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Haematologica. 2019 Oct 3. pii: haematol.2019.222562. doi: 10.3324/haematol.2019.222562. [Epub ahead of print]

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.

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Tichelli, Division of Hematology, University Hospital Basel, Petersgraben 4; 4031 Basel, Switzerland;
Université de Paris, and Hematology-Transplantation,Saint Louis Hospital (AP-HP),Paris,France.
Department of Hematology, University Hospital Basel, Petersgraben 4; 4031 Basel, Switzerland.
EBMT Registry Office, Leiden 2333, Netherland.
Université de Paris,INSERM U976, and Hematology-Transplantation, Saint Louis Hospital (AP-HP).
Department of Haematological Medicine, King's College Hospital/King's College London, London, UK.
Institute of Tranfusion Medicine, University of Ulm, Ulm, Germany.
Instituto di Ematologia, Fondazione Policlinico Universitario Gemelli IRCSS, Roma, Italy.
Great Ormond Street Hospital, London, United Kingdom.
Department of Hematology and Central Hematology Laboratory, Bern University Hospital, Bern.
Department of Haematological Medicine, Kings College Hospital, London, United Kingdom.
Department of Hematology, West German Cancer Center, University Hospital Essen, Essen, Germany.
University Children Hospital Frankfurt, Frankfurt, Germany.
Hematology Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples.
Hemato-Onco-SCT Pole, Hematology Unit. G. Gaslini Children Research Hospital, Genova, Italy.


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).


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

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