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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 LU ; Schrezenmeier, Hubert; Passweg, Jakob; Fuhrer, MoniKa and of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), on the behalf of the Severe Aplastic Anemia Working Party (2007) In Haematologica 92(1). p.11-18
Abstract
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... (More)
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. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
acquired aplastic anemia, immunosuppression, BMT
in
Haematologica
volume
92
issue
1
pages
11 - 18
publisher
Ferrata Storti Foundation
external identifiers
  • pmid:17229630
  • scopus:33846898991
ISSN
1592-8721
DOI
10.3324/haematol.10075
language
English
LU publication?
yes
id
10808b76-574f-45f2-b7d4-8c775b7f0eb8 (old id 1138480)
date added to LUP
2008-08-19 11:50:55
date last changed
2017-11-19 04:10:37
@article{10808b76-574f-45f2-b7d4-8c775b7f0eb8,
  abstract     = {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&lt;0.0001). Multivariate analysis: favorable predictors (p&lt;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 &gt; 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.},
  author       = {Locasciulli, Anna and Oneto, Rosi and Bacigalupo, Andrea and Socie, Gerard and Korthof, Elisabeth and Békássy, Albert and Schrezenmeier, Hubert and Passweg, Jakob and Fuhrer, MoniKa and of the European Group for Blood and Marrow Transplantation (SAA-WP, BMT), on the behalf of the Severe Aplastic Anemia Working Party},
  issn         = {1592-8721},
  keyword      = {acquired aplastic anemia,immunosuppression,BMT},
  language     = {eng},
  number       = {1},
  pages        = {11--18},
  publisher    = {Ferrata Storti Foundation},
  series       = {Haematologica},
  title        = {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)},
  url          = {http://dx.doi.org/10.3324/haematol.10075},
  volume       = {92},
  year         = {2007},
}