Indirect Treatment Comparison of Oral Versus Injectable Azacitidine as Maintenance Therapy for Acute Myeloid Leukemia

Abstract

Aim: Evaluate the relative efficacy of oral versus injectable azacitidine (AZA) maintenance therapy in acute myeloid leukemia (AML) after complete remission. Materials & methods: Systematic literature review identified QUAZAR AML-001, HOVON 97 AML, UK NCRI AML16 and QoLESS-AZA-AMLE (sensitivity analysis) trials. Network meta-analysis and matching-adjusted indirect comparisons assessed survival outcomes. Results: In the network meta-analysis, combining the HOVON 97 and UK NCRI trials, oral AZA (QUAZAR) was associated with significantly improved overall survival (OS) versus injectable AZA (hazard ratio: 0.744; 95% credible interval: 0.557–0.998). After matching-adjusted indirect comparisons, to address differences in patient characteristics across trials, OS improvements were maintained with oral versus injectable AZA (hazard ratio: 0.753; credible interval: 0.563–0.998). Conclusion: In AML, maintenance therapy with oral AZA was associated with improved OS versus injectable AZA.

Transplant Eligible and Ineligible Elderly Patients with AML—A Genomic Approach and Next Generation Questions

“…the success encountered with the development and approval of oral decitabine/ cedazuridine (DEC-C, Inqovi ® , Otsuka Pharmaceutical Co., Pleasanton, CA, USA) for MDS and CMML, preliminary animal data of the oral DNA methyltransferase inhibitor (DNMTi) azacitidine (AZA) in combination with cedazuridine suggests a comparable pharmacokinetic to intravenous AZA [170,171]. Phase 2 clinical trials are currently accruing in the United States evaluating the clinical efficacy in MDS, CMML, and AML.…”