Evidence map›Paper›PMID 21293051›Full record

Trial reportOncotarget2010

Phase 2 clinical trial of 5-azacitidine, valproic acid, and all-trans retinoic acid in patients with high-risk acute myeloid leukemia or myelodysplastic syndrome.

Emmanuel Raffoux, Audrey Cras, Christian Recher, Pierre-Yves Boëlle, Adrienne de Labarthe, Pascal Turlure, Jean-Pierre Marolleau, Oumedaly Reman, Claude Gardin, Maud Victor and 10 more

Registry-linked trialAbstract readClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in Oncotarget, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02608736 (Phase 0 Clinical Trial With Valproic Acid as a Chemopreventive Agent in Patients With Head and Neck Squamous Cell Carcinoma Previously Treated), which is not on this map. Cited by 60 papers.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

NCT02608736 early_phase1completednot on this mapstarted 2015, after this paper: background citation

Phase 0 Clinical Trial With Valproic Acid as a Chemopreventive Agent in Patients With Head and Neck Squamous Cell Carcinoma Previously Treated

TypeinterventionalSponsorBarretos Cancer HospitalRan2015 to 2017Enrolled42ConditionsHead and Neck Squamous Cell CarcinomaArmsValproic Acid, Placebo
3 · Its place in the literature

Who cites it

60 citing papers in PubMed.

  1. Decitabine plus all-Haematologica · 2026
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  19. Key actors in cancer therapy: epigenetic modifiers.Turkish journal of biology = Turk biyoloji dergisi · 2019
    Article
  20. Epigenetics in myelodysplastic syndromes.Seminars in cancer biology · 2018
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

20 authors.

Emmanuel RaffouxDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.
Audrey CrasUMR-S-940, Université Denis Diderot-Paris 7, IUH, Paris.
Christian RecherDépartement d'Hématologie, Hôpital Purpan, Toulouse.
Pierre-Yves BoëlleUMR-S-707, Université Pierre et Marie Curie - Paris 6, Hôpital Saint-Antoine, AP-HP, Paris.
Adrienne de LabartheDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.
Pascal TurlureDépartement d'Hématologie, Hôpital Dupuytren, Limoges.
Jean-Pierre MarolleauDépartement d'Hématologie, Hôpital Nord, Amiens.
Oumedaly RemanDépartement d'Hématologie, Centre Hospitalier Universitaire, Caen.
Claude GardinDépartement d'Hématologie, Hôpital Avicenne, AP-HP, Bobigny.
Maud VictorUMR-S-940, Université Denis Diderot-Paris 7, IUH, Paris.
Sébastien MauryDépartement d'Hématologie, Hôpital Henri Mondor, AP-HP, Créteil.
Philippe RousselotDépartement d'Hématologie, Hôpital Mignot, Versailles.
Jean-Valère MalfusonDépartement d'Hématologie, Hôpital Percy, Clamart.
Odile MaarekDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.
Marie-Thérèse DanielDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.
Pierre FenauxDépartement d'Hématologie, Hôpital Avicenne, AP-HP, Bobigny.
Laurent DegosDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.
Christine ChomienneUMR-S-940, Université Denis Diderot-Paris 7, IUH, Paris.
Sylvie ChevretDépartement de Biostatistiques et d'Informatique Médicale, Hôpital Saint-Louis, AP-HP, Paris, France.
Hervé DombretDépartement d'Hématologie, Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris (AP-HP), and Université Denis Diderot - Paris 7, EA 3518, Institut Universitaire d'Hématologie (IUH), Paris.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In this Phase 2 study, we evaluated the efficacy of combination of 5-azacitidine (AZA), valproic acid (VPA), and all-trans retinoic acid (ATRA) in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Treatment consisted of six cycles of AZA and VPA for 7 days, followed by ATRA for 21 days. Sixty-five patients were enrolled (median age, 72 years; 55 AML including 13 relapsed/refractory patients, 10 MDS; 30 unfavorable karyotypes). Best responses included 14 CR and 3 PR (26%), 75% of the responders and 36% of the non-responders achieving an erythroid response. Median overall survival (OS) was 12.4 months. Untreated patients had a longer OS than relapsed/refractory patients. In patients who fulfilled the 6 planned cycles, OS did not appear to depend on CR/PR achievement, suggesting that stable disease while on-treatment would be a surrogate for survival with this approach. During therapy, early platelet response and demethylation of the FZD9, ALOX12, HPN, and CALCA genes were associated with clinical response. Finally, there was no evidence for the restoration of an ATRA-induced differentiation during therapy. Epigenetic modulation deserves prospective comparisons to conventional care in patients with high-risk AML, at least in those presenting previously untreated disease and low blast count.

Indexed as

AgedAged, 80 and overAnticonvulsantsAntineoplastic Combined Chemotherapy ProtocolsAzacitidineDNA MethylationDNA, NeoplasmEpigenomicsFemaleHumansLeukemia, Myeloid, AcuteMaleMiddle AgedMyelodysplastic SyndromesPolymerase Chain ReactionSurvival RateAnticonvulsantsAzacitidineDNA, NeoplasmTretinoinValproic Acid

Identifiers

PMID21293051
PMCPMC4053545

What OpenQuestion holds

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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.