Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Carlo Dufour; Marta Pillon; Jakob Passweg; Gerard Socie; Andrea Bacigalupo; Genny Franceschetto; Elisa Carraro; Rosi Oneto; Antonio Maria Risitano; Regis Peffault de Latour; Andre Tichelli; Alicia Rovo; Christina Peters; Britta Hoechsmann; Sujith Samarasinghe; Austin Kulasekararaj; Hubert Schrezenmeier; Mahmoud Aljurf; Judith Marsh

doi:10.3324/haematol.2014.106096

Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Carlo Dufour^*, Marta Pillon, Jakob Passweg, Gerard Socie, Andrea Bacigalupo, Genny Franceschetto, Elisa Carraro, Rosi Oneto, Antonio Maria Risitano, Regis Peffault de Latour, Andre Tichelli, Alicia Rovo, Christina Peters, Britta Hoechsmann, Sujith Samarasinghe, Austin Kulasekararaj, Hubert Schrezenmeier, Mahmoud Aljurf, Judith Marsh

^*Corresponding author for this work

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

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Abstract

We analyzed the outcome of 537 adolescents (age 12-18 years) with idiopathic aplastic anemia included in the database of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation comparing: i) matched family donor hematopoietic stem cell transplantation performed as first-line treatment with ii) front-line immunosuppressive therapy not followed by subsequent transplant given for failure and with iii) hematopoietic stem cell transplantation performed after failed front-line immunosuppressive therapy. Overall survival was 86% in the matched family donor hematopoietic stem cell transplantation group, 90% in patients given front-line immunosuppressive alone (those who did not fail this treatment and who did not receive subsequent rescue with hematopoietic stem cell transplantation) and 78% in subjects who underwent hematopoietic stem cell transplantation post failed front-line immunosuppressive therapy (P=0.14). Event-free survival in the same groups was respectively 83%, 64% and 71% (P=0.04). Cumulative incidence of rejection was 8% in matched family donor hematopoietic stem cell transplantation and 9% in transplants post failed front-line immunosuppression (P=0.62). Cumulative incidence of acute graft-versus-host disease was 12% in matched family donor transplants and 18% in transplants post failed immunosuppression (P=0.18). Chronic graft-versus-host disease was higher in matched family donor hematopoietic stem cell transplantation (8%) than in transplants post failed immunosuppressive therapy (20%) (P=0.0009). Cumulative incidence of post-therapy malignancies was 0.7% in matched family donor transplantations, 7% in transplantations post failed immunosuppression and 21% after front-line immunosuppression (P=0.0017). In the whole cohort, under multivariate analysis, the diagnosis to treatment interval of two months or under positively affected overall survival whereas up-front immunosuppression alone (with no subsequent rescue transplants) negatively affected event-free survival. In transplanted patients an interval from diagnosis to treatment of 2 months or under, bone marrow as source of cells and first-line matched family donor transplants provided a significant advantage in overall and event-free survival. Aplastic anemia in adolescents has a very good outcome. If a matched family donor is available, hematopoietic stem cell transplantation using bone marrow cells is the first choice treatment. If such a donor is not available, immunosuppressive treatment may still be an acceptable second choice, also because, in case of failure, hematopoietic stem cell transplantation is a very good rescue option.

Original language	English
Pages (from-to)	1574-1581
Number of pages	8
Journal	Haematologica
Volume	99
Issue number	10
DOIs	https://doi.org/10.3324/haematol.2014.106096
Publication status	Published - 1 Oct 2014

Keywords

RABBIT ANTITHYMOCYTE GLOBULIN
UNRELATED DONOR TRANSPLANTATION
STEM-CELL TRANSPLANTATION
IMMUNOSUPPRESSIVE THERAPY
BONE-MARROW
PERIPHERAL-BLOOD
CYCLOSPORINE
CHILDREN
HORSE
AGE

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10.3324/haematol.2014.106096

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Dufour, C., Pillon, M., Passweg, J., Socie, G., Bacigalupo, A., Franceschetto, G., Carraro, E., Oneto, R., Risitano, A. M., de Latour, R. P., Tichelli, A., Rovo, A., Peters, C., Hoechsmann, B., Samarasinghe, S., Kulasekararaj, A., Schrezenmeier, H., Aljurf, M., & Marsh, J. (2014). Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Haematologica, 99(10), 1574-1581. https://doi.org/10.3324/haematol.2014.106096

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title = "Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation",

abstract = "We analyzed the outcome of 537 adolescents (age 12-18 years) with idiopathic aplastic anemia included in the database of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation comparing: i) matched family donor hematopoietic stem cell transplantation performed as first-line treatment with ii) front-line immunosuppressive therapy not followed by subsequent transplant given for failure and with iii) hematopoietic stem cell transplantation performed after failed front-line immunosuppressive therapy. Overall survival was 86% in the matched family donor hematopoietic stem cell transplantation group, 90% in patients given front-line immunosuppressive alone (those who did not fail this treatment and who did not receive subsequent rescue with hematopoietic stem cell transplantation) and 78% in subjects who underwent hematopoietic stem cell transplantation post failed front-line immunosuppressive therapy (P=0.14). Event-free survival in the same groups was respectively 83%, 64% and 71% (P=0.04). Cumulative incidence of rejection was 8% in matched family donor hematopoietic stem cell transplantation and 9% in transplants post failed front-line immunosuppression (P=0.62). Cumulative incidence of acute graft-versus-host disease was 12% in matched family donor transplants and 18% in transplants post failed immunosuppression (P=0.18). Chronic graft-versus-host disease was higher in matched family donor hematopoietic stem cell transplantation (8%) than in transplants post failed immunosuppressive therapy (20%) (P=0.0009). Cumulative incidence of post-therapy malignancies was 0.7% in matched family donor transplantations, 7% in transplantations post failed immunosuppression and 21% after front-line immunosuppression (P=0.0017). In the whole cohort, under multivariate analysis, the diagnosis to treatment interval of two months or under positively affected overall survival whereas up-front immunosuppression alone (with no subsequent rescue transplants) negatively affected event-free survival. In transplanted patients an interval from diagnosis to treatment of 2 months or under, bone marrow as source of cells and first-line matched family donor transplants provided a significant advantage in overall and event-free survival. Aplastic anemia in adolescents has a very good outcome. If a matched family donor is available, hematopoietic stem cell transplantation using bone marrow cells is the first choice treatment. If such a donor is not available, immunosuppressive treatment may still be an acceptable second choice, also because, in case of failure, hematopoietic stem cell transplantation is a very good rescue option.",

keywords = "RABBIT ANTITHYMOCYTE GLOBULIN, UNRELATED DONOR TRANSPLANTATION, STEM-CELL TRANSPLANTATION, IMMUNOSUPPRESSIVE THERAPY, BONE-MARROW, PERIPHERAL-BLOOD, CYCLOSPORINE, CHILDREN, HORSE, AGE",

author = "Carlo Dufour and Marta Pillon and Jakob Passweg and Gerard Socie and Andrea Bacigalupo and Genny Franceschetto and Elisa Carraro and Rosi Oneto and Risitano, {Antonio Maria} and {de Latour}, {Regis Peffault} and Andre Tichelli and Alicia Rovo and Christina Peters and Britta Hoechsmann and Sujith Samarasinghe and Austin Kulasekararaj and Hubert Schrezenmeier and Mahmoud Aljurf and Judith Marsh",

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doi = "10.3324/haematol.2014.106096",

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Dufour, C, Pillon, M, Passweg, J, Socie, G, Bacigalupo, A, Franceschetto, G, Carraro, E, Oneto, R, Risitano, AM, de Latour, RP, Tichelli, A, Rovo, A, Peters, C, Hoechsmann, B, Samarasinghe, S, Kulasekararaj, A, Schrezenmeier, H, Aljurf, M & Marsh, J 2014, 'Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation', Haematologica, vol. 99, no. 10, pp. 1574-1581. https://doi.org/10.3324/haematol.2014.106096

TY - JOUR

T1 - Outcome of aplastic anemia in adolescence

T2 - a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

AU - Dufour, Carlo

AU - Pillon, Marta

AU - Passweg, Jakob

AU - Socie, Gerard

AU - Bacigalupo, Andrea

AU - Franceschetto, Genny

AU - Carraro, Elisa

AU - Oneto, Rosi

AU - Risitano, Antonio Maria

AU - de Latour, Regis Peffault

AU - Tichelli, Andre

AU - Rovo, Alicia

AU - Peters, Christina

AU - Hoechsmann, Britta

AU - Samarasinghe, Sujith

AU - Kulasekararaj, Austin

AU - Schrezenmeier, Hubert

AU - Aljurf, Mahmoud

AU - Marsh, Judith

PY - 2014/10/1

Y1 - 2014/10/1

N2 - We analyzed the outcome of 537 adolescents (age 12-18 years) with idiopathic aplastic anemia included in the database of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation comparing: i) matched family donor hematopoietic stem cell transplantation performed as first-line treatment with ii) front-line immunosuppressive therapy not followed by subsequent transplant given for failure and with iii) hematopoietic stem cell transplantation performed after failed front-line immunosuppressive therapy. Overall survival was 86% in the matched family donor hematopoietic stem cell transplantation group, 90% in patients given front-line immunosuppressive alone (those who did not fail this treatment and who did not receive subsequent rescue with hematopoietic stem cell transplantation) and 78% in subjects who underwent hematopoietic stem cell transplantation post failed front-line immunosuppressive therapy (P=0.14). Event-free survival in the same groups was respectively 83%, 64% and 71% (P=0.04). Cumulative incidence of rejection was 8% in matched family donor hematopoietic stem cell transplantation and 9% in transplants post failed front-line immunosuppression (P=0.62). Cumulative incidence of acute graft-versus-host disease was 12% in matched family donor transplants and 18% in transplants post failed immunosuppression (P=0.18). Chronic graft-versus-host disease was higher in matched family donor hematopoietic stem cell transplantation (8%) than in transplants post failed immunosuppressive therapy (20%) (P=0.0009). Cumulative incidence of post-therapy malignancies was 0.7% in matched family donor transplantations, 7% in transplantations post failed immunosuppression and 21% after front-line immunosuppression (P=0.0017). In the whole cohort, under multivariate analysis, the diagnosis to treatment interval of two months or under positively affected overall survival whereas up-front immunosuppression alone (with no subsequent rescue transplants) negatively affected event-free survival. In transplanted patients an interval from diagnosis to treatment of 2 months or under, bone marrow as source of cells and first-line matched family donor transplants provided a significant advantage in overall and event-free survival. Aplastic anemia in adolescents has a very good outcome. If a matched family donor is available, hematopoietic stem cell transplantation using bone marrow cells is the first choice treatment. If such a donor is not available, immunosuppressive treatment may still be an acceptable second choice, also because, in case of failure, hematopoietic stem cell transplantation is a very good rescue option.

AB - We analyzed the outcome of 537 adolescents (age 12-18 years) with idiopathic aplastic anemia included in the database of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation comparing: i) matched family donor hematopoietic stem cell transplantation performed as first-line treatment with ii) front-line immunosuppressive therapy not followed by subsequent transplant given for failure and with iii) hematopoietic stem cell transplantation performed after failed front-line immunosuppressive therapy. Overall survival was 86% in the matched family donor hematopoietic stem cell transplantation group, 90% in patients given front-line immunosuppressive alone (those who did not fail this treatment and who did not receive subsequent rescue with hematopoietic stem cell transplantation) and 78% in subjects who underwent hematopoietic stem cell transplantation post failed front-line immunosuppressive therapy (P=0.14). Event-free survival in the same groups was respectively 83%, 64% and 71% (P=0.04). Cumulative incidence of rejection was 8% in matched family donor hematopoietic stem cell transplantation and 9% in transplants post failed front-line immunosuppression (P=0.62). Cumulative incidence of acute graft-versus-host disease was 12% in matched family donor transplants and 18% in transplants post failed immunosuppression (P=0.18). Chronic graft-versus-host disease was higher in matched family donor hematopoietic stem cell transplantation (8%) than in transplants post failed immunosuppressive therapy (20%) (P=0.0009). Cumulative incidence of post-therapy malignancies was 0.7% in matched family donor transplantations, 7% in transplantations post failed immunosuppression and 21% after front-line immunosuppression (P=0.0017). In the whole cohort, under multivariate analysis, the diagnosis to treatment interval of two months or under positively affected overall survival whereas up-front immunosuppression alone (with no subsequent rescue transplants) negatively affected event-free survival. In transplanted patients an interval from diagnosis to treatment of 2 months or under, bone marrow as source of cells and first-line matched family donor transplants provided a significant advantage in overall and event-free survival. Aplastic anemia in adolescents has a very good outcome. If a matched family donor is available, hematopoietic stem cell transplantation using bone marrow cells is the first choice treatment. If such a donor is not available, immunosuppressive treatment may still be an acceptable second choice, also because, in case of failure, hematopoietic stem cell transplantation is a very good rescue option.

KW - RABBIT ANTITHYMOCYTE GLOBULIN

KW - UNRELATED DONOR TRANSPLANTATION

KW - STEM-CELL TRANSPLANTATION

KW - IMMUNOSUPPRESSIVE THERAPY

KW - BONE-MARROW

KW - PERIPHERAL-BLOOD

KW - CYCLOSPORINE

KW - CHILDREN

KW - HORSE

KW - AGE

U2 - 10.3324/haematol.2014.106096

DO - 10.3324/haematol.2014.106096

M3 - Article

SN - 0390-6078

VL - 99

SP - 1574

EP - 1581

JO - Haematologica

JF - Haematologica

IS - 10

ER -

Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Abstract

Keywords

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