Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation

André Tichelli; Régis Peffault De Latour; Jakob Passweg; Cora Knol-Bout; Gérard Socié; Judith Marsh; Hubert Schrezenmeier; Britta Höchsmann; Andrea Bacigalupo; Sujith Samarasinghe; Alicia Rovó; Austin Kulasekararaj; Alexander Röth; Dirk Jan Eikema; Paul Bosman; Peter Bader; Antonio Risitano; Carlo Dufour

doi:10.3324/HAEMATOL.2019.222562

Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation

André Tichelli^*, Régis Peffault De Latour, Jakob Passweg, Cora Knol-Bout, Gérard Socié, Judith Marsh, Hubert Schrezenmeier, Britta Höchsmann, Andrea Bacigalupo, Sujith Samarasinghe, Alicia Rovó, Austin Kulasekararaj, Alexander Röth, Dirk Jan Eikema, Paul Bosman, Peter Bader, Antonio Risitano, Carlo Dufour

^*Corresponding author for this work

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Abstract

This follow-up study of a randomized, prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving antithymoglobulin and cyclosporine, with or without granulocyte colony-stimulating factor (G-CSF). We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (95% CI, 10.9- 12.5). The overall survival rate at 15 years was 57±12% in the group given GCSF and 63±12% in the group not given G-CSF (P=0.92); the corresponding event-free survival rates were 24±10% and 23±10%, respectively (P=0.36). In total, 9 patients developed MDS or AML, 10 only a clonal cytogenetic abnormality, 7 a solid cancer, 18 clinical paroxysmal nocturnal hemoglobinuria, 8 osteonecrosis, and 12 chronic kidney disease, without any difference between patients treated with or without G-CSF. The cumulative incidence of MDS, AML or isolated cytogenetic abnormality at 15 years was 8.5±3% for the G-CSF group and 8.2±3% for the non-G-CSF group (P=0.90). The cumulative incidence of any late event including myelodysplastic syndrome or acute myeloid leukemia, isolated cytogenetic abnormalities, solid cancer, clinical paroxysmal nocturnal hemoglobinuria, aseptic osteonecrosis, chronic kidney disease and relapse was 50±12% for the G-CSF group and 49±12% for the non-G-CSF group (P=0.65). Our results demonstrate that it is unlikely ABSTRACT A. Tichelli that G-CSF has an impact on the outcome of severe aplastic anemia; nevertheless, very late events are common and eventually affect the prognosis of these patients, irrespectively of their age at the time of immunosuppressive therapy (NCT01163942).

Original language	English
Pages (from-to)	1223-1231
Number of pages	9
Journal	Haematologica
Volume	105
Issue number	5
DOIs	https://doi.org/10.3324/HAEMATOL.2019.222562
Publication status	Published - May 2020

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Tichelli, A., De Latour, R. P., Passweg, J., Knol-Bout, C., Socié, G., Marsh, J., Schrezenmeier, H., Höchsmann, B., Bacigalupo, A., Samarasinghe, S., Rovó, A., Kulasekararaj, A., Röth, A., Eikema, D. J., Bosman, P., Bader, P., Risitano, A., & Dufour, C. (2020). Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. Haematologica, 105(5), 1223-1231. https://doi.org/10.3324/HAEMATOL.2019.222562

Tichelli, André ; De Latour, Régis Peffault ; Passweg, Jakob et al. / Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor : A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. In: Haematologica. 2020 ; Vol. 105, No. 5. pp. 1223-1231.

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title = "Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation",

abstract = "This follow-up study of a randomized, prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving antithymoglobulin and cyclosporine, with or without granulocyte colony-stimulating factor (G-CSF). We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (95% CI, 10.9- 12.5). The overall survival rate at 15 years was 57±12% in the group given GCSF and 63±12% in the group not given G-CSF (P=0.92); the corresponding event-free survival rates were 24±10% and 23±10%, respectively (P=0.36). In total, 9 patients developed MDS or AML, 10 only a clonal cytogenetic abnormality, 7 a solid cancer, 18 clinical paroxysmal nocturnal hemoglobinuria, 8 osteonecrosis, and 12 chronic kidney disease, without any difference between patients treated with or without G-CSF. The cumulative incidence of MDS, AML or isolated cytogenetic abnormality at 15 years was 8.5±3% for the G-CSF group and 8.2±3% for the non-G-CSF group (P=0.90). The cumulative incidence of any late event including myelodysplastic syndrome or acute myeloid leukemia, isolated cytogenetic abnormalities, solid cancer, clinical paroxysmal nocturnal hemoglobinuria, aseptic osteonecrosis, chronic kidney disease and relapse was 50±12% for the G-CSF group and 49±12% for the non-G-CSF group (P=0.65). Our results demonstrate that it is unlikely ABSTRACT A. Tichelli that G-CSF has an impact on the outcome of severe aplastic anemia; nevertheless, very late events are common and eventually affect the prognosis of these patients, irrespectively of their age at the time of immunosuppressive therapy (NCT01163942).",

author = "Andr{\'e} Tichelli and {De Latour}, {R{\'e}gis Peffault} and Jakob Passweg and Cora Knol-Bout and G{\'e}rard Soci{\'e} and Judith Marsh and Hubert Schrezenmeier and Britta H{\"o}chsmann and Andrea Bacigalupo and Sujith Samarasinghe and Alicia Rov{\'o} and Austin Kulasekararaj and Alexander R{\"o}th and Eikema, {Dirk Jan} and Paul Bosman and Peter Bader and Antonio Risitano and Carlo Dufour",

year = "2020",

month = may,

doi = "10.3324/HAEMATOL.2019.222562",

language = "English",

volume = "105",

pages = "1223--1231",

journal = "Haematologica",

issn = "0390-6078",

publisher = "Ferrata Storti Foundation",

number = "5",

}

Tichelli, A, De Latour, RP, Passweg, J, Knol-Bout, C, Socié, G, Marsh, J, Schrezenmeier, H, Höchsmann, B, Bacigalupo, A, Samarasinghe, S, Rovó, A, Kulasekararaj, A, Röth, A, Eikema, DJ, Bosman, P, Bader, P, Risitano, A & Dufour, C 2020, 'Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation', Haematologica, vol. 105, no. 5, pp. 1223-1231. https://doi.org/10.3324/HAEMATOL.2019.222562

Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. / Tichelli, André; De Latour, Régis Peffault; Passweg, Jakob et al.
In: Haematologica, Vol. 105, No. 5, 05.2020, p. 1223-1231.

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

TY - JOUR

T1 - Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor

T2 - A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation

AU - Tichelli, André

AU - De Latour, Régis Peffault

AU - Passweg, Jakob

AU - Knol-Bout, Cora

AU - Socié, Gérard

AU - Marsh, Judith

AU - Schrezenmeier, Hubert

AU - Höchsmann, Britta

AU - Bacigalupo, Andrea

AU - Samarasinghe, Sujith

AU - Rovó, Alicia

AU - Kulasekararaj, Austin

AU - Röth, Alexander

AU - Eikema, Dirk Jan

AU - Bosman, Paul

AU - Bader, Peter

AU - Risitano, Antonio

AU - Dufour, Carlo

PY - 2020/5

Y1 - 2020/5

N2 - This follow-up study of a randomized, prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving antithymoglobulin and cyclosporine, with or without granulocyte colony-stimulating factor (G-CSF). We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (95% CI, 10.9- 12.5). The overall survival rate at 15 years was 57±12% in the group given GCSF and 63±12% in the group not given G-CSF (P=0.92); the corresponding event-free survival rates were 24±10% and 23±10%, respectively (P=0.36). In total, 9 patients developed MDS or AML, 10 only a clonal cytogenetic abnormality, 7 a solid cancer, 18 clinical paroxysmal nocturnal hemoglobinuria, 8 osteonecrosis, and 12 chronic kidney disease, without any difference between patients treated with or without G-CSF. The cumulative incidence of MDS, AML or isolated cytogenetic abnormality at 15 years was 8.5±3% for the G-CSF group and 8.2±3% for the non-G-CSF group (P=0.90). The cumulative incidence of any late event including myelodysplastic syndrome or acute myeloid leukemia, isolated cytogenetic abnormalities, solid cancer, clinical paroxysmal nocturnal hemoglobinuria, aseptic osteonecrosis, chronic kidney disease and relapse was 50±12% for the G-CSF group and 49±12% for the non-G-CSF group (P=0.65). Our results demonstrate that it is unlikely ABSTRACT A. Tichelli that G-CSF has an impact on the outcome of severe aplastic anemia; nevertheless, very late events are common and eventually affect the prognosis of these patients, irrespectively of their age at the time of immunosuppressive therapy (NCT01163942).

AB - This follow-up study of a randomized, prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving antithymoglobulin and cyclosporine, with or without granulocyte colony-stimulating factor (G-CSF). We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (95% CI, 10.9- 12.5). The overall survival rate at 15 years was 57±12% in the group given GCSF and 63±12% in the group not given G-CSF (P=0.92); the corresponding event-free survival rates were 24±10% and 23±10%, respectively (P=0.36). In total, 9 patients developed MDS or AML, 10 only a clonal cytogenetic abnormality, 7 a solid cancer, 18 clinical paroxysmal nocturnal hemoglobinuria, 8 osteonecrosis, and 12 chronic kidney disease, without any difference between patients treated with or without G-CSF. The cumulative incidence of MDS, AML or isolated cytogenetic abnormality at 15 years was 8.5±3% for the G-CSF group and 8.2±3% for the non-G-CSF group (P=0.90). The cumulative incidence of any late event including myelodysplastic syndrome or acute myeloid leukemia, isolated cytogenetic abnormalities, solid cancer, clinical paroxysmal nocturnal hemoglobinuria, aseptic osteonecrosis, chronic kidney disease and relapse was 50±12% for the G-CSF group and 49±12% for the non-G-CSF group (P=0.65). Our results demonstrate that it is unlikely ABSTRACT A. Tichelli that G-CSF has an impact on the outcome of severe aplastic anemia; nevertheless, very late events are common and eventually affect the prognosis of these patients, irrespectively of their age at the time of immunosuppressive therapy (NCT01163942).

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SN - 0390-6078

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JO - Haematologica

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Tichelli A, De Latour RP, Passweg J, Knol-Bout C, Socié G, Marsh J et al. Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: A Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. Haematologica. 2020 May;105(5):1223-1231. doi: 10.3324/HAEMATOL.2019.222562

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