Trial reportHaematologica2016
A randomized phase II trial of azacitidine +/- epoetin-β in lower-risk myelodysplastic syndromes resistant to erythropoietic stimulating agents.
Trial report in Haematologica, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 4 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 4 syntheses or guidelines pooled it, 61 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
- Intravenous iron versus oral iron versus no iron with or without erythropoiesis- stimulating agents (ESA) for cancer patients with anaemia: a systematic review and network meta-analysis.The Cochrane database of systematic reviews · 2022Pooled it
- Pooled it
- Pooled it
- The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort.Blood advances · 2018Trial
- Management of Anemia in Lower-Risk Myelodysplastic Syndromes/Neoplasms With Novel Agents.JCO oncology practice · 2026Review
- Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome.Current treatment options in oncology · 2024Review
- Current Therapeutic Landscape in Lower Risk Myelodysplastic Syndromes.Current treatment options in oncology · 2023Review
- Review
- Review
- New Approaches to Myelodysplastic Syndrome Treatment.Current treatment options in oncology · 2022Review
- Past, present and future in low-risk myelodysplastic syndrome.Frontiers in medicine · 2022Review
- Review
- Treatment options for lower-risk myelodysplastic syndromes. Where are we now?Therapeutic advances in hematology · 2021Review
- Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options.Cancer management and research · 2021Review
- Therapy for lower-risk MDS.Hematology. American Society of Hematology. Education Program · 2020Article
- Patient stratification in myelodysplastic syndromes: how a puzzle may become a map.Hematology. American Society of Hematology. Education Program · 2020Article
- Mutations in Splicing Factor Genes in Myeloid Malignancies: Significance and Impact on Clinical Features.Cancers · 2019Review
- Clinical update on hypomethylating agents.International journal of hematology · 2019Review
- Transforming growth factor (TGF)-β pathway as a therapeutic target in lower risk myelodysplastic syndromes.Leukemia · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors at 18 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The efficacy of azacitidine in patients with anemia and with lower-risk myelodysplastic syndromes, if relapsing after or resistant to erythropoietic stimulating agents, and the benefit of combining these agents to azacitidine in this setting are not well known. We prospectively compared the outcomes of patients, all of them having the characteristics of this subset of lower-risk myelodysplastic syndrome, if randomly treated with azacitidine alone or azacitidine combined with epoetin-β. High-resolution cytogenetics and gene mutation analysis were performed at entry. The primary study endpoint was the achievement of red blood cell transfusion independence after six cycles. Ninety-eight patients were randomised (49 in each arm). Median age was 72 years. In an intention to treat analysis, transfusion independence was obtained after 6 cycles in 16.3% versus 14.3% of patients in the azacitidine and azacitidine plus epoetin-β arms, respectively (P=1.00). Overall erythroid response rate (minor and major responses according to IWG 2000 criteria) was 34.7% vs. 24.5% in the azacitidine and azacitidine plus epoetin-β arms, respectively (P=0.38). Mutations of the SF3B1 gene were the only ones associated with a significant erythroid response, 29/59 (49%) versus 6/27 (22%) in SF3B1 mutated and unmutated patients, respectively, P=0.02. Detection of at least one "epigenetic mutation" and of an abnormal single nucleotide polymorphism array profile were the only factors associated with significantly poorer overall survival by multivariate analysis. The transfusion independence rate observed with azacitidine in this lower-risk population, but resistant to erythropoietic stimulating agents, was lower than expected, with no observed benefit of added epoetin, (clinicaltrials.gov identifier: 01015352).
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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.