Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2021
Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes.
Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06004765 (A Prospective Single-center Study on the Efficacy and Safety of Lenalidomide Combined With Azacitidine vs Azacitidine in the Treatment of MDS-RS), which is not on this map. Cited by 42 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
A Prospective Single-center Study on the Efficacy and Safety of Lenalidomide Combined With Azacitidine vs Azacitidine in the Treatment of MDS-RS
Who cites it
42 citing papers in PubMed, 2 syntheses or guidelines pooled it, 76 citations in OpenAlex.
- Comparative efficacy and safety of pharmacological treatments for lower-risk myelodysplastic syndromes: a Systematic Review and network meta-analysis.Frontiers in oncology · 2026Pooled it
- Patient-Reported Outcomes in Phase 3 Clinical Trials for Blood Cancers: A Systematic Review.JAMA network open · 2024Pooled it
- A randomized, phase II trial of oral azacitidine (CC-486) in patients with resected pancreatic adenocarcinoma at high risk for recurrence.Clinical epigenetics · 2022Trial
- FDA Approval Summary: Oral Azacitidine for Continued Treatment of Adults with Acute Myeloid Leukemia Unable to Complete Intensive Curative Therapy.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022Trial
- 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
- Novel and emerging therapeutic strategies for clonal hematopoiesis.Haematologica · 2026Review
- DNA methyltransferase inhibitors in hematological malignancies and solid tumors.International journal of cancer · 2026Review
- Molecular Insights and Therapeutic Advances in Low-Risk Myelodysplastic Neoplasms: A Clinical Review.Cancers · 2025Review
- Erythropoietin-dependent Acquisition of CD71hiCD105hi Phenotype within CD235a- Early Erythroid Progenitors.Stem cells (Dayton, Ohio) · 2025Article
- An unconquered challenge in MDS: review of pathophysiology, clinical manifestations, and management options of MDS with thrombocytopenia.Annals of hematology · 2025Review
- Oxidative Stress and Mitochondrial Dysfunction in Myelodysplastic Syndrome: Roles in Development, Diagnosis, Prognosis, and Treatment.International journal of molecular sciences · 2025Review
- The epigenetic revolution in hematology: from benchside breakthroughs to clinical transformations.Clinical and experimental medicine · 2025Review
- Recent advances on gene-related DNA methylation in cancer diagnosis, prognosis, and treatment: a clinical perspective.Clinical epigenetics · 2025Review
- Treating Hematological Malignancies With OR-2100, an Orally Bioavailable Prodrug of Decitabine.Cancer science · 2025Review
- Treatment of high-risk myelodysplastic syndromes.Haematologica · 2025Review
- Hotspot gene mutations and treatment response in myelodysplastic syndromes (MDS): predictive biomarkers and targeted strategies.Frontiers in medicine · 2025Review
- Infections and antimicrobial prophylaxis in patients with myelodysplastic syndromes.Seminars in hematology · 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
- Treatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities.Current hematologic malignancy reports · 2024Review
- Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome.Current treatment options in oncology · 2024Review
Corrections and comments
- Commented on by
Authors and funding
28 authors at 20 institutions in 13 countries.
Funding
Abstract
purposeTreatment options are limited for patients with lower-risk myelodysplastic syndromes (LR-MDS). This phase III, placebo-controlled trial evaluated CC-486 (oral azacitidine), a hypomethylating agent, in patients with International Prognostic Scoring System LR-MDS and RBC transfusion-dependent anemia and thrombocytopenia.
methodsPatients were randomly assigned 1:1 to CC-486 300-mg or placebo for 21 days/28-day cycle. The primary end point was RBC transfusion independence (TI).
resultsTwo hundred sixteen patients received CC-486 (n = 107) or placebo (n = 109). The median age was 74 years, median platelet count was 25 × 10
conclusionCC-486 significantly improved RBC-TI rate and induced durable bilineage improvements in patients with LR-MDS and high-risk disease features. More early deaths occurred in the CC-486 arm, most related to infections in patients with significant pretreatment neutropenia. Further evaluation of CC-486 in MDS is needed.
Indexed as
Identifiers
What OpenQuestion holds
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.