Bone marrow hypocellularity does not affect tolerance or efficacy of azacitidine in patients with higher‐risk myelodysplastic syndromes. (28th January 2014)

• Record Type: Journal Article
• Authors: Seymour, John F.; Bennett, John M.; List, Alan F.; Mufti, Ghulam J.; Gore, Steven D.; Fenaux, Pierre; Santini, Valeria; Hetzer, Joel; Songer, Stephen; Skikne, Barry S.; Beach, Charles L.
• Is Part Of: British journal of haematology. Volume 165:Number 1(2014:Apr.)
• Page Start: 49
• Page End: 56
• Publication Date: 2014-01-28
• Subjects: Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.12723
• Languages: English
• ISSNs: 0007-1048

Abstract

<abstract abstract-type="main" id="bjh12723-abs-0001"> <title>Summary</title> <p>The efficacy and tolerance of azacitidine in higher‐risk myelodysplasia with hypocellular bone marrow (BM) are unknown. This <italic>post hoc </italic>AZA‐001 trial analysis assessed whether baseline BM cellularity affected the overall survival (OS) advantage demonstrated with azacitidine <italic>versus</italic> conventional care regimens (CCR). Baseline BM biopsies of <30% cellularity were considered hypocellular with data evaluable from 299 patients (azacitidine <italic>n</italic> = 154, CCR <italic>n</italic> = 145); 13% (<italic>n</italic> = 39) hypocellular, 87% (<italic>n</italic> = 260) non‐hypocellular. Patient characteristics were balanced between cellularity and treatment groups. Most patients (90–100%) had 2–3 cytopenias at baseline. Median (range) azacitidine treatment cycle lengths were 35·5 (28–54) and 33·0 (15–75) d in hypocellular and non‐hypocellular groups, respectively. At 33 months, median OS was not reached (NR) [95% confidence interval (CI): 19·2, NR] in hypocellular patients receiving azacitidine <italic>versus</italic> 16·9 months (95% CI: 11·1, 19·3) with CCR (<italic>P</italic> = 0·001); and in non‐hypocellular patients, it was 21·1 months (95% CI: 16·2, 34·7) <italic>versus</italic> 15·3 months (95% CI: 9·3, 17·6) (<italic>P</italic> = 0·012). Azacitidine tolerance was similar regardless of cellularity. Grade 3–4 thrombocytopenia and neutropenia occurred similarly in hypocellular patients treated with azacitidine <italic>versus </italic>CCR (80% vs. 92% and 88% vs. 75%). Azacitidine OS results are consistent with those from AZA‐001, regardless of cellularity, and demonstrate its safety and efficacy in higher‐risk myelodysplasia with hypocellular BM.</p> </abstract>