Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome: the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective

Kód výsledku v 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>

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome: the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective

Popis výsledku v původním jazyce

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.

Název v anglickém jazyce

Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome: the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective

Popis výsledku anglicky

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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30205 - Hematology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2020

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Haematologica

ISSN

0390-6078

e-ISSN

—

Svazek periodika

105

Číslo periodika v rámci svazku

11

Stát vydavatele periodika

IT - Italská republika

Počet stran výsledku

8

Strana od-do

2516-2523

Kód UT WoS článku

000583303700009

EID výsledku v databázi Scopus

2-s2.0-85093539032