Evidence map›Paper›PMID 42681647›Full record

ArticleJournal of hematology & oncology2026

A phase 1 trial of CD123-directed chimeric antigen receptor T-cell therapy in adults with relapsed/refractory acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm.

Lihua E Budde, Marissa M Del Real, Joo Song, Tracey Stiller, Yan Wang, Ahmed Aribi, Karamjeet Sandhu, Ryotaro Nakamura, Emanuela C Marcucci, Jian J Wu and 11 more

Registry-linked trialAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of hematology & oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02159495 (Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm), which is not on this map. Not yet cited in PubMed.

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

NCT02159495 phase1active not recruitingnot on this map

Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

TypeinterventionalSponsorCity of Hope Medical CenterRan2015 to 2027Enrolled31ConditionsAdult Acute Myeloid Leukemia in Remission, Acute Biphenotypic Leukemia, Early Relapse of Acute Myeloid Leukemia, Late Relapse of Acute Myeloid LeukemiaArmscyclophosphamide, Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes, laboratory biomarker analysis, Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytes, Fludarabine Phosphate
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

21 authors.

Lihua E BuddeHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Marissa M Del RealHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Joo SongDepartment of Pathology, City of Hope, Duarte, CA, USA.
Tracey StillerDepartment of Computational and Quantitative Medicine/BRI, City of Hope, Duarte, CA, USA.
Yan WangDepartment of Computational and Quantitative Medicine/BRI, City of Hope, Duarte, CA, USA.
Ahmed AribiHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Karamjeet SandhuHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Ryotaro NakamuraHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Emanuela C MarcucciHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Jian J WuHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Kenneth NgHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Mary C ClarkHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Brent WoodDepartment of Laboratory Medicine, University of Washington, Seattle, WA, USA.
Jamie R WagnerHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Jinny PaulHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Christine E BrownHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Anthony SteinHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
Guido MarcucciHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA.
M Suzette BlanchardDepartment of Computational and Quantitative Medicine/BRI, City of Hope, Duarte, CA, USA.
Joycelynne PalmerDepartment of Computational and Quantitative Medicine/BRI, City of Hope, Duarte, CA, USA.
Stephen J FormanHematological Malignancies Research Institute, City of Hope, Duarte, CA, USA. sforman@coh.org.

Funding

Transgenic Mouse FacilityP30CA033572 · NCI · CITY OF HOPE/BECKMAN RESEARCH INSTITUTE · PI John Charles Williams · 1985 to 2026
$86.3M
PURPOSE OF THE NGVB CONTRACT IS TO CONTINUE SUPPORTING GENE THERAPY RESEARCH.75N92019D00018 · OD · INDIANA UNIVERSITY INDIANAPOLIS · 2019 to 2023
$1.7M
NCI NIH HHS P30 CA033572NHLBI NIH HHS 75N92019D00018
6 · The paper itself

Abstract

backgroundPatients with relapsed acute myeloid leukemia (AML), particularly following allogeneic stem cell transplant (alloHCT), have extremely limited therapeutic options. CD123 is an AML-associated antigen and represents an attractive target for immunotherapy. We report outcomes of a phase 1 trial evaluating CD123-targeting chimeric antigen receptor (CAR) T cells in patients with relapsed/refractory (r/r) AML or blastic plasmacytoid dendritic cell neoplasm (BPDCN).

methodsThis was a single center, open-label, phase 1 dose escalation study enrolling patients with either CD123-positive r/r AML (Arm 1) or BPDCN (Arm 2). Patients received autologous or donor-derived allogeneic CD123 CAR T cells following lymphodepletion. The co-primary objectives were to examine safety and anti-tumor activity using an activity-constrained for toxicity design and to determine the recommended phase 2 dose. Secondary objectives included assessments of progression-free and overall survival.

resultsWe enrolled 41 patients, of whom 21 patients (n = 19 on Arm 1 and n = 2 on Arm 2) received CD123 CAR T-cell infusion. The median age of treated AML patients was 45 years (range: 20-71); the two BPDCN patients were aged 24 and 75 years. Among AML patients, 17 (89%) had undergone prior alloHCT. AML patients received 50 × 10

conclusionsCD123-directed CAR T-cell therapy is feasible and demonstrates a favorable safety profile in patients with r/r AML and BPDCN, including those with prior alloHCT. Although the estimated overall complete remission rate was modest, these findings provide a foundation for further optimization of CD123 CAR T-cell design, patient selection, and combination strategies.

trial registrationThis trial was registered at ClinicalTrials.gov as NCT02159495.

Indexed as

Blastic Plasmacytoid Dendritic Cell NeoplasmImmunotherapy, AdoptiveInterleukin-3 Receptor alpha SubunitLeukemia, Myeloid, AcuteReceptors, Chimeric AntigenAdultAgedFemaleHumansMaleMiddle AgedRecurrenceYoung AdultIL3RA protein, humanInterleukin-3 Receptor alpha SubunitReceptors, Chimeric AntigenAcute myeloid leukemiaBPDCNCAR T cellsCD123

Identifiers

PMID42681647
PMCPMC13536854

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