hubert; Passweg, Jakob; Fuhrer, MoniKa; of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), on the behalf of the Severe Aplastic Anemia Working Party.

In: Haematologica, Vol. 92, No. 1, 2007, p. 11-18.

Research output: Contribution to journalArticle

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Locasciulli, A, Oneto, R, Bacigalupo, A, Socie, G, Korthof, E, Békássy, A, Schrezenmeier, H, Passweg, J, Fuhrer, M & of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), OTBOTSAAWP 2007, 'Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT)', Haematologica, vol. 92, no. 1, pp. 11-18. https://doi.org/10.3324/haematol.10075

APA

Locasciulli, A., Oneto, R., Bacigalupo, A., Socie, G., Korthof, E., Békássy, A., ... of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), O. T. B. O. T. S. A. A. W. P. (2007). Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica, 92(1), 11-18. https://doi.org/10.3324/haematol.10075

CBE

Locasciulli A, Oneto R, Bacigalupo A, Socie G, Korthof E, Békássy A, Schrezenmeier H, Passweg J, Fuhrer M, of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT) OTBOTSAAWP. 2007. Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica. 92(1):11-18. https://doi.org/10.3324/haematol.10075

MLA

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Locasciulli, Anna ; Oneto, Rosi ; Bacigalupo, Andrea ; Socie, Gerard ; Korthof, Elisabeth ; Békássy, Albert ; Schrezenmeier, Hubert ; Passweg, Jakob ; Fuhrer, MoniKa ; of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), on the behalf of the Severe Aplastic Anemia Working Party. / Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). In: Haematologica. 2007 ; Vol. 92, No. 1. pp. 11-18.

RIS

TY - JOUR

T1 - Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT)

AU - Locasciulli, Anna

AU - Oneto, Rosi

AU - Bacigalupo, Andrea

AU - Socie, Gerard

AU - Korthof, Elisabeth

AU - Békássy, Albert

AU - Schrezenmeier, Hubert

AU - Passweg, Jakob

AU - Fuhrer, MoniKa

AU - of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), on the behalf of the Severe Aplastic Anemia Working Party

PY - 2007

Y1 - 2007

N2 - BACKGROUND AND OBJECTIVES: The treatment of acquired aplastic anemia (AA) is based on allogeneic bone marrow transplantation (BMT) and immunosuppressive therapy. The aim of this study was to assess the outcome of children and adults with AA treated in the last decade, and to determine whether results have improved in two sequential time periods,1991-1996 and 1997-2002. DESIGN AND METHODS: Two-thousands and seventy-nine consecutive patients with AA, classified according to first-line treatment: BMT (n=1567) or immunosuppressive therapy (n= 912), the patients for the two sequential time periods were studied. Analyses included variables related to patients, disease and transplant. RESULTS: The actuarial 10-year survival was 73% and 68% for BMT or immunosuppressive treatment, respectively (p=0.002). BMT outcome improved significantly with time (69% and 77%, p=001) for both matched sibling donor (MSD) (74% and 80%; p=0.003 ), alternative donor (38% and 65% p=0.0001), and was better in children (79% versus 68%, p<0.0001). Multivariate analysis: favorable predictors (p<0.001) were younger age, transplant beyond 1996, MSD, a short interval diagnosis-transplant , no irradiation. IS: no significant improvement over time (69% and 73% p=0.29). Survival was significantly better in children (81% versus 70%, p=0.001), especially in vSAA(83% versus 62%, p=0.0002). Combined IS was superior to single drug treatment (77% versus 62%, p=0.002). Multivariate analysis: significant predictors of survival: age > or =16 years (p=0.0009), longer interval between diagnosis -treatment (p=0.04), single drug versus combined IS (p=0.02). INTERPRETATION AND CONCLUSIONS: Outcome has improved in subsets of AA patients: those receiving first- line BMT and children with vSAA treated with IS. Age remains a major predictor for both treatments. Early intervention is associated with a significantly better outcome and is strongly recommended, whatever the first-line therapy.

AB - BACKGROUND AND OBJECTIVES: The treatment of acquired aplastic anemia (AA) is based on allogeneic bone marrow transplantation (BMT) and immunosuppressive therapy. The aim of this study was to assess the outcome of children and adults with AA treated in the last decade, and to determine whether results have improved in two sequential time periods,1991-1996 and 1997-2002. DESIGN AND METHODS: Two-thousands and seventy-nine consecutive patients with AA, classified according to first-line treatment: BMT (n=1567) or immunosuppressive therapy (n= 912), the patients for the two sequential time periods were studied. Analyses included variables related to patients, disease and transplant. RESULTS: The actuarial 10-year survival was 73% and 68% for BMT or immunosuppressive treatment, respectively (p=0.002). BMT outcome improved significantly with time (69% and 77%, p=001) for both matched sibling donor (MSD) (74% and 80%; p=0.003 ), alternative donor (38% and 65% p=0.0001), and was better in children (79% versus 68%, p<0.0001). Multivariate analysis: favorable predictors (p<0.001) were younger age, transplant beyond 1996, MSD, a short interval diagnosis-transplant , no irradiation. IS: no significant improvement over time (69% and 73% p=0.29). Survival was significantly better in children (81% versus 70%, p=0.001), especially in vSAA(83% versus 62%, p=0.0002). Combined IS was superior to single drug treatment (77% versus 62%, p=0.002). Multivariate analysis: significant predictors of survival: age > or =16 years (p=0.0009), longer interval between diagnosis -treatment (p=0.04), single drug versus combined IS (p=0.02). INTERPRETATION AND CONCLUSIONS: Outcome has improved in subsets of AA patients: those receiving first- line BMT and children with vSAA treated with IS. Age remains a major predictor for both treatments. Early intervention is associated with a significantly better outcome and is strongly recommended, whatever the first-line therapy.

KW - acquired aplastic anemia

KW - immunosuppression

KW - BMT

U2 - 10.3324/haematol.10075

DO - 10.3324/haematol.10075

M3 - Article

VL - 92

SP - 11

EP - 18

JO - Haematologica-The Hematology Journal

T2 - Haematologica-The Hematology Journal

JF - Haematologica-The Hematology Journal

SN - 1592-8721

IS - 1

ER -