Purpose: The impact of azacytidine (Aza C) on the quality of life of 191 patients with myelodysplastic syndrome was assessed in a phase III Cancer and Leukemia Group B trial (9221).

Patients and Methods: One hundred ninety-one pa-tients (mean age, 67.5 years; 69% male) were randomized to receive either Aza C (75 mg/m2 subcutaneous for 7 days every 4 weeks) or supportive care, with supportive care patients crossing over to Aza C upon disease progression. Quality of life was assessed by centrally conducted telephone interviews at baseline and days 50, 106, and 182. Overall quality of life, psychological state, and social functioning were assessed by the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire C30 and the Mental Health Inventory (MHI).

Results: Patients on the Aza C arm experienced signif-icantly greater improvement in fatigue (EORTC, P. 001), dyspnea (EORTC, P. 0014), physical functioning (EORTC, P. 0002), positive affect (MHI, P. 0077), and psychological distress (MHI, P. 015) over the course of the study period than those in the supportive care arm. Particularly striking were improvements in fatigue and psychological state (MHI) in patients treated with Aza C compared with those receiving supportive care for patients who remained on study through at least day 106, corresponding to four cycles of Aza C. Significant differences between the two groups in quality of life were maintained even after controlling for the number of RBC transfusions.

Conclusion: Improved quality of life for patients treated with Aza C coupled with significantly greater treatment response and delayed time to transformation to acute myeloid leukemia or death compared with patients on supportive care (P<. 001) establishes Aza C as an important treatment option for myelodysplastic syndrome.