ArticleBlood cancer journal2014
Limited clinical efficacy of azacitidine in transfusion-dependent, growth factor-resistant, low- and Int-1-risk MDS: Results from the nordic NMDSG08A phase II trial.
Article in Blood cancer journal, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
29 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Pooled it
- Azacitidine in Lower-Risk Myelodysplastic Syndromes: A Meta-Analysis of Data from Prospective Studies.The oncologist · 2018Pooled it
- The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort.Blood advances · 2018Trial
- A randomized phase II trial of azacitidine +/- epoetin-β in lower-risk myelodysplastic syndromes resistant to erythropoietic stimulating agents.Haematologica · 2016Trial
- Trial
- FDA approval of imetelstat: a new era in the treatment of lower-risk myelodysplastic syndrome.Annals of medicine and surgery (2012) · 2025Review
- Review
- Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome.Current treatment options in oncology · 2024Review
- Role of reactive oxygen species in myelodysplastic syndromes.Cellular & molecular biology letters · 2024Review
- Myeloid leukemoid reaction after initial azacitidine therapy for chronic myelomonocytic leukemia.International journal of hematology · 2022Article
- 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
- 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
- No clear survival benefit of azacitidine for lower-risk myelodysplastic syndromes: A retrospective study of Nagasaki.Cancer science · 2020Observational
- Myelodysplastic syndromes: moving towards personalized management.Haematologica · 2020Review
- 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
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This prospective phase II study evaluated the efficacy of azacitidine (Aza)+erythropoietin (Epo) in transfusion-dependent patients with lower-risk myelodysplastic syndrome (MDS). Patients ineligible for or refractory to full-dose Epo+granulocyte colony stimulation factors for >8 weeks and a transfusion need of 4 units over 8 weeks were included. Aza 75 mg m(-2) d(-1), 5/28 days, was given for six cycles; non-responding patients received another three cycles combined with Epo 60 000 units per week. Primary end point was transfusion independence (TI). All patients underwent targeted mutational screen for 42 candidate genes. Thirty enrolled patients received one cycle of Aza. Ten patients discontinued the study early, 7 due to adverse events including 2 deaths. Thirty-eight serious adverse events were reported, the most common being infection. Five patients achieved TI after six cycles and one after Aza+Epo, giving a total response rate of 20%. Mutational screening revealed a high frequency of recurrent mutations. Although no single mutation predicted for response, SF3A1 (n=3) and DNMT3A (n=4) were only observed in non-responders. We conclude that Aza can induce TI in severely anemic MDS patients, but efficacy is limited, toxicity substantial and most responses of short duration. This treatment cannot be generally recommended in lower-risk MDS. Mutational screening revealed a high frequency of mutations.
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.