Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum

M. Van Steekelenburg; M. H. Van Weel-Sipman; A. H. Zwinderman; P. M. Hoogerbrugge; J. M.J.J. Vossen; R. M. Egeler

Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum

Translated title of the contribution: Favourable current prognosis after HLA-identical bone marrow transplantation for children with acquired severe aplastic anaemia; evaluation of 30 years of bone marrow transplantation at the Leiden University Medical Center

M. Van Steekelenburg, M. H. Van Weel-Sipman, A. H. Zwinderman, P. M. Hoogerbrugge, J. M.J.J. Vossen, R. M. Egeler

Research output: Contribution to journal › Article › peer-review

2 Citations (Scopus)

Abstract

Objective. To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia. Design. Retrospective, descriptive. Method. Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graftversus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin). Results. There was an increase in the 1-year actuarial survival rate from 67% in the period before 1989 to 90% thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001). Conclusion. The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90%.

Translated title of the contribution	Favourable current prognosis after HLA-identical bone marrow transplantation for children with acquired severe aplastic anaemia; evaluation of 30 years of bone marrow transplantation at the Leiden University Medical Center
Original language	Dutch
Pages (from-to)	1542-1546
Number of pages	5
Journal	Nederlands Tijdschrift voor Geneeskunde
Volume	146
Issue number	33
Publication status	Published - 17 Aug 2002
Externally published	Yes

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Van Steekelenburg, M., Van Weel-Sipman, M. H., Zwinderman, A. H., Hoogerbrugge, P. M., Vossen, J. M. J. J., & Egeler, R. M. (2002). Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum. Nederlands Tijdschrift voor Geneeskunde, 146(33), 1542-1546.

Van Steekelenburg, M. ; Van Weel-Sipman, M. H. ; Zwinderman, A. H. et al. / Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum. In: Nederlands Tijdschrift voor Geneeskunde. 2002 ; Vol. 146, No. 33. pp. 1542-1546.

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title = "Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum",

abstract = "Objective. To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia. Design. Retrospective, descriptive. Method. Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graftversus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin). Results. There was an increase in the 1-year actuarial survival rate from 67% in the period before 1989 to 90% thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001). Conclusion. The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90%.",

author = "{Van Steekelenburg}, M. and {Van Weel-Sipman}, {M. H.} and Zwinderman, {A. H.} and Hoogerbrugge, {P. M.} and Vossen, {J. M.J.J.} and Egeler, {R. M.}",

year = "2002",

month = aug,

day = "17",

language = "Nederlands",

volume = "146",

pages = "1542--1546",

journal = "Nederlands Tijdschrift voor Geneeskunde",

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Van Steekelenburg, M, Van Weel-Sipman, MH, Zwinderman, AH, Hoogerbrugge, PM, Vossen, JMJJ & Egeler, RM 2002, 'Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum', Nederlands Tijdschrift voor Geneeskunde, vol. 146, no. 33, pp. 1542-1546.

Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum. / Van Steekelenburg, M.; Van Weel-Sipman, M. H.; Zwinderman, A. H. et al.
In: Nederlands Tijdschrift voor Geneeskunde, Vol. 146, No. 33, 17.08.2002, p. 1542-1546.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum

AU - Van Steekelenburg, M.

AU - Van Weel-Sipman, M. H.

AU - Zwinderman, A. H.

AU - Hoogerbrugge, P. M.

AU - Vossen, J. M.J.J.

AU - Egeler, R. M.

PY - 2002/8/17

Y1 - 2002/8/17

N2 - Objective. To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia. Design. Retrospective, descriptive. Method. Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graftversus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin). Results. There was an increase in the 1-year actuarial survival rate from 67% in the period before 1989 to 90% thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001). Conclusion. The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90%.

AB - Objective. To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia. Design. Retrospective, descriptive. Method. Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graftversus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin). Results. There was an increase in the 1-year actuarial survival rate from 67% in the period before 1989 to 90% thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001). Conclusion. The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90%.

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Van Steekelenburg M, Van Weel-Sipman MH, Zwinderman AH, Hoogerbrugge PM, Vossen JMJJ, Egeler RM. Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum. Nederlands Tijdschrift voor Geneeskunde. 2002 Aug 17;146(33):1542-1546.