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

被引：171

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

共 50 条

[1] Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party (vol 119, pg 5391, 2012)
Marsh, J. C.
Bacigalupo, A.
Schrezenmeier, H.
BLOOD, 2013, 121 (25) : 5104 - 5104
[2] Treatment of aplastic anaemia with Antithymocyte globulin (ATG): current situation regarding horse vs rabbit ATG; a report from the EBMT Severe Aplastic Anaemia Working Party
Marsh, J.
Bacigalupo, A.
Schrezenmeier, H.
Tichelli, A.
Risitano, A. M.
Passweg, J.
Aljurf, M.
Hochsmann, B.
de la Tour, R. Peffault
Korthof, E.
Rovo, A.
Samarasinghe, S.
Kulasekararaj, A.
Dufour, C.
Oneto, R.
Socie, G.
BONE MARROW TRANSPLANTATION, 2013, 48 : S9 - S9
[3] Outcomes of Antithymocyte Globulin and Cyclosporine for Severe Aplastic Anemia
Ullah, Fauzia
Dima, Danai
Gurnari, Carmelo
Ahmed, Arooj
Ogbue, Olisaemeka
Orland, Mark
Kawashima, Naomi
Bravo-Perez, Carlos
Unlu, Serhan
Guarnera, Luca
Omar, Najiullah
Visconte, Valeria
Maciejewski, Jaroslaw P.
BLOOD, 2023, 142
[4] Pregnancy in patients with aplastic anemia treated with immunosuppression:: on behalf of the EBMT severe aplastic anaemia working party
Tichelli, A
Socié, G
Marsh, J
Barge, R
Frickhofen, N
McCann, S
Bacigalupo, A
Hows, J
Marin, P
Nachbaur, D
Symeonidis, U
Symeonidis, A
Passweg, JR
Schrezenmeier, H
BONE MARROW TRANSPLANTATION, 2001, 27 : S68 - S68
[5] Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A
Garanito, Marlene Pereira
Aivazoglou Carneiro, Jorge David
Odone Filho, Vicente
Scheinberg, Phillip
JORNAL DE PEDIATRIA, 2014, 90 (05) : 523 - 527
[6] Unrelated donor HSCT for acquired severe aplastic anaemia: report from the EBMT Severe Aplastic Anaemia Working Party
Kulasekararaj, A.
Bacigalupo, A.
Schrezenmeier, H.
Tichelli, A.
Risitano, A. M.
Aljurf, M.
Hochsmann, B.
de la Tour, R. Peffault
Korthof, E.
Rovo, A.
Samarasinghe, S.
Dufour, S.
Oneto, R.
Socie, G.
Marsh, J.
Passweg, J.
BONE MARROW TRANSPLANTATION, 2013, 48 : S8 - S9
[7] Rabbit Antithymocyte globulin in treatment of children with severe aplastic anemia
Pawelec, Katarzyna
Matysiak, Michal J.
Niewiadomska, Edyta
Jerzy, Kowalczyk
Stefaniak, Jolanta
Balwierz, Walentyna
Zaleska-Czepko, Ewa
Chybicka, Alicia
Szmyd, Krzysztof
Sonta-Jakimczyk, Danuta
Bubala, Halina
Wysocki, Mariusz
Kurylak, Andrzej
Wachowiak, Jacek
Kaczmarek-Karnold, Malgorzata
Stolarska, Malgorzata
Krawczuk-Rybak, Maryna
Leszczynska, Elzbieta
Urasinski, Tomasz
Peregud-Pogorzelski, Jaroslaw
Balcerska, Anna
Wlazlowski, Marek
BLOOD, 2007, 110 (11) : 8B - 8B
[8] RABBIT ANTITHYMOCYTE GLOBULIN IN CHILDHOOD ACQUIRED SEVERE APLASTIC ANEMIA
Karapinar, D.
Karadas, N.
Ay, Y.
Akin, M.
Balkan, C.
Aydinok, Y.
Kavakli, K.
HAEMATOLOGICA, 2013, 98 : 582 - 582
[9] Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: A report from the European blood and marrow transplant (EBMT) severe aplastic anaemia working party
Marsh, J
Schrezenmeier, H
Marin, P
Ilhan, O
Ljungman, P
McCann, S
Socie, G
Tichelli, A
Passweg, J
Hows, J
Raghavachar, A
Locasciulli, A
Bacigalupo, A
BLOOD, 1999, 93 (07) : 2191 - 2195
[10] Should irradiated blood products be given routinely to all patients with aplastic anaemia undergoing immunosuppressive therapy with antithymocyte globulin (ATG)? A survey from the EBMT Severe Aplastic Anaemia Working Party
Marsh, J.
Socie, G.
Tichelli, A.
Schrezenmeier, H.
Hochsmann, B.
Risitano, A.
Fuehrer, M.
Bekassy, A.
Korthof, E. T.
Locasciulli, A.
Ljungman, P.
Bacigalupo, A.
Camitta, B.
Young, N. S.
Passweg, J.
BONE MARROW TRANSPLANTATION, 2009, 43 : S57 - S57

← 1 2 3 4 5 →