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Efficacy of Azacitidine Among Treatment-Naive Patients With Higher-Risk Myelodysplastic Syndromes
According to a recent retrospective study, standard-of-care treatment azacitidine demonstrated low complete remission rates (CRR) and median overall survival (OS) as a frontline therapy among patients with high-risk myelodysplastic syndromes (HR-MDS).
This large, real-world observational cohort study evaluated azacitidine effectiveness among 382 treatment-naive patients with HR-MDS from a US electronic health record (EHR)–derived database. The measured primary endpoint was International Working Group (IWG) 2006-based CRR. Secondary endpoints included EHR-based CRR, IWG 2006- and EHR-based objective response rates (ORRs), duration of complete response (CR), duration of response (DOR), progression-free survival (PFS), time-to-next-treatment, and overall survival (OS).
Using IWG 2006 criteria, study results demonstrated the CRR was 7.9%; median duration of CR was 12 months (95% confidence interval [CI], 7.7 to 15.6). In poor cytogenetic risk and TP53 mutation subgroups, CRRs were 7.9% and 8.7%, respectively. The ORR was 62.8%, which included a hematologic improvement rate (HIR) of 46.9%.
Using EHR-based data, it was noted that the CRR was 3.7%; median duration of CR was 13.5 months (95% CI, 4.5 to 21.5). The ORR was 67.8%, which included an HIR of 29.3%. The recorded median follow-up was 12.9 months, and the median OS was 17.9 months (95% CI, 15.5 to 21.7).
Nishanthan Rajakumaraswamy, MBBS, MRCP, Foster City, California, and colleagues concluded, “Consistent with other studies, CRRs and median OS with [azacitidine] 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,” they added.
Source:
Rajakumaraswamy N, Gandhi M, Wei A, et al. Real-world effectiveness of azacitidine in treatment-naive patients with higher-risk myelodysplastic syndromes. Clinical Lymphoma, Myeloma & Leukemia. Published online December 19, 2023. doi: 10.1016/j.clml.2023.12.008
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