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Blood, 1 December 2000, Vol. 96, No. 12, pp. 3671-3674

PERSPECTIVE

Report of an international working group to standardize response criteria for myelodysplastic syndromes

Bruce D. Cheson, John M. Bennett, Hagop Kantarjian, Antonio Pinto, Charles A. Schiffer, Stephen D. Nimer, Bob Löwenberg, Miroslav Beran, Theo M. de Witte, Richard M. Stone, Moshe Mittelman, Guillermo F. Sanz, Pierre W. Wijermans, Steven Gore, and Peter L. Greenberg

From the National Cancer Institute, Bethesda, MD; University of Rochester Cancer Center, Rochester, NY; MD Anderson Cancer Center, University of Texas, Houston, TX; Centro di Riferimento Oncologico, Aviano, Italy; Karmanos Cancer Center, Wayne State University, Detroit, MI; Memorial Sloan-Kettering Cancer Center, New York, NY; University Hospital, Rotterdam, The Netherlands; University Hospital Nijmegen, Rijmegen, The Netherlands; Dana Farber Cancer Institute, Boston, MA; Rabin Medical Center-Hasharon Hospital, Petah-Tikva Tel-Aviv University, Tel-Aviv, Israel; Hospital Universitario La Fe, Valencia, Spain; Ziekenhuis, Leyenburg, The Hague, The Netherlands; Johns Hopkins Oncology Center, Baltimore, MD; Stanford University Medical Center and Veterans Administration Hospital, Palo Alto, CA.

Standardized criteria for assessing response are essential to ensure comparability among clinical trials for patients with myelodysplastic syndromes (MDS). An international working group of experienced clinicians involved in the management of patients with MDS reviewed currently used response definitions and developed a uniform set of guidelines for future clinical trials in MDS. The MDS differ from many other hematologic malignancies in their chronicity and the morbidity and mortality caused by chronic cytopenias, often without disease progression to acute myeloid leukemia. Whereas response rates may be an important endpoint for phase 2 studies of new agents and may assist regulatory agencies in their evaluation and approval processes, an important goal of clinical trials in MDS should be to prolong patient survival. Therefore, these response criteria reflected 2 sets of goals in MDS: altering the natural history of the disease and alleviating disease-related complications with improved quality of life. It is anticipated that the recommendations presented will require modification as more is learned about the molecular biology and genetics of these disorders. Until then, it is hoped these guidelines will serve to improve communication among investigators and to ensure comparability among clinical trials.

© 2000 by The American Society of Hematology.
 

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A. F. List
New Approaches to the Treatment of Myelodysplasia
Oncologist, April 1, 2002; 7(90001): 39 - 49.
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R. Stasi, M. Brunetti, E. Terzoli, and S. Amadori
Sustained response to recombinant human erythropoietin and intermittent all-trans retinoic acid in patients with myelodysplastic syndromes
Blood, March 1, 2002; 99(5): 1578 - 1584.
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M. B. Steins, T. Padro, R. Bieker, S. Ruiz, M. Kropff, J. Kienast, T. Kessler, T. Buechner, W. E. Berdel, and R. M. Mesters
Efficacy and safety of thalidomide in patients with acute myeloid leukemia
Blood, February 1, 2002; 99(3): 834 - 839.
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P. L. Greenberg, N. S. Young, and N. Gattermann
Myelodysplastic Syndromes
Hematology, January 1, 2002; 2002(1): 136 - 161.
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B. D. Cheson, J. M. Bennett, H. Kantarjian, C. A. Schiffer, S. D. Nimer, B. Lowenberg, R. M. Stone, M. Mittelman, G. F. Sanz, P. W. Wijermans, et al.
Myelodysplastic syndromes standardized response criteria: further definition
Blood, September 15, 2001; 98(6): 1985 - 1986.
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A. Raza, P. Meyer, D. Dutt, F. Zorat, L. Lisak, F. Nascimben, M. du Randt, C. Kaspar, C. Goldberg, J. Loew, et al.
Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes
Blood, August 15, 2001; 98(4): 958 - 965.
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A. Raza
Improve or abandon the standardized response criteria for myelodysplastic syndromes recommended by the International Working Group
Blood, July 1, 2001; 98(1): 251 - 251.
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D. P. Steensma, L. Letendre, A. Tefferi, B. D. Cheson, J. M. Bennett, and P. L. Greenberg
Clarifications to the standard neutrophil response criteria for clinical trials in myelodysplastic syndromes are needed
Blood, May 15, 2001; 97(10): 3321 - 3322.
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