EUROPEAN PATTERN AND IMPACT OF THE USE ERYTHROPOIETIC AGENTS (ESAS)IN LOW AND INT-1 RISK MDS WITH PROPENSITY SCORING TECHNIQUES IN OBSERVATIONAL LONGITUDINAL STUDIES

Konference: 2013 18th Congress of the European Hematology Association - účast ČR

Kategorie: Myelodysplastický syndrom

Téma: Myelodysplastic syndromes - Clinical

Číslo abstraktu: P202

Autoři: Hege Garelius; W. Thomas Johnston; M.D. Arjan A. van de Loosdrecht, Ph.D.; Sophie Park; MD Louise de Swart; MD Reinhard Stauder; Dr. Argyris Symeonidis, Ph.D.; MD Guillermo F Sanz, PhD; prof. MUDr. Jaroslav Čermák, CSc.; Otilia Georgescu; MD Mette Skov-Holm, PhD; MD Ulrich Germing; MD Pierre Fenaux, PhD; Marius MacKenzie; MD Luca Malcovati; MD Krzysztof Madry, PhD; MD Antonio Medina Almeida, PhD; MD David Bowen, Ph.D.; MD Theo M. de Witte; Alexandra Smith; MD Eva Hellström-Lindberg

Background:

The EU MDS Registry is a non-interventional, observational longitudinal study enrolling patients with lower-risk MDS <3 months from diagnosis. Between 2008 and 2011 1000 patients were recruited from 14 countries and 118 sites, with a follow-up every 6 months.

Aims:

This substudy aims to describe the usage of erythropoietic agents (ESAs), and the impact of treatment on overall survival and transfusion patterns in anemic patients. 

Methods:

Two cohorts were investigated; all patients for distribution of ESA usage, and patients with hemoglobin levels <10 g/dl at registration for the evaluation of outcome. The effect of ESA treatment on survival and other outcomes was assessed using a proportional hazards model.  To account for the non-randomised use of ESA within the cohort, a propensity score for receiving ESA treatment was derived using logistic regression and included in the proportional hazards modeling.

Results:

he ESA use varies by country, varying from 16.7% in Poland to 61.1% of the patients in Denmark, as did the pre-treatment Hb levels (range: 4.6-14.9 g/dL). Of the first 1000 patients (median age 74.6 years, range=18.7-95.3), 487 patients (median age 75.8, range: 42-95) were ESA-treated.  The median duration of treatment was 8.1 months (0-48 months). The majority of patients started ESA within 6 months after their first visit. In a multivariable logistic regression model, the strongest predictors of receiving ESA were country, WHO diagnosis subtype, baseline hemoglobin, the percentage of bone marrow blasts and age at diagnosis.  

The outcome cohort consisted of 419 patients; 260 received ESA and 159 were untreated before their second visit. For a treated patient to be classed as a responder there had to be at least 28 days between the start of ESA treatment and their second visit, with an increased Hb to >10g/dL or an increase of at least 1.5 g/dL. A patient with transfusions prior to receiving ESA was considered a responder if the transfusion need was abrogated for at least 56 days. Responders appear to have a longer time to first post-ESA transfusion. (p<0,0011) ( Fig1) Median time to transfusion in patients transfused before start of ESA was 5 months (IQR: 2.8-30 months) compared to 15 months (IQR: 3.9-47 months) in patients with no prior transfusions (p=0.0064).

The proportional hazards model shows a (non-significant) decreased risk of death (HR=0.74, 95% CI:0.51-1.08, p=0.11) associated with ESA treatment. 

 Abstrakta v časopise Haematologica 2013, Suppl1

 Online Program

Datum přednesení příspěvku: 14. 6. 2013