Darbepoetin alfa for the treatment of anemia associated with myelodysplastic syndromes: efficacy and quality of life

Abstract

To evaluate efficacy, safety, changes in biological features, and quality of life (QoL) in low-risk anemic patients with MDS treated with darbepoetin alfa (DPO), 41 patients received DPO 150 microg weekly for 24 weeks. The dose was increased to 300 microg weekly in non-responsive patients. During treatment, 10/17 (59%) transfusion-dependent (TD) and 13/23 (56%) transfusion-free (TF) patients responded. In TF patients, Hb increased from 9.2 +/- 0.9 g/dL to 10.3 +/- 1.4 g/dL by 24 weeks (p = 0.004). The mean response duration was 22 weeks (95% CI: 19.7-24.0) in TF patients compared with 15.1 weeks (95% CI: 13.3-17.5) in TD patients. Response to treatment was associated with increases in QoL. Decreases in the percentage of apoptotic progenitor cells (p = 0.007) and CD34+ cells (p = 0.005) were observed. These results confirm previous studies demonstrating the safety and efficacy of DPO in anemic patients with MDS. Biological changes and improvement in QoL were associated with response. Adequate dosing is to be determined.

Development and validation of QOL-E© instrument for the assessment of health-related quality of life in myelodysplastic syndromes

“…Several studies have reported a positive correlation between Hb levels and HRQoL [9,19,20,[24][25][26][27][28][29][30][31], although this correlation is not consistent [6]. The current data reinforce the notion of a relationship between Hb levels and QOL-E© as specific patient-reported outcome in MDS patients.…”

“…QOL-E© V2.0 data was collected cross-sectionally at baseline in all studies, alongside other measures of clinical effect and HRQoL, such as the EORTC QLQ-C30 questionnaire. Some cross-sectional and longitudinal data have been reported separately [19][20][21][22].…”

Erythropoiesis-stimulating agents in myelodysplastic syndromes

“…With this background, it is important to further explore the potentially positive effects of pro-erythroid growth factors in MDS, both on survival and on quality of life. Oliva et al report in the current issue of Leukemia and Lymphoma on 41 patients receiving darbepoetin treatment for MDS-related anemia [10]. The response rate in both transfusion-independent patients and those with a moderate transfusion need was relatively high, 450%, and the study corroborates previous results that treatment is associated with decreased apoptosis of bone marrow progenitors.…”

Validation of Disability Status, a Claims-based Measure of Functional Status for Cancer Treatment and Outcomes Studies

“…While there is no evidence that ESA use modifies survival, it is associated with improved quality of life. 16–18 While ESAs are not contraindicated for individuals with poor PS, poor PS may interfere with the ability to travel to a physician office to receive therapy.…”