Evidence map›Paper›PMID 39701584›Full record

Trial reportBritish journal of haematology2025

Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukaemia arising from a prior myeloproliferative neoplasm.

Stéphane De Botton, Christian Récher, Jorge Cortes, Antonio Curti, Pierre Fenaux, Pierre Peterlin, Arnaud Pigneux, Karen Yee, Andrew Wei, Alice Mims and 4 more

Abstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in British journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Review
  4. Article
  5. Review
  6. Article
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

14 authors.

Stéphane De BottonHematologie Clinique, Institut Gustave Roussy, Villejuif, France.ORCID https://orcid.org/0000-0002-8126-4942
Christian RécherService d'hématologie, CHU de Toulouse, Institut Universitaire du Cancer Toulouse - Oncopole, Toulouse, France.
Jorge CortesGeorgia Cancer Center, Augusta University, Augusta, Georgia, USA.
Antonio CurtiIRCCS Azienda Ospedaliero-Universitaria di Bologna, Institute of Hematology Seràgnoli, Bologna, Italy.ORCID https://orcid.org/0000-0002-7752-6049
Pierre FenauxDépartement (DMU) d'hematologie et immunologie, APHP Nord, Service d'hématologie séniors, Hôpital St Louis/Université de Paris, Paris, France.ORCID https://orcid.org/0000-0002-0468-3553
Pierre PeterlinNantes University Hospital, Nantes, France.ORCID https://orcid.org/0000-0001-5463-6686
Arnaud PigneuxBordeaux Haut-Leveque University Hospital, Pessac, France.
Karen YeePrincess Margaret Cancer Centre, University of Toronto, Toronto, Ontario, Canada.
Andrew WeiPeter MacCallum Cancer Centre, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
Alice MimsThe Ohio State University Comprehensive Cancer Center, Columbus, Ohio, USA.
Gary SchillerDavid Geffen School of Medicine at UCLA, Los Angeles, California, USA.
Mwe Mwe ChaoRigel Pharmaceuticals, Inc., South San Francisco, California, USA.
Hua TianRigel Pharmaceuticals, Inc., South San Francisco, California, USA.
Justin M WattsDivision of Hematology, Department of Medicine, University of Miami Sylvester Comprehensive Cancer Center, Miami, Florida, USA.

Funding

Forma TherapeuticsRigel Pharmaceuticals, Inc.
6 · The paper itself

Abstract

Acute myeloid leukaemia (AML) arising from a myeloproliferative neoplasm (MPN) is more aggressive and less responsive to therapies compared to de novo AML. Olutasidenib, an oral small-molecule inhibitor of mutated IDH1 (mIDH1), showed encouraging and durable responses in a phase 1/2 study of adults with post-MPN mIDH1 AML. Patients received olutasidenib 150 mg BID monotherapy or in combination with azacitidine. Primary end-points: safety and best response defined as complete remission (CR), CR with partial haematological recovery or morphological leukaemia-free state (MLFS). Analysis included 15 patients with post-MPN mIDH1 AML; 10 had relapsed or refractory AML and five had newly diagnosed AML. Six were treated with olutasidenib monotherapy and nine in combination with azacitidine. Treatment emergent adverse events occurred in 15 patients, three of whom discontinued therapy. CR: 40% (n = 6/15); median duration of response: 15.6 months (range: 1.7-44.3); CR with incomplete haematological recovery: 13% (n = 2/15); MLFS: 7% (n = 1/15); composite complete remission (CRc): 53% (n = 8/15); and overall response rate (ORR): 60% (9/18). Median duration of CRc and ORR: 13.15 (range: 2.4-48.7) and 14.3 months (range: 2.4-48.7), respectively, and median overall survival: 13.8 months (95% confidence interval: 3.70-23.7). Olutasidenib demonstrated encouraging response rates with a manageable safety profile for patients with post-MPN mIDH1 AML.

Indexed as

Antineoplastic AgentsIsocitrate DehydrogenaseLeukemia, Myeloid, AcuteMutationMyeloproliferative DisordersNeoplasms, Second PrimaryPyridinesAdultAgedAged, 80 and overAntineoplastic Combined Chemotherapy ProtocolsAzacitidineFemaleHumansMaleMiddle AgedAntineoplastic AgentsAzacitidineIDH1 protein, humanIsocitrate DehydrogenasePyridinesacute myeloid leukaemiablast‐phase myeloproliferative neoplasmIDH1 mutationmyeloproliferative neoplasms

Identifiers

PMID39701584
PMCPMC11985372

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

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.