Evidence map›Paper›PMID 40594997›Full record

ArticleNPJ precision oncology2025

Functional cure with single agent olutasidenib in relapsed IDH1/NPM1 co-mutated AML.

Justin Watts, Tiffany Nong, Katarina Micin, Deborah Soong, Ellen Madarang, Maurizio Affer, Shefali Mehra, Jessica Alvarez Lesmes, Jennifer Chapman, Yi Zhou and 5 more

Abstract read
In one paragraph

Article in NPJ precision oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. 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

15 authors.

Justin WattsUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA. jxw401@miami.edu.
Tiffany Nong *University of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Katarina Micin *University of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Deborah Soong *University of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Ellen MadarangUniversity of Miami Sylvester Comprehensive Cancer Center, Department of Pharmacy, Miami, FL, USA.
Maurizio AfferUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Shefali MehraUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Jessica Alvarez LesmesUniversity of Miami Miller School of Medicine, Department of Pathology, Miami, FL, USA.
Jennifer ChapmanUniversity of Miami Miller School of Medicine, Department of Pathology, Miami, FL, USA.
Yi ZhouUniversity of Miami Miller School of Medicine, Department of Pathology, Miami, FL, USA.
Amber ThomassenUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Terrence BradleyUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Tulasigeri TotigerUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA.
Ronan SwordsOregon Health & Science University, Knight Cancer Institute, Division of Hematology/Medical Oncology, Portland, OE, USA.
Justin TaylorUniversity of Miami Sylvester Comprehensive Cancer Center, Division of Hematology, Miami, FL, USA. jxt1091@miami.edu.

Funding

Tumor Biology Research ProgramP30CA240139 · NCI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI Stephen D. Nimer · 2019 to 2026
$24.1M
Defining the Biological and Mechanistic Implications of XPO1 Mutations in Hematologic MalignanciesK08CA230319 · NCI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI TAYLOR, JUSTIN · 2018 to 2023
$1.2M
Doris Duke Charitable Foundation CSDAEdward P. Evans Foundation DRGNCI NIH HHS K08 CA230319NCI NIH HHS K08CA230319, P30CA240139NCI NIH HHS P30 CA240139
6 · The paper itself

Abstract

Olutasidenib is a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1) that was recently approved by the US FDA for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring mutant IDH1. In the pivotal Phase II trial of olutasidenib, the median duration of complete response (CR) was 28.1 months. Here we report the first patient in the world to receive olutasidenib, for relapsed NPM1 and IDH1 co-mutated AML, who remains in continuous CR for over 7 years on olutasidenib monotherapy. We detail the clinical course as well as the pathologic and genomic evolution of the disease. Furthermore, using a novel single cell measurable residual disease assay and digital PCR and qPCR for the detection of IDH1 and NPM1 mutations, we found no evidence of residual detectable leukemia. To our knowledge, this is the first report of an AML patient functionally cured by IDH1 inhibitor monotherapy.

Identifiers

PMID40594997
PMCPMC12216377

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.