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

Judith Marsh; Andrea Bacigalupo; Hubert Schrezenmeier; Andre Tichelli; Antonio M. Risitano; Jakob R. Passweg; Sally B. Killick; Alan J. Warren; Theodora Foukaneli; Mahmoud Aljurf; H. A. Al-Zahrani; Philip Schafhausen; Alexander Roth; Anke Franzke; Tim H. Brummendorf; Carlo Dufour; Rosi Oneto; Philip Sedgwick; Alain Barrois; Shahram Kordasti; Modupe O. Elebute; Ghulam J. Mufti; Gerard Socie; European Blood Marrow Transplant

doi:10.1182/blood-2012-02-407684

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

Judith Marsh, Andrea Bacigalupo, Hubert Schrezenmeier, Andre Tichelli, Antonio M. Risitano, Jakob R. Passweg, Sally B. Killick, Alan J. Warren, Theodora Foukaneli, Mahmoud Aljurf, H. A. Al-Zahrani, Philip Schafhausen, Alexander Roth, Anke Franzke, Tim H. Brummendorf, Carlo Dufour, Rosi Oneto, Philip Sedgwick, Alain Barrois, Shahram KordastiModupe O. Elebute, Ghulam J. Mufti, Gerard Socie, European Blood Marrow Transplant

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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. (Blood. 2012;119(23):5391-5396)

Original language	English
Pages (from-to)	5391-5396
Number of pages	6
Journal	Blood
Volume	119
Issue number	23
Early online date	27 Apr 2012
DOIs	https://doi.org/10.1182/blood-2012-02-407684
Publication status	Published - 7 Jun 2012

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Marsh, J., 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., ... European Blood Marrow Transplant (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. https://doi.org/10.1182/blood-2012-02-407684

@article{83ca110f07fb4acf8c06e6d282f4394f,

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. (Blood. 2012;119(23):5391-5396)",

author = "Judith Marsh 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 Philip 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 Marrow Transplant}",

year = "2012",

month = jun,

day = "7",

doi = "10.1182/blood-2012-02-407684",

language = "English",

volume = "119",

pages = "5391--5396",

journal = "Blood",

issn = "0006-4971",

publisher = "American Society of Hematology",

number = "23",

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Marsh, J, 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 & European Blood Marrow Transplant 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, pp. 5391-5396. https://doi.org/10.1182/blood-2012-02-407684

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

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, Philip

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 - European Blood Marrow Transplant

PY - 2012/6/7

Y1 - 2012/6/7

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. (Blood. 2012;119(23):5391-5396)

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. (Blood. 2012;119(23):5391-5396)

U2 - 10.1182/blood-2012-02-407684

DO - 10.1182/blood-2012-02-407684

M3 - Article

SN - 0006-4971

VL - 119

SP - 5391

EP - 5396

JO - Blood

JF - Blood

IS - 23

ER -

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

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