Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globuline, cyclosporine, with or without G-CSF: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation.

Tichelli, André; Peffault de Latour, Régis; Passweg, Jakob; Knol-Bout, Cora; Socié, Gérard; Marsh, Judith; Schrezenmeier, Hubert; Höchsmann, Britta; Bacigalupo, Andrea; Samarasinghe, Sujith; Rovó, Alicia; Kulasekararaj, Austin; Röth, Alexander; Eikema, Dirk-Jan; Bosman, Paul; Bader, Peter; Risitano, Antonio; Dufour, Carlo (2020). Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globuline, cyclosporine, with or without G-CSF: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. Haematologica - the hematology journal, 105(5), pp. 1223-1231. Ferrata-Storti Foundation 10.3324/haematol.2019.222562

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This follow-up study of the randomized prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving Antithymoglobulin and Cyclosporine, with and without G-CSF. We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome or acute myeloid leukemia, clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (10.9-12.5). Overall survival at 15 years was 57±12% for the G-CSF and 63±12% for the non-G-CSF group (P=0.92), event-free survival 24±10% for the G-CSF, and 23±10% for the non-G-CSF group (P=0.36). In total, 9 patients developed myelodysplastic syndrome or acute myeloid leukemia, and 10 clonal cytogenetic abnormality only, 7 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. Cumulative incidence at 15 years of myelodysplastic syndrome, acute myeloid leukemia or isolated cytogenetic abnormality was 8.5±3% for the G-CSF, and 8.2±3% for the non-G-CSF group (P=0.90). 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, and 49±12% for the non-G-CSF group (P=0.65). Our results demonstrate that it is unlikely that G-CSF impacts on the outcome of severe aplastic anemia; nevertheless, very late events are common and eventually impact on the prognosis of these patients, irrespectively of their age at immunosuppressive therapy (NCT01163942).

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory

UniBE Contributor:

Rovó, Alicia

Subjects:

600 Technology > 610 Medicine & health

ISSN:

0390-6078

Publisher:

Ferrata-Storti Foundation

Language:

English

Submitter:

Pierrette Durand Lüthi

Date Deposited:

01 Nov 2019 15:46

Last Modified:

04 May 2020 01:31

Publisher DOI:

10.3324/haematol.2019.222562

PubMed ID:

31582549

Uncontrolled Keywords:

Bone Marrow Failure G-CSF immunosuppressive therapy long-term outcome

BORIS DOI:

10.7892/boris.134270

URI:

https://boris.unibe.ch/id/eprint/134270

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