Page 63 - Haematologica May 2020

Bone Marrow Failure
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 granu- locyte colony-stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation
Ferrata Storti Foundation
Haematologica 2020 Volume 105(5):1223-1231
André Tichelli,1 Régis Peffault de Latour,2 Jakob Passweg,1 Cora Knol-Bout,3 Gérard Socié,4 Judith Marsh,5 Hubert Schrezenmeier,6 Britta Höchsmann,6 Andrea Bacigalupo,7 Sujith Samarasinghe,8 Alicia Rovó,9 Austin Kulasekararaj,10 Alexander Röth,11 Dirk-Jan Eikema,3 Paul Bosman,3 Peter Bader,12 Antonio Risitano13 and Carlo Dufour14 on behalf of the SAA Working Party of the EBMT
1Division of Hematology, University Hospital Basel, Basel, Switzerland; 2Université de Paris, and Hematology-Transplantation, Saint Louis Hospital (AP-HP), Paris, France; 3EBMT Registry Office, Leiden, the Netherlands; 4Université de Paris, INSERM U976 and Hematology- Transplantation, Saint Louis Hospital (AP-HP), Paris, France; 5Department of Haematological Medicine, King's College Hospital/King's College London, London, UK; 6Institute of Tranfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Transfusion Service Baden-Württemberg- Hessen and University Hospital Ulm, Ulm, Germany; 7Instituto di Ematologia, Fondazione Policlinico Universitario Gemelli IRCSS, Università Cattolica del Sacro Cuore, Roma, Italy; 8Sujith Samarasinghe, Great Ormond Street Hospital, London, UK; 9Department of Hematology and Central Hematology Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland; 10Department of Haematological Medicine, King’s College Hospital, NIHR/Wellcome King’s Clinical Research Facility, London, UK; 11Department of Hematology, West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany; 12University Children’s Hospital Frankfurt, Frankfurt, Germany; 13Hematology Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy and 14Hemato-Onco-SCT Pole, Hematology Unit. G. Gaslini Children’s Research Hospital, Genova, Italy.
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 G- CSF 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 abnor- mality, 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
Correspondence:
ANDRÉ TICHELLI
tichelli@datacomm.ch
Received: March 20, 2019. Accepted: September 30, 2019. Pre-published: October 3, 2019.
doi:10.3324/haematol.2019.222562
Check the online version for the most updated information on this article, online supplements, and information on authorship & disclosures: www.haematologica.org/content/105/5/1223
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