Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome: the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00023736%3A_____%2F20%3A00013173" target="_blank" >RIV/00023736:_____/20:00013173 - isvavai.cz</a>
Result on the web
<a href="https://doi.org/10.3324/haematol.2020.266817" target="_blank" >https://doi.org/10.3324/haematol.2020.266817</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.3324/haematol.2020.266817" target="_blank" >10.3324/haematol.2020.266817</a>
Alternative languages
Result language
angličtina
Original language name
Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome: the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective
Original language description
Available evidence suggests that in most patients with LR-MDS the risk of death is not related to disease progression but is mainly attributable to non-leukemic death. In addition, a proportion of these patients have prolonged survival that precludes the design of clinical trials adopting OS as a primary endpoint. These challenges have resulted in potentially biased assessment of the effectiveness and appropriate use of the available interventions in this patient population. The EUMDS Registry has identified novel meaningful outcome indicators and clinical endpoints, and reliable measures of response to HCI. The results of our analysis indicate that RBCT density is strongly associated with a decreased OS, even at relatively low dose densities. In addition, we observed that an early decrease in platelet count is an independent adverse prognostic indicator in LR-MDS, and combining relative platelet drop and transfusion dependency allows early identification of patients at risk of rapid progression, and may guide early therapeutic interventions, including allogeneic hematopoietic stem cell transplantation or experimental interventions. Taken together, these results indicate that regular RBCT requirement, early platelet count kinetics, and restriction in HRQoL are early independent and meaningful outcome indicators, and reliable measures of effectiveness of therapeutic interventions, evaluated in this set of studies.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30205 - Hematology
Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2020
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Haematologica
ISSN
0390-6078
e-ISSN
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Volume of the periodical
105
Issue of the periodical within the volume
11
Country of publishing house
IT - ITALY
Number of pages
8
Pages from-to
2516-2523
UT code for WoS article
000583303700009
EID of the result in the Scopus database
2-s2.0-85093539032