ArticleAmerican journal of hematology2018
Extended dosing with CC-486 (oral azacitidine) in patients with myeloid malignancies.
Article in American journal of hematology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 29 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
AZA PH GL 2003 CL 001 - Extension A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)
A phase 1, multicenter, open-label study to evaluate the pharmacokinetics and effect of food of a new tablet formulation of oral azacitidine, and to evaluate the safety and efficacy of oral azacitidine in subjects with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia
Phase 1b/2a Safety and Immunogenicity of the DNMT Inhibitor Azacitidine During Anti-Tuberculosis Therapy
Who cites it
29 citing papers in PubMed.
- Management of adverse events in patients with acute myeloid leukemia in remission receiving oral azacitidine: experience from the phase 3 randomized QUAZAR AML-001 trial.Journal of hematology & oncology · 2021Trial
- An open-label, phase II multicohort study of an oral hypomethylating agent CC-486 and durvalumab in advanced solid tumors.Journal for immunotherapy of cancer · 2020Trial
- Spray-Drying Process Optimization and Modeling for an Inhaled Dry Powder of 5-Azacytidine for Treating Local and Metastatic Lung Cancer.Molecular pharmaceutics · 2025Article
- Epigenetics-targeted drugs: current paradigms and future challenges.Signal transduction and targeted therapy · 2024Review
- Clinical activity, pharmacokinetics, and pharmacodynamics of oral hypomethylating agents for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: A multidisciplinary review.Journal of oncology pharmacy practice : official publication of the International Society of Oncology Pharmacy Practitioners · 2024Review
- New Perspectives in Treating Acute Myeloid Leukemia: Driving towards a Patient-Tailored Strategy.International journal of molecular sciences · 2022Review
- Pedigree investigation, clinical characteristics, and prognosis analysis of haematological disease patients with germline TET2 mutation.BMC cancer · 2022Article
- Article
- The genetics of myelodysplastic syndromes and the opportunities for tailored treatments.Frontiers in oncology · 2022Review
- Oral hypomethylating agents: beyond convenience in MDS.Hematology. American Society of Hematology. Education Program · 2021Article
- Silylation of Deoxynucleotide Analog Yields an Orally Available Drug with Antileukemia Effects.Molecular cancer therapeutics · 2021Article
- Myelodysplastic Syndromes in the Postgenomic Era and Future Perspectives for Precision Medicine.Cancers · 2021Review
- Treatment advances for pediatric and adult onset neoplasms with monocytosis.Current hematologic malignancy reports · 2021Review
- Review
- 5-Azacytidine Transiently Restores Dysregulated Erythroid Differentiation Gene Expression in TET2-Deficient Erythroleukemia Cells.Molecular cancer research : MCR · 2021Article
- Molecular Targeted Therapy in Myelodysplastic Syndromes: New Options for Tailored Treatments.Cancers · 2021Review
- Advances in Acute Myeloid Leukemia: Recently Approved Therapies and Drugs in Development.Cancers · 2020Review
- Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes?Leukemia & lymphoma · 2020Review
- Epigenetic Attire in Ovarian Cancer: The Emperor's New Clothes.Cancer research · 2020Review
- Oral Azacitidine and Cedazuridine Approximate Parenteral Azacitidine Efficacy in Murine Model.Targeted oncology · 2020Article
Corrections and comments
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Authors and funding
12 authors.
Funding
Abstract
CC-486 (oral azacitidine) is an epigenetic modifier in clinical development for treatment of hematological cancers. This study of extended CC-486 dosing included patients with myelodysplastic syndromes (MDSs), chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML). After a pharmacokinetic assessment period, 31 patients (MDS n = 18, CMML n = 4, and AML n = 9) entered a clinical phase in which they received CC-486 300 mg once-daily for 21 days of repeated 28-day cycles. Median age was 71 years (range: 53-93); 42% of patients were aged ≥75 years. A total of 5 patients with AML (63%) had prior MDS. Median number of CC-486 treatment cycles was 4 (range: 1-32). The most common treatment-emergent adverse events (TEAEs) were gastrointestinal (84% of patients) and hematologic (81%). Most common grade 3-4 TEAEs were neutropenia (n = 13, 42%) and anemia (n = 9, 29%). Ten patients experienced grade 4 neutropenia. Infrequently, CC-486 dose was interrupted or reduced due to gastrointestinal (n = 5, 16%) or hematologic (n = 6, 19%) TEAEs. Overall response rate (complete remission [CR], CR with incomplete hematological recovery [CRi], partial remission [PR], marrow CR) in the MDS/CMML subgroups was 32% and in the AML subgroup (CR/CRi/PR) was 22%. Red blood cell transfusion independence rates in the MDS/CMML and AML subgroups were 33% and 25%, respectively, and 2 MDS/CMML patients attained hematologic improvement as a best response on-study. No baseline gene mutation was predictive of response/nonresponse. CC-486 allows flexible dosing and schedules to improve tolerability or response. Neutropenia in early treatment cycles deserves scrutiny and may warrant initiation of prophylactic antibiotics. KEY POINTS: The safety profile of oral CC-486 was comparable to that of injectable azacitidine; most adverse events were hematological and gastrointestinal. Extended (21-day/cycle) CC-486 dosing induced responses in patients with hematological malignancies, many of whom had prior DNMTi failure.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.