Association of Changes in Transfusion Status with Changes in Health-Related Quality of Life of Real-World Patients with MDS Across Six Months of Treatment with Azacitidine
Authors: Pashos Chris L. , Grinblatt David L. , Sekeres Mikkael A. , Komrokji Rami S. , Narang Mohit , Swern Arlene S , Street Thomas K. , Sullivan Kristen A , Harding Gale , Khan Zeba M. 
Published: October 2019

Abstract
Abstract 2796

Introduction:
The health-related quality of life (HRQOL) of patients with myelodysplastic syndromes (MDS) is worse if they are red blood cell transfusion dependent (RBC TD) than if they are RBC transfusion independent (TI). Little is known whether a change in status from RBC TD to RBC TI is associated with improved HRQOL. This analysis characterized the HRQOL of real-world patients with MDS across 6 months of treatment with Azacitidine (AZA) by their RBC TD/TI status.

Methods:
Data were collected from AVIDA®, a prospective, US, community-based registry of patients treated with AZA. Patients with MDS who were originally RBC TD at baseline, and who received 56 days or more of AZA were analyzed. RBC TD, defined as having received > 1 RBC transfusion within 56 consecutive days, was determined and verified centrally. Clinicians provided data on patient demographics and clinical characteristics, including RBC transfusions. Patients reported HRQOL by completing the EORTC-QLQ-C30 instrument at baseline and quarterly thereafter. Summary statistics (e.g., mean scores and changes in scores) on global health status, five functional scales, and nine symptom/other scales were analyzed. Statistical significance was ascertained by ANOVA using SAS 9.1.

Results:
In the full AVIDA cohort, 328 MDS patients received at least 56 days of treatment with AZA, of whom 153 reported HRQOL data at baseline and at six months. At baseline, 85 of the 153 were RBC TD, while the rest were RBC TI. At six months, 41 of the 85 had become RBC TI, while 44 remained RBC TD. Global health status improved among those who became RBC TI, but declined among those who stayed RBC TD. Statistically significant and clinically meaningful (i.e., greater than 7 points) differences in change between baseline and 6 months also were seen in physical and role function, but not in emotional, cognitive or social function. Fatigue was the only symptom score in which changes were statistically significantly different between groups, with RBC TI patients reporting less fatigue, and RBC TD patients reporting more.

Conclusions:
Findings from AVIDA® indicate that HRQOL among RBC TD MDS patients treated with AZA improves significantly overall and on certain domains if they achieve RBC transfusion independence. The improved global health status; better physical and role functioning; and less fatigue associated with RBC TD patients achieving RBC transfusion independence should be recognized by US clinicians as they manage patients with MDS.

Disclosures:
Pashos: Celgene: Membership on an entity's Board of Directors or advisory committees. Grinblatt:Celgene: Membership on an entity's Board of Directors or advisory committees. Sekeres:Celgene: Consultancy, Honoraria, Speakers Bureau. Komrokji:Celgene: Honoraria, Research Funding, Speakers Bureau. Narang:Celgene: Membership on an entity's Board of Directors or advisory committees. Swern:Celgene Corporation: Employment, Equity Ownership. Street:Celgene: Employment, Equity Ownership. Sullivan:Celgene: Employment, Equity Ownership. Khan:Celgene: Employment, Equity Ownership.