Real-world effectiveness of azacitidine in treatment-naive patients with higher-risk myelodysplastic syndromes

Journal article

Introduction

Azacitidine (AZA) is an approved frontline therapy for higher-risk myelodysplastic syndromes (HR-MDS); however, poor survival denotes unmet needs to increase depth/duration of response (DOR).

Methods

This retrospective study with patient chart review evaluated AZA effectiveness in 382 treatment-naive patients with HR-MDS from a US electronic health record (EHR)–derived database. Responses were assessed using International Working Group (IWG) 2006 criteria; real-world equivalents were derived from EHRs. Primary endpoint was IWG 2006-based complete remission rate (CRR). Secondary endpoints were EHR-based CRR, IWG 2006- and EHR-based objective response rates (ORRs), duration of CR, DOR, progression-free survival, time-to-next-treatment, and overall survival (OS).

Results

Using IWG 2006 criteria, the CRR was 7.9% (n = 30); median duration of CR was 12.0 months (95% CI, 7.7-15.6). In poor cytogenetic risk (n = 101) and TP53 mutation (n = 46) subgroups, CRRs were 7.9% (n = 8) and 8.7% (n = 4), respectively. ORR was 62.8% (n = 240), including a hematologic improvement rate (HIR) of 46.9% (n = 179). Using EHR-based data, CRR was 3.7% (n = 14); median duration of CR was 13.5 months (95% CI, 4.5-21.5). ORR was 67.8% (n = 259), including an HIR of 29.3% (n = 112). Median follow-up was 12.9 months; median OS was 17.9 months (95% CI, 15.5-21.7).

Conclusions

Consistent with other studies, CRRs and median OS with AZA in treatment-naive patients with HR-MDS were low in this large, real-world cohort. Novel agents/combinations are urgently needed to improve these outcomes in HR-MDS.

Authors: Rajakumaraswamy, N; Gandhi, M; Wei, AH; Sallman, DA; Daver, NG

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Elsevier
• Journal: Clinical Lymphoma, Myeloma and Leukemia
• Volume: 24
• Issue: 4
• Pages: 260-268.e2
• Place of publication: United States
• Publication date: 2023-12-20
• Acceptance date: 2023-12-14
• DOI: 10.1016/j.clml.2023.12.008
• EISSN: 2152-2669
• ISSN: 2152-2650
• Pmid: 38216397
• Language: English
• Keywords: HR-MDS; real-world data; MDS