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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, Vol. 119, No. 23, 23, 2012, p. 5391-5396.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Marsh, JC, Bacigalupo, A, Schrezenmeier, H, Tichelli, A, Risitano, AM, Passweg, JR, Killick, SB, Warren, AJ, Foukaneli, T, Aljurf, M, Al-Zahrani, HA, Schafhausen, P, Roth, A, Franzke, A, Brummendorf, TH, Dufour, C, Oneto, R, Sedgwick, P, Barrois, A, Kordasti, S, Elebute, MO, Mufti, GJ, Socie, G, Blood, E & Party, MTGSAAW 2012, 'Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party.', BLOOD, vol. 119, no. 23, 23, pp. 5391-5396. <http://www.ncbi.nlm.nih.gov/pubmed/22544699?dopt=Citation>

APA

Marsh, J. C., Bacigalupo, A., Schrezenmeier, H., Tichelli, A., Risitano, A. M., Passweg, J. R., Killick, S. B., Warren, A. J., Foukaneli, T., Aljurf, M., Al-Zahrani, H. A., Schafhausen, P., Roth, A., Franzke, A., Brummendorf, T. H., Dufour, C., Oneto, R., Sedgwick, P., Barrois, A., ... Party, M. T. G. S. A. A. W. (2012). Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. BLOOD, 119(23), 5391-5396. [23]. http://www.ncbi.nlm.nih.gov/pubmed/22544699?dopt=Citation

Vancouver

Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR et al. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. BLOOD. 2012;119(23):5391-5396. 23.

Bibtex

@article{9029df4f3e934dfe9c61e50978b178d9,
title = "Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party.",
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.",
keywords = "Adult, Animals, Humans, Male, Aged, Female, Middle Aged, Adolescent, Young Adult, Prospective Studies, Europe, Survival Analysis, Rabbits, Pilot Projects, Drug Therapy, Combination, Horses, Immunosuppressive Agents/adverse effects/*therapeutic use, Anemia, Aplastic/*drug therapy, Antilymphocyte Serum/adverse effects/*therapeutic use, CD4-Positive T-Lymphocytes/drug effects, Cyclosporine/adverse effects/*therapeutic use, Adult, Animals, Humans, Male, Aged, Female, Middle Aged, Adolescent, Young Adult, Prospective Studies, Europe, Survival Analysis, Rabbits, Pilot Projects, Drug Therapy, Combination, Horses, Immunosuppressive Agents/adverse effects/*therapeutic use, Anemia, Aplastic/*drug therapy, Antilymphocyte Serum/adverse effects/*therapeutic use, CD4-Positive T-Lymphocytes/drug effects, Cyclosporine/adverse effects/*therapeutic use",
author = "Marsh, {Judith C} and Andrea Bacigalupo and Hubert Schrezenmeier and Andre Tichelli and Risitano, {Antonio M} and Passweg, {Jakob R} and Killick, {Sally B} and Warren, {Alan J} and Theodora Foukaneli and Mahmoud Aljurf and Al-Zahrani, {H A} and Philippe Schafhausen and Alexander Roth and Anke Franzke and Brummendorf, {Tim H} and Carlo Dufour and Rosi Oneto and Philip Sedgwick and Alain Barrois and Shahram Kordasti and Elebute, {Modupe O} and Mufti, {Ghulam J} and Gerard Socie and European Blood and Party, {Marrow Transplant Group Severe Aplastic Anaemia Working}",
year = "2012",
language = "English",
volume = "119",
pages = "5391--5396",
journal = "BLOOD",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "23",

}

RIS

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 -