FDA Approval Summary: Oral Azacitidine For Continued Treatment of Adults with Acute Myeloid Leukemia Unable to Complete Intensive Curative Therapy
Cette étude analyse les données de l'essai ayant conduit la "Food and Drug Administration" à autoriser l'utilisation de l'azacitidine par voie orale pour traiter les patients atteints d'une leucémie myéloïde aiguë et dont le traitement intensif standard ne peut être achevé
On September 1, 2020, the FDA granted approval for oral azacitidine (Onureg, CC-486) for continued treatment of adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy. Approval was based on improvement in overall survival using CC-486 300 mg daily in a two weeks on/two weeks off schedule in comparison to placebo (HR 0.69 [95% CI: 0.55, 0.86], p = 0.0009) in the randomized trial CC‑486-AML-001 (QUAZAR) in adults {greater than or equal to} 55 years old with AML in CR/CRi who did not complete standard intensive induction and post-remission therapy. Of note, the study was not designed to test CC-486 as maintenance after standard post-remission therapy nor as an alternative to standard post-remission therapy. Gastrointestinal toxicities, fatigue, and pneumonia were more common in patients treated with CC-486 compared to placebo. Additional studies are needed to establish safe dosing for patients with hepatic impairment. The pharmacokinetic parameters, recommended dose, and recommended schedule of CC-486 differ substantially from those of other azacitidine formulations; therefore, inappropriate substitutions between formulations pose a considerable risk for harm.
Clinical Cancer Research , résumé, 2021