Back
Full-text
Journal article

Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party

  • Marsh, Judith C. Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom;
  • Bacigalupo, Andrea Department of Hematology II, Azienda Ospedaliera Universitaria San Martino, Genoa, Italy;
  • Schrezenmeier, Hubert Institute for Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University of Ulm, Ulm, Germany;
  • Tichelli, Andre Department of Hematology, University Hospital, Basel, Switzerland;
  • Risitano, Antonio M. Department of Biochemistry and Medical Biotechnology, Hematology, Federico II University of Naples, Naples, Italy;
  • Passweg, Jakob R. Department of Hematology, University Hospital, Basel, Switzerland;
  • Killick, Sally B. Department of Haematology, Royal Bournemouth Hospital, Bournemouth, United Kingdom;
  • Warren, Alan J. Department of Haematology, University of Cambridge, Cambridge, United Kingdom;
  • Foukaneli, Theodora Department of Haematology, University of Cambridge, Cambridge, United Kingdom;
  • Aljurf, Mahmoud Adult Hematology/HSCT Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;
  • Al-Zahrani, H. A. Adult Hematology/HSCT Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;
  • Schafhausen, Philip Department of Oncology and Hematology, University Cancer Centre Hamburg, Hamburg, Germany;
  • Roth, Alexander Department of Hematology, West German Cancer Center, University Hospital Essen, Essen, Germany;
  • Franzke, Anke Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany;
  • Brummendorf, Tim H. Department of Hematology and Oncology, University Hospital of the Rheinisch-Westfälische Technische Hochschule Aachen, Aachen, Germany;
  • Dufour, Carlo Hematology Unit G. Gaslini Childrens' Research Hospital, Genova, Italy;
  • Oneto, Rosi Department of Hematology II, Azienda Ospedaliera Universitaria San Martino, Genoa, Italy;
  • Sedgwick, Philip Centre for Medical and Healthcare Education, St George's, University of London, London, United Kingdom;
  • Barrois, Alain European Blood and Marrow Transplant Clinical Trials Office, Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands; and
  • Kordasti, Shahram Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom;
  • Elebute, Modupe O. Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom;
  • Mufti, Ghulam J. Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom;
  • Socie, Gerard Hospital Saint Louis, Hematology-Transplantation and Université Paris VII, Paris, France
Show more…
Published in:
  • Blood. - American Society of Hematology. - 2012, vol. 119, no. 23, p. 5391-5396
English Abstract
Rabbit antithymocyte globulin (rATG; thymoglobulin, Genzyme) in combination with cyclosporine, as first-line immunosuppressive therapy, was evaluated prospectively in a multicenter, European, phase 2 pilot study, in 35 patients with aplastic anemia. Results were compared with 105 age- and disease severity–matched patients from the European Blood and Marrow Transplant registry, treated with horse ATG (hATG; lymphoglobulin) and cyclosporine. The primary end point was response at 6 months. At 3 months, no patients had achieved a complete response to rATG. Partial response occurred in 11 (34%). At 6 months, complete response rate was 3% and partial response rate 37%. There were 10 deaths after rATG (28.5%) and 1 after subsequent HSCT. Infections were the main cause of death in 9 of 10 patients. The best response rate was 60% for rATG and 67% for hATG. For rATG, overall survival at 2 years was 68%, compared with 86% for hATG (P = .009). Transplant-free survival was 52% for rATG and 76% for hATG (P = .002). On multivariate analysis, rATG (hazard ratio = 3.9, P = .003) and age more than 37 years (hazard ratio = 4.7, P = .0008) were independent adverse risk factors for survival. This study was registered at www.clinicaltrials.gov as NCT00471848.
Language
  • English
Open access status
green
Identifiers
Persistent URL
https://sonar.ch/global/documents/238372
Statistics
Document views: 45 File downloads:
  • Full-text: 0