A retrospective study of azacitidine treatment in patients with intermediate-2 or high risk myelodysplastic syndromes in a real-world clinical setting in Greece

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• 作者：Vasiliki Pappa ; Achilles Anagnostopoulos…
• 关键词：Myelodysplastic syndromes ; Azacitidine ; Response rate ; Hematologic improvement ; AML transformation ; Safety
• 刊名：International Journal of Hematology
• 出版年：2017
• 出版时间：February 2017
• 年：2017
• 卷：105
• 期：2
• 页码：184-195
• 全文大小：
• 刊物主题：Hematology; Oncology;
• 出版者：Springer Japan
• ISSN：1865-3774
• 卷排序：105

文摘

For patients with intermediate-2 or high risk [according to the International Prognostic Scoring System (IPSS)] myelodysplastic syndromes (MDS), azacitidine treatment offers hematologic improvement (HI) but also has the potential to modify the natural disease course. ‘RETRO-AZA-MDS-001’, a retrospective chart review study was conducted from February to November 2012 across 17 hematology hospital sites of Greece, aiming to evaluate the clinical efficacy and safety profile of azacitidine in IPSS intermediate-2/high risk adult MDS patients in routine care. A total of 88 patients (median age 74.7 years), with a 6.6 month median (range 1.0–49.5) azacitidine treatment duration were enrolled. The overall response rate [complete response (CR), marrow CR and partial response] was 37.7% (23/61), while stable disease with HI was achieved by 21.3% (13/61). The HI rate was 33.0 % (29/88) and the AML transformation rate 6.8% (6/88). Of the transfusion-dependent patients, 7.3% (3/41) became transfusion-independent during azacitidine treatment. The incidence of non-serious and serious adverse events related to azacitidine was 50.0 and 42.0%, respectively. Patients not receiving prior ESA therapy were expected to be 7.6 times more likely to achieve a clinical response (p = 0.012). The study corroborates the favorable risk–benefit profile of azacitidine for intermediate-2/high risk MDS patients in routine clinical practice.