Evidence map›Paper›PMID 42120728›Full record

Trial reportNature medicine2026

CRISPR-Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial.

John F DiPersio, Guenther Koehne, Nirali N Shah, Léa Bernard, Hyung C Suh, Divya Koura, Roni Tamari, Muhammad Umair Mushtaq, Joseph Maakaron, Joseph Rimando and 18 more

Abstract readClinical Trial, Phase IIClinical Trial, Phase IMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Nature medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

28 authors.

John F DiPersioDepartment of Medicine, Division of Oncology, Washington University School of Medicine, St. Louis, MO, USA. jdipersi@wustl.edu.ORCID http://orcid.org/0000-0002-0429-3133
Guenther KoehneMiami Cancer Institute, Baptist Health South Florida, Miami, FL, USA.ORCID http://orcid.org/0009-0008-6281-4655
Nirali N ShahPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-8474-9080
Léa BernardDivision of Hematology, Oncology and Transplantation, Hôpital Maisonneuve-Rosemont/Université de Montréal, Montréal, Québec, Canada.
Hyung C SuhJohn Theurer Cancer Center, Hackensack University Medical Center, Hackensack, NJ, USA.
Divya KouraUC San Diego Moores Cancer Center, San Diego, CA, USA.
Roni TamariDepartment of Medicine, Adult Bone Marrow Transplant Services, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID http://orcid.org/0000-0002-2386-0850
Muhammad Umair MushtaqDivision of Hematologic Malignancies & Cellular Therapeutics, University of Kansas Cancer Center, Kansas City, KS, USA.
Joseph MaakaronDivision of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN, USA.
Joseph RimandoWinship Cancer Institute, Emory University, Atlanta, GA, USA.
Vanessa E KennedyStanford University, Department of Medicine, Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford, CA, USA.
Sagar S PatelUniversity of Utah, Division of Hematology & Hematologic Malignancies, Huntsman Cancer Institute, Salt Lake City, UT, USA.ORCID http://orcid.org/0000-0001-9949-3615
Chad HudsonHematologics, Inc., Seattle, WA, USA.
Michael R LokenHematologics, Inc., Seattle, WA, USA.
Christopher A SlapakVor Biopharma, Cambridge, MA, USA.
Deborah M LloydVor Biopharma, Cambridge, MA, USA.
Darren A StanizziVor Biopharma, Cambridge, MA, USA.
Melissa M Lee-SundlovVor Biopharma, Cambridge, MA, USA.
Sanjana ThosarVor Biopharma, Cambridge, MA, USA.
Guy MundelboimVor Biopharma, Cambridge, MA, USA.
Guangwu GuoVor Biopharma, Cambridge, MA, USA.
Huanying Gary GeVor Biopharma, Cambridge, MA, USA.
Bin E LiVor Biopharma, Cambridge, MA, USA.
Juliana Xavier-FerrucioVor Biopharma, Cambridge, MA, USA.
Sharon L HyzyVor Biopharma, Cambridge, MA, USA.
Michelle I LinVor Biopharma, Cambridge, MA, USA.ORCID http://orcid.org/0009-0005-9297-2855
Glen D RaffelVor Biopharma, Cambridge, MA, USA.
Brenda W CooperAdult Hematologic Malignancies & Stem Cell Transplant, Seidman Cancer Center, University Hospitals Cleveland Medical Center, Cleveland, OH, USA.

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
NCI NIH HHS P30 CA008748
6 · The paper itself

Abstract

Patients with high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) are likely to relapse despite allogenic hematopoietic cell transplantation (HCT). Post-HCT preventative maintenance can be limited by toxicity toward the normal donor cells. Tremtelectogene empogeditemcel (trem-cel) is a CRISPR-Cas9 gene-edited allogeneic HCT product lacking CD33, designed to shield the donor graft from cytotoxicity of subsequent CD33-targeted therapies such as gemtuzumab ozogamicin (GO). In this multicenter, phase 1/2a, open-label study, adult patients with AML/MDS with high relapse risk received trem-cel after myeloablative conditioning followed by GO maintenance (0.5-2.0 mg m

Indexed as

CRISPR-Cas SystemsGemtuzumabHematopoietic Stem Cell TransplantationLeukemia, Myeloid, AcuteSialic Acid Binding Ig-like Lectin 3AdultAgedFemaleGraft vs Host DiseaseHumansMaleMiddle AgedMyelodysplastic SyndromesTransplantation ConditioningTransplantation, HomologousCD33 protein, humanGemtuzumabSialic Acid Binding Ig-like Lectin 3

Identifiers

PMID42120728
PMCPMC13190247

What OpenQuestion holds

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Registered trials

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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.