Core Outcome Measures in Effectiveness Trials

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

General Information

Abstract:
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. (Blood. 2000;96:3671-3674)

Authors:
Cheson, B. D. Bennett, J. M. Kantarjian, H. Pinto, A. Schiffer, C. A. Nimer, S. D. Lowenberg, B. Beran, M. de Witte, T. M. Stone, R. M. Mittelman, M. Sanz, G. F. Wijermans, P. W. Gore, S. Greenberg, P. L. World Health Organization international working, group

Publication

Journal:
Blood
Volume:
96
Issue:
12
Pages:
3671 - 4
Year:
2000
DOI:
Further Study Information

Date:
Not stated
Funding source(s):
None

Health Area

Disease Category
Cancer

Disease Name
Myelodysplastic syndromes (MDS)

Target Population

Age Range
-

Sex
Either


Nature / type of Intervention
Not specified

Method(s)

Unknown

Report of a working group


Stakeholders Involved

Clinical experts

Study Type

COS for clinical trials or clinical research

The site uses cookies, some may have been set already. Please refer to our privacy policy & cookie usage statement.
If you continue to use the site we'll assume you're happy to accept the cookies.