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

被引：174

作者：

Marsh, Judith C. ^{[1
]}

Bacigalupo, Andrea ^{[2
]}

Schrezenmeier, Hubert ^{[3
,4
]}

Tichelli, Andre ^{[5
]}

Risitano, Antonio M. ^{[6
]}

Passweg, Jakob R. ^{[5
]}

Killick, Sally B. ^{[7
]}

Warren, Alan J. ^{[8
]}

Foukaneli, Theodora ^{[8
]}

Aljurf, Mahmoud ^{[9
]}

Al-Zahrani, H. A. ^{[9
]}

Schafhausen, Philip ^{[10
]}

Roth, Alexander ^{[11
]}

Franzke, Anke ^{[12
]}

Brummendorf, Tim H. ^{[13
]}

Dufour, Carlo ^{[14
]}

Oneto, Rosi ^{[2
]}

Sedgwick, Philip ^{[15
]}

Barrois, Alain ^{[16
]}

Kordasti, Shahram ^{[1
]}

Elebute, Modupe O. ^{[1
]}

Mufti, Ghulam J. ^{[1
]}

Socie, Gerard ^{[17
,18
]}

机构：

[1] Kings Coll London, Dept Haematol Med, Univ London Kings Coll Hosp, London SE5 9RS, England

[2] Azienda Osped Univ San Martino, Dept Hematol 2, Genoa, Italy

[3] German Red Cross Blood Transfus Serv, Inst Clin Transfus Med & Immunogenet, Ulm, Germany

[4] Univ Ulm, Ulm, Germany

[5] Univ Basel Hosp, Dept Hematol, CH-4031 Basel, Switzerland

[6] Univ Naples Federico II, Dept Biochem & Med Biotechnol, Naples, Italy

[7] Royal Bournemouth Hosp, Dept Haematol, Bournemouth, Dorset, England

[8] Univ Cambridge, Dept Haematol, Cambridge, England

[9] King Faisal Specialist Hosp & Res Ctr, Adult Hematol HSCT Oncol Ctr, Riyadh 11211, Saudi Arabia

[10] Univ Canc Ctr Hamburg, Dept Oncol & Hematol, Hamburg, Germany

[11] Univ Hosp Essen, W German Canc Ctr, Dept Hematol, Essen, Germany

[12] Hannover Med Sch, Dept Hematol Hemostasis Oncol & Stem Cell Transpl, D-3000 Hannover, Germany

[13] Rhein Westfal TH Aachen, Univ Hosp, Dept Hematol & Oncol, D-52062 Aachen, Germany

[14] G Gaslini Childrens Res Hosp, Hematol Unit, Genoa, Italy

[15] Univ London, Ctr Med & Healthcare Educ, London, England

[16] Leiden Univ, Med Ctr, Dept Med Stat & Bioinformat, European Blood & Marrow Transplant Clin Trials Of, Leiden, Netherlands

[17] Hosp St Louis, Paris, France

[18] Univ Paris 07, Paris, France

来源：

BLOOD | 2012年 / 119卷 / 23期

关键词：

STEM-CELL TRANSPLANTATION; ALEMTUZUMAB PLUS CYCLOSPORINE; ANTI-THYMOCYTE GLOBULIN; IMMUNOSUPPRESSIVE THERAPY; ALTERNATIVE DONOR; STIMULATING FACTOR; FOLLOW-UP; T-CELLS; HORSE; CYCLOPHOSPHAMIDE;

D O I：

10.1182/blood-2012-02-407684

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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)

引用

页码：5391 / 5396

页数：6

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