AVIDA: A Longitudinal Registry of Clinical and Quality of Life Outcomes in Patients with Hematologic Disorders Receiving Azacitidine.

Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid neoplasms characterized by ineffective hematopoiesis and peripheral cytopenias. Treatment decisions are often based on age, performance status (PS), cytopenias, International Prognostic Scoring System (IPSS) classification, and MDS subtype. Patient-reported results from a few clinical trials suggest that MDS can have a negative effect on a patient's quality of life (QoL) with responses to treatment having a positive effect. Azacitidine (Vidaza) is a hypomethylating agent approved in the US for the treatment of MDS. In a phase III study, patients (pts) treated with azacitidine experienced significantly greater improvement in QoL compared with supportive care (Kornblith AB, et al. J Clin Oncol . 2002; 20:2441). Evaluation of QoL in MDS pts treated in community-based hematology clinics is not well characterized. Azacitidine is approved for a dosing schedule of 75 mg/m2/day x 7 day q 28 days. However, the dose and schedule of azacitidine used in clinical practice varies. AVIDA is a unique, longitudinal, multicenter patient registry designed to prospectively collect data from community-based hematology clinics on the natural history and management of pts with MDS and other hematologic disorders, including acute myeloid leukemia, who are treated with azacitidine. It aims to further the understanding of current azacitidine treatment patterns in the community, identify common care procedures and concomitant treatments, explore correlation between duration and number of treatment cycles with ongoing clinical response, and to investigate the effect of azacitidine on patient satisfaction and QoL. Patient-reported QoL will be based on the EORTC QLQ-C30 questionnaire with QoL measures obtained at baseline and at quarterly intervals for 2 years. Scores on the EORTC QLQ-C30 range from 0 to 100. Higher scores on the global health and functioning scales indicate better QoL in each measure/domain. Lower scores on the symptom and single-item scales indicate less impairment due to that symptom/single item. To date, 47 pts (34 males, 13 females; mean age, 73.2 years) with predominantly low-risk MDS have been enrolled in the registry. The majority (90%) is white and has an ECOG PS of 0 or 1. Median time from first MDS diagnosis is 2.2 months (mean, 14 months); 43 have primary and 4 have secondary MDS. IPSS is known for 36 pts; low for 8 pts, intermediate-1 for 21 pts, intermediate-2 for 6 pts, and high for 1 patient. Baseline QoL data are currently available for 42 pts. At baseline, pts reported a lower level (mean score) of global health (52), physical functioning (66) and role functioning (61) compared with cognitive (81), emotional (76), and social (71) functioning. Among the symptom/single item scales, fatigue scored the worst with a mean score of 48. Other symptom/single item scales that indicated pts were experiencing a moderate level of the measure included dyspnea (37), insomnia (29), and pain (22). Constipation (17), appetite loss (15), financial difficulties (15), and nausea/vomiting (8) were reported at lower levels. Ongoing results from this patient registry will provide insight into the QoL of pts with MDS and other hematologic malignancies in the real-world setting, and will explore any change in QoL associated with treatment and/or disease progression.