|
|
Prepublished online as a Blood First Edition Paper on October 10, 2002; DOI 10.1182/blood-2002-04-1134.
Submitted April 15, 2002
Accepted August 14, 2002
Eleven years follow-up of a randomized trial comparing antithymocyte globulin with or without cyclosporine A for treatment of aplastic anemia
Norbert Frickhofen*, Hermann Heimpel, Joachim P Kaltwasser, and Hubert Schrezenmeier
Department of Hematology / Oncology, Dr.-Horst-Schmidt-Kliniken, Wiesbaden, Germany
Department of Hematology / Oncology, University of Ulm, Ulm, Germany
Department of Hematology / Oncology, University of Frankfurt, Frankfurt, Germany
Department of Hematology / Oncology, Free University of Berlin, Benjamin-Franklin-Hospital, Berlin, Germany
* Corresponding author; email: n.frickhofen{at}t-online.de.
Immunosuppression with antithymocyte globulin, (methyl)prednisolone and cyclosporine A is considered the treatment of choice for patients with aplastic anemia without a donor for standard risk stem cell transplantation. This consensus is sup-ported by the results of several series including a randomized German trial. Here we report 11 years-results of the latter trial. With more stringent response criteria and using 4 months as time to evaluate response, this analysis confirms superiority of the cyclosporine regimen regarding the response rate in all patients treated (70% vs. 41%, with or without cyclosporine; P=0.015) as well as in patients with severe aplas-tic anemia (65% vs 31%; P=0.011). Patients responded more rapidly after treatment with cyclosporine (median 60 vs. 82 days; P=0.019). Most patients treated with cyc-losporine needed only one course of immunosuppression whereas many patients treated without cyclosporine required repeated immunosuppressive treatment. Due to the efficacy of salvage treatment, overall survival was not different between the two treatment groups. However, failure free survival favored the cyclosporine regimen (39 vs. 24%; P=0.04). The relapse rate, projected at 38% after 11.3 years, was similar between the two treatment groups. Remissions were cyclosporine-dependent in 26% of the patients responding to a regimen including cyclosporine. Clonal or malignant diseases developed in 25% of the patients. This data demonstrates, that antithymo-cyte globulin, methylprednisolone and cyclosporine A is an effective regimen for treatment of aplastic anemia. However, remissions are unstable and secondary dis-eases are common. In contrast to stem cell transplantation, most patient are not cured.
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
J. R. Passweg and A. Tichelli
Immunosuppressive treatment for aplastic anemia: are we hitting the ceiling?
Haematologica,
March 1, 2009;
94(3):
310 - 312.
[Full Text]
[PDF]
|
|
|
|
|
|
|
P. Scheinberg, C. O. Wu, O. Nunez, P. Scheinberg, C. Boss, E. M. Sloand, and N. S. Young
Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study
Haematologica,
March 1, 2009;
94(3):
348 - 354.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
R. K Barakat, J. P Schmolck, K. W Finkel, and J. R Foringer
Prolonged Renal Failure Secondary to Antithymocyte Globulin Treatment in Severe Aplastic Anemia
Ann. Pharmacother.,
May 1, 2007;
41(5):
895 - 898.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
G. Socie, J.-Y. Mary, H. Schrezenmeier, J. Marsh, A. Bacigalupo, A. Locasciulli, M. Fuehrer, A. Bekassy, A. Tichelli, and J. Passweg
Granulocyte-stimulating factor and severe aplastic anemia: a survey by the European Group for Blood and Marrow Transplantation (EBMT)
Blood,
April 1, 2007;
109(7):
2794 - 2796.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. C.W. Marsh
Treatment of acquired aplastic anemia
Haematologica,
January 1, 2007;
92(1):
2 - 5.
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. Locasciulli, R. Oneto, A. Bacigalupo, G. Socie, E. Korthof, A. Bekassy, H. Schrezenmeier, J. Passweg, M. Fuhrer, and on the behalf of the Severe Aplastic Anemia Workin
Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation
Haematologica,
January 1, 2007;
92(1):
11 - 18.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. Bacigalupo
Aplastic Anemia: Pathogenesis and Treatment
Hematology,
January 1, 2007;
2007(1):
23 - 28.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N. Inoue, T. Izui-Sarumaru, Y. Murakami, Y. Endo, J.-I. Nishimura, K. Kurokawa, M. Kuwayama, H. Shime, T. Machii, Y. Kanakura, et al.
Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH)
Blood,
December 15, 2006;
108(13):
4232 - 4236.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N. S. Young, R. T. Calado, and P. Scheinberg
Current concepts in the pathophysiology and treatment of aplastic anemia
Blood,
October 15, 2006;
108(8):
2509 - 2519.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Hall and T. M. Hagemann
Successful desensitization to antithymocyte globulin in a child with aplastic anemia.
Am. J. Health Syst. Pharm.,
September 1, 2006;
63(17):
1633 - 1636.
[Full Text]
[PDF]
|
|
|
|
|
|
|
E. Sepulveda, U. Brethauer, J. Rojas, and P. Le Fort
Oral manifestations of aplastic anemia in children
J Am Dent Assoc,
April 1, 2006;
137(4):
474 - 478.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
C. Sugimori, T. Chuhjo, X. Feng, H. Yamazaki, A. Takami, M. Teramura, H. Mizoguchi, M. Omine, and S. Nakao
Minor population of CD55-CD59- blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemia
Blood,
February 15, 2006;
107(4):
1308 - 1314.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
W. Fureder, M.-T. Krauth, W. R. Sperr, K. Sonneck, I. Simonitsch-Klupp, L. Mullauer, M. Willmann, H.-P. Horny, and P. Valent
Evaluation of Angiogenesis and Vascular Endothelial Growth Factor Expression in the Bone Marrow of Patients with Aplastic Anemia
Am. J. Pathol.,
January 1, 2006;
168(1):
123 - 130.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. Marsh
Making Therapeutic Decisions in Adults with Aplastic Anemia
Hematology,
January 1, 2006;
2006(1):
78 - 85.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Fuhrer, U. Rampf, I. Baumann, A. Faldum, C. Niemeyer, G. Janka-Schaub, W. Friedrich, W. Ebell, A. Borkhardt, C. Bender-Goetze, et al.
Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival
Blood,
September 15, 2005;
106(6):
2102 - 2104.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
E. C. Guinan
Aplastic Anemia: Management of Pediatric Patients
Hematology,
January 1, 2005;
2005(1):
104 - 109.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. P. Maciejewski and A. M. Risitano
Aplastic Anemia: Management of Adult Patients
Hematology,
January 1, 2005;
2005(1):
110 - 117.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
L. Ades, J.-Y. Mary, M. Robin, C. Ferry, R. Porcher, H. Esperou, P. Ribaud, A. Devergie, R. Traineau, E. Gluckman, et al.
Long-term outcome after bone marrow transplantation for severe aplastic anemia
Blood,
April 1, 2004;
103(7):
2490 - 2497.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
G. C. Bagby, J. M. Lipton, E. M. Sloand, and C. A. Schiffer
Marrow Failure
Hematology,
January 1, 2004;
2004(1):
318 - 336.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
W. Zeng, G. Chen, S. Kajigaya, O. Nunez, A. Charrow, E. M. Billings, and N. S. Young
Gene expression profiling in CD34 cells to identify differences between aplastic anemia patients and healthy volunteers
Blood,
January 1, 2004;
103(1):
325 - 332.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
