Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party.
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Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. / Marsh, Judith C; Bacigalupo, Andrea; Schrezenmeier, Hubert; Tichelli, Andre; Risitano, Antonio M; Passweg, Jakob R; Killick, Sally B; Warren, Alan J; Foukaneli, Theodora; Aljurf, Mahmoud; Al-Zahrani, H A; Schafhausen, Philippe; Roth, Alexander; Franzke, Anke; Brummendorf, Tim H; Dufour, Carlo; Oneto, Rosi; Sedgwick, Philip; Barrois, Alain; Kordasti, Shahram; Elebute, Modupe O; Mufti, Ghulam J; Socie, Gerard; Blood, European; Party, Marrow Transplant Group Severe Aplastic Anaemia Working.
in: BLOOD, Jahrgang 119, Nr. 23, 23, 2012, S. 5391-5396.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party.
AU - Marsh, Judith C
AU - Bacigalupo, Andrea
AU - Schrezenmeier, Hubert
AU - Tichelli, Andre
AU - Risitano, Antonio M
AU - Passweg, Jakob R
AU - Killick, Sally B
AU - Warren, Alan J
AU - Foukaneli, Theodora
AU - Aljurf, Mahmoud
AU - Al-Zahrani, H A
AU - Schafhausen, Philippe
AU - Roth, Alexander
AU - Franzke, Anke
AU - Brummendorf, Tim H
AU - Dufour, Carlo
AU - Oneto, Rosi
AU - Sedgwick, Philip
AU - Barrois, Alain
AU - Kordasti, Shahram
AU - Elebute, Modupe O
AU - Mufti, Ghulam J
AU - Socie, Gerard
AU - Blood, European
AU - Party, Marrow Transplant Group Severe Aplastic Anaemia Working
PY - 2012
Y1 - 2012
N2 - 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.
AB - 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.
KW - Adult
KW - Animals
KW - Humans
KW - Male
KW - Aged
KW - Female
KW - Middle Aged
KW - Adolescent
KW - Young Adult
KW - Prospective Studies
KW - Europe
KW - Survival Analysis
KW - Rabbits
KW - Pilot Projects
KW - Drug Therapy, Combination
KW - Horses
KW - Immunosuppressive Agents/adverse effects/therapeutic use
KW - Anemia, Aplastic/drug therapy
KW - Antilymphocyte Serum/adverse effects/therapeutic use
KW - CD4-Positive T-Lymphocytes/drug effects
KW - Cyclosporine/adverse effects/therapeutic use
KW - Adult
KW - Animals
KW - Humans
KW - Male
KW - Aged
KW - Female
KW - Middle Aged
KW - Adolescent
KW - Young Adult
KW - Prospective Studies
KW - Europe
KW - Survival Analysis
KW - Rabbits
KW - Pilot Projects
KW - Drug Therapy, Combination
KW - Horses
KW - Immunosuppressive Agents/adverse effects/therapeutic use
KW - Anemia, Aplastic/drug therapy
KW - Antilymphocyte Serum/adverse effects/therapeutic use
KW - CD4-Positive T-Lymphocytes/drug effects
KW - Cyclosporine/adverse effects/therapeutic use
M3 - SCORING: Journal article
VL - 119
SP - 5391
EP - 5396
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 23
M1 - 23
ER -