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    BIOMARKER:

    CD123 positive

    i
    Other names: IL3RA, Interleukin 3 receptor subunit alpha, CD123, Interleukin 3 Receptor Alpha, IL-3 Receptor Subunit Alpha, IL-3R Subunit Alpha, CD123 Antigen, IL-3R-Alpha, IL-3RA , IL3R, IL-3 Receptor Alpha SP2 Isoform
    Entrez ID:
    3563
    Related biomarkers:
    Expression
    Others
    5d
    IMGN632-0802: IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia (clinicaltrials.gov)
    P1/2, N=218, Active, not recruiting, AbbVie | Trial completion date: Dec 2024 --> Apr 2026 | Trial primary completion date: Dec 2024 --> Apr 2026
    Trial completion date • Trial primary completion date
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • decitabine • pivekimab sunirine (PVEK)
    7d
    CADENZA: Study of IMGN632 in Patients With Untreated BPDCN and Relapsed/Refractory BPDCN (clinicaltrials.gov)
    P1/2, N=179, Active, not recruiting, AbbVie | Trial completion date: Dec 2025 --> Dec 2026
    Trial completion date
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA expression
    |
    pivekimab sunirine (PVEK)
    3ms
    A clinical trial to assess the safety, distribution, effects on certain immune cells and anti-leukaemia effects of LAVA-1266 in patients with acute myeloid leukaemia or certain types of myelodysplastic syndrome (ACTRN12624001214527)
    P1, N=50, Not yet recruiting, LAVA Therapeutics N.V.
    New P1 trial
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    3ms
    First-in-Human Dose Escalation Study of AFM28 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=30, Recruiting, Affimed GmbH | Active, not recruiting --> Recruiting
    Enrollment open
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    AFM28
    4ms
    Flow cytometric immunophenotypic features of acute myeloid leukemia with mast cell differentiation. (PubMed, Am J Clin Pathol)
    The MCs in AML-MC cases are characterized by dim CD45, low side scatter, CD34 and CD123 positivity, and uniform and increased CD38 expression. Flow cytometry is an excellent tool for identifying AML-MC cases.
    Journal • IO biomarker
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • CD38 (CD38 Molecule) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL2RA (Interleukin 2 receptor, alpha) • CD34 (CD34 molecule) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • NCAM1 (Neural cell adhesion molecule 1) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • ISG20 (Interferon Stimulated Exonuclease Gene 20)
    |
    CD38 expression • CD123 positive • NCAM1 expression • CD34 positive
    8ms
    TAGALONG: Tagraxofusp and Azaxitadine With or Without Venetoclax in Newly Diagnosed Secondary AML After Hypomethylating Agents (clinicaltrials.gov)
    P2, N=53, Recruiting, Joshua Zeidner | Not yet recruiting --> Recruiting
    Enrollment open
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive
    |
    Venclexta (venetoclax) • azacitidine • Elzonris (tagraxofusp-erzs)
    8ms
    Bortezomib suppresses acute myelogenous leukaemia stem-like KG-1a cells via NF-κB inhibition and the induction of oxidative stress. (PubMed, J Cell Mol Med)
    BTZ also increased mitochondrial superoxide levels in KG-1a cells, and BTZ-induced apoptosis was partially prevented by pretreatment with the antioxidant N-acetylcysteine, indicating that BTZ induces oxidative stress-mediated apoptosis in KG-1a cells. At a dosage of 0.1 mg/kg every other day for 2 weeks, BTZ significantly reduced the percentage of hCD45-positive cells in the bone marrow and peripheral blood of NSG mice engrafted with KG-1a cells with tolerable toxicity. Taken together, these data indicate that the anti-LSC potential of BTZ appears to be an important strategy for AML treatment.
    Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • CD34 (CD34 molecule) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • CASP3 (Caspase 3) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD34 positive • IL3RA positive
    |
    bortezomib
    8ms
    IMGN632-0802: IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia (clinicaltrials.gov)
    P1/2, N=218, Active, not recruiting, ImmunoGen, Inc. | Recruiting --> Active, not recruiting | Phase classification: P1b/2 --> P1/2
    Enrollment closed • Phase classification
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • decitabine • pivekimab sunirine (PVEK)
    8ms
    Flotetuzumab as a salvage immunotherapy in advanced CD123-positive hematological malignancies, a phase 1 pilot study. (PubMed, Leuk Lymphoma)
    Responses only occurred in Cohort B, with a partial response in one patient with Hodgkin's lymphoma and morphological complete remission in the bone marrow in one patient with blastic plasmacytoid dendritic cell neoplasm. In conclusion, flotetuzumab had a manageable safety profile in advanced CD123-positive hematological malignancies.
    P1 data • Journal • IO biomarker • Metastases
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA expression • IL3RA positive
    |
    flotetuzumab (MGD006)
    8ms
    Tagraxofusp, a first-in-class CD123-targeted agent: Five-year postapproval comprehensive review of the literature. (PubMed, Cancer)
    The successful targeting of CD123 in BPDCN has also encouraged research into a variety of other CD123-positive hematological neoplasms, including acute myeloid leukemia (AML), and informed the development of other novel agents targeting CD123. This review examines the clinical data leading to the development and approval of tagraxofusp in BPDCN, how it is being used in combination to improve outcomes in BPDCN and AML, and its developing role in other hematological malignancies.
    Review • Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Elzonris (tagraxofusp-erzs)
    10ms
    Tagraxofusp to Eradicate Measurable Residual Disease in Patients With Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=0, Withdrawn, Jonsson Comprehensive Cancer Center | N=29 --> 0 | Not yet recruiting --> Withdrawn
    Enrollment change • Trial withdrawal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA expression
    |
    azacitidine • Elzonris (tagraxofusp-erzs)
    10ms
    Pivekimab sunirine (IMGN632), a novel CD123-targeting antibody-drug conjugate, in relapsed or refractory acute myeloid leukaemia: a phase 1/2 study. (PubMed, Lancet Oncol)
    Pivekimab sunirine showed single-agent activity across multiple doses, with a recommended phase 2 dose of 0·045 mg/kg once every 3 weeks. These findings led to a phase 1b/2 study of pivekimab sunirine plus azacitidine and venetoclax in patients with CD123-positive acute myeloid leukaemia.
    P1/2 data • Clinical Trial,Phase II • Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA expression • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • pivekimab sunirine (PVEK)
    10ms
    Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm (clinicaltrials.gov)
    P1, N=31, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024
    Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive
    |
    cyclophosphamide • fludarabine IV • CD123R(EQ)28zeta/EGFRt+ T cells
    1year
    TAGALONG: Study of Tagraxofusp in Newly Diagnosed Secondary AML After Previous Exposure to Hypomethylating Agents (clinicaltrials.gov)
    P2, N=53, Not yet recruiting, Joshua Zeidner
    Trial completion date
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive
    |
    azacitidine • Elzonris (tagraxofusp-erzs)
    1year
    Phase 1b trial of tagraxofusp in combination with azacitidine with or without venetoclax in acute myeloid leukemia. (PubMed, Blood Adv)
    Median overall survival and progression-free survival were 14 months (95% CI, 9.5-NA) and 8.5 months (95% CI, 5.1-NA), respectively. In summary, TAG-AZA-VEN shows encouraging safety and activity in high-risk AML, including TP53-mutated disease, supporting further clinical development of TAG combinations.
    P1 data • Journal • Combination therapy • IO biomarker
    |
    TP53 (Tumor protein P53) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    TP53 mutation • CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • Elzonris (tagraxofusp-erzs)
    1year
    Tagraxofusp Shows Promising Anti-Tumoral Efficacy in Preclinical in Vitro Models of Myelofibrosis, Both As a Single Agent and in Combination with Janus Kinase Inhibitors (ASH 2023)
    The role of the JAK/Signal transducer and activator of transcriptions (STAT) pathway in MF has led to the recent approval of three different JAK2 inhibitors (ruxolitinib, pacritinib, and fedratinib) for MF treatment. Our studies showed high sensitivity of MF cell lines to tagraxofusp as a single agent, which is expected given phase 2 results demonstrated clinical efficacy of single-agent tagraxofusp in R/R MF (Yacoub et al. ASH 2021). In addition, synergism was observed in combination with JAK inhibitors.
    Preclinical • Combination therapy
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • JAK2 V617F • CD123 expression • IL3RA expression • IL3RA positive
    |
    Jakafi (ruxolitinib) • Elzonris (tagraxofusp-erzs) • Vonjo (pacritinib) • Inrebic (fedratinib)
    1year
    First Disclosure of AZD9829, a TOP1i-ADC Targeting CD123: Promising Preclinical Activity in AML Models with Minimal Effect on Healthy Progenitors (ASH 2023)
    Furthermore, AZD9829 demonstrated durable blast reduction at day 28 after the first dose with leukemic blast reduction in blood (7/13 models) and in bone marrow (5/13 models). Safety studies in cynomolgus monkey support the clinical development of AZD9829, a promising therapeutic candidate for the treatment of AML across the spectrum of CD123-expression and genetic mutations.
    Preclinical
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • CD33 (CD33 Molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA expression • IL3RA positive
    1year
    Diagnostic Challenges in the Leukemia Phase of Blastic Plasmacytoid Dendritic Cell Neoplasm without Skin Involvement: A Clinical and Pathological Study (ASH 2023)
    Only one cycle of decitabine + CAG was given and 5 months later, the disease progressed to the leukemia phase...The latter case had ASXL1 and TET2 mutations detected by NGS analysis, achieved complete remission after receiving venetoclax + azacitidine for one cycle, followed by intermittent venoclax +demethylation agent or low-dose chemotherapy maintenance treatment and live for more than two years now... BPDCN without skin lesions is clinically rare, and its diagnosis is challenging. Comprehensive immunophenotyping and cautious interpretation of immunohistochemistry results such as dim lysozyme expression are crucial. Venetoclax-containing regimens have shown promising therapeutic effects.
    Clinical
    |
    TP53 (Tumor protein P53) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • CD20 (Membrane Spanning 4-Domains A1) • CD8 (cluster of differentiation 8) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CD33 (CD33 Molecule) • CD34 (CD34 molecule) • CD36 (thrombospondin receptor) • NCAM1 (Neural cell adhesion molecule 1) • CD5 (CD5 Molecule) • CD14 (CD14 Molecule) • ITGAM (Integrin, alpha M) • MME (Membrane Metalloendopeptidase) • CD7 (CD7 Molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • NRP1 (Neuropilin 1) • SPN (Sialophorin) • TCL1A (TCL1 Family AKT Coactivator A) • ANPEP (Alanyl Aminopeptidase, Membrane) • CLEC4C (C-Type Lectin Domain Family 4 Member C) • MPO (Myeloperoxidase)
    |
    TP53 mutation • ASXL1 mutation • TET2 mutation • CD123 positive • NCAM1 expression • CD123 expression • CD4 expression • IDH2 mutation + TP53 mutation • IL3RA positive • NCAM1 positive
    |
    Venclexta (venetoclax) • azacitidine • decitabine
    1year
    First-in-Human Dose Escalation Study of AFM28 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=50, Recruiting, Affimed GmbH
    Trial completion date • Trial primary completion date
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    AFM28
    1year
    IRF8 in Conjunction With CD123 and CD20 to Distinguish Lupus Erythematosus Panniculitis From Subcutaneous Panniculitis-like T-Cell Lymphoma. (PubMed, Am J Surg Pathol)
    However, a panel combining IRF8, CD123, and CD20, with at least 1 positive marker was more accurate than any individual marker by receiver operating characteristic curve analysis. Our study provides a rationale for potentially including IRF8 as part of an immunohistochemical panel composed of other currently available markers used to differentiate LEP from SPTCL.
    Journal
    |
    CD20 (Membrane Spanning 4-Domains A1) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IRF8 (Interferon Regulatory Factor 8) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA positive • IRF8 expression
    1year
    Venetoclax Synergizes with IMGN632, a Novel CD123-Targeting Antibody Conjugated to a DNA Alkylating Payload, By Suppressing DNA Damage Response and Potentiating Apoptosis in Acute Myeloid Leukemia in Vitro Models (ASH 2023)
    Importantly, we previously showed high synergy of IMGN632 combination with BCL-2 inhibitor venetoclax (VEN) and DNA hypomethylating azacytidine (AZA) in AML cell lines and xenograft models (ASH 2020, #617). Together, these results suggest that VEN, apart from its canonical inhibitory effect on anti-apoptotic BCL-2, exerts previously unrecognized ability to suppress DDR program in AML and augments activity of DNA damaging IMGN632. Failure of cells to sustain DDR in the presence of VEN constitutes a key aspect of high efficacy of IMGN/VEN/AZA combination in AML.
    Preclinical • PARP Biomarker • IO biomarker
    |
    FLT3 (Fms-related tyrosine kinase 3) • MCL1 (Myeloid cell leukemia 1) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CHEK1 (Checkpoint kinase 1) • CASP3 (Caspase 3) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    FLT3-ITD mutation • FLT3 mutation • CD123 positive • MCL1 expression • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • pivekimab sunirine (PVEK)
    1year
    First-in-Human Study of the CD123 NK Cell Engager SAR443579 in Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia or High Risk-Myelodysplasia: Updated Safety, Efficacy, Pharmacokinetics and Pharmacodynamics (ASH 2023)
    Pts had received a median of 2.0 (1.0 –10.0) prior lines of treatment with 13 pts (30.2%) reporting prior hematopoietic stem cell transplantation and 36 pts (83.7%) with prior exposure to venetoclax. SAR'579 was well tolerated up to doses of 6000 µg/kg QW with observed clinical benefit in pts with R/R AML. The results are consistent with the predicted favorable safety profile.
    Clinical • P1 data • PK/PD data
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • SAR443579
    1year
    Pivekimab Sunirine (PVEK, IMGN632), a CD123-Targeting Antibody-Drug Conjugate, in Combination with Azacitidine and Venetoclax in Patients with Newly Diagnosed Acute Myeloid Leukemia (ASH 2023)
    Encouraging CCRMRD- rates were observed across cytogenetic/molecular subsets, and the majority of responding pts achieved early and deep remissions, which may translate to improved clinical outcomes. The regimen was well tolerated with no new safety signals, and the addition of PVEK to the AZA-VEN backbone did not appear to meaningfully prolong count recovery.
    Clinical • Combination therapy
    |
    FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    TP53 wild-type • CD123 positive • CD123 expression • IL3RA expression • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • pivekimab sunirine (PVEK)
    1year
    Potent in Vitro and In Vivo Efficacy of BYON4413, a Duba-Based Antibody-Drug Conjugate Targeting CD123 in Acute Myeloid Leukemia (ASH 2023)
    In sum, BYON4413 shows great potential to be an effective targeted therapy against AML, MDS, and other CD123+ hematological malignancies such as blastic plasmacytoid dendritic cell neoplasm (BPDCN). Readied with these promising pre-clinical results, we have designed a first-in-human dose-escalation and expansion trial enrolling AML and high-risk MDS patients scheduled to begin in Q12024.
    Preclinical
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • CD34 (CD34 molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA positive
    |
    BYON4413
    1year
    Functional Characterization and Optimization of Switchable Allogeneic Chimeric Antigen Receptor T Cells for Targeting CD19 and CD20 in B Cell Malignancies (ASH 2023)
    A summary of functional characterization data will be presented at the meeting with a focus on the lack of predictability of certain in vitro models and underlying hypotheses. Insights from these studies are now flowing into preclinical development of a novel switchable allogeneic CAR-T cell product candidate targeting CD19 and CD20 in B cell malignancies engineered to fully overcome graft-versus-host disease as well as graft rejection by host T and NK cells.
    CAR T-Cell Therapy
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    AVC-201
    1year
    Dual-Targeting CD33/CD123 NANOBODY® T Cell Engager Targeting Leukemia Blasts and Stem/Progenitor Cells in Relapsed AML (ASH 2023)
    Immunophenotypic profiling confirmed the effectiveness of CD33/CD123-TCE in eliminating various blast- and LSC-subsets, activating CD4+ and CD8+ T cells or expanding central/effector memory CD4+ and CD8+ T cells. These dual-targeting immune engagers may constitute an effective treatment option for patients who are older/unfit for or relapsed from/refractory to current regimens.
    PD(L)-1 Biomarker • IO biomarker
    |
    PD-L1 (Programmed death ligand 1) • FLT3 (Fms-related tyrosine kinase 3) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • CD8 (cluster of differentiation 8) • PD-1 (Programmed cell death 1) • CD38 (CD38 Molecule) • CD123 (Interleukin 3 Receptor Subunit Alpha) • HAVCR2 (Hepatitis A Virus Cellular Receptor 2) • CD33 (CD33 Molecule) • CD34 (CD34 molecule) • CD69 (CD69 Molecule) • CCR7 (Chemokine (C-C motif) receptor 7) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD8 expression • HAVCR2 expression • CD123 expression
    1year
    Frontline Hyper-CVAD Plus Venetoclax for Pediatric Blastic Plasmacytoid Dendritic Cell Neoplasm. (PubMed, J Pediatr Hematol Oncol)
    This patient achieved complete remission after receiving modified hyper-CVAD (hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone with venetoclax and intrathecal chemotherapy. He remains disease-free 200 days after receiving a stem cell transplant. This represents the first known published pediatric case using a modified hyper-CVAD plus venetoclax regimen for treating a pediatric BPDCN patient in the frontline setting.
    Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • CD4 (CD4 Molecule) • NCAM1 (Neural cell adhesion molecule 1) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • TCF4 (Transcription Factor 4)
    |
    CD123 positive
    |
    Venclexta (venetoclax) • doxorubicin hydrochloride • cyclophosphamide • vincristine
    1year
    Tagraxofusp in myeloid malignancies. (PubMed, Hematol Oncol)
    The triplet tagraxofusp-azacytidine-venetoclax appears to be of particular interest among these combinations. Nowadays, several ongoing trials are exploring the use of tagraxofusp in different myeloid neoplasms. This review aims to summarize the actual role of tagraxofusp in BPDCN and other CD123-positive myeloid malignancies.
    Review • Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • Elzonris (tagraxofusp-erzs)
    1year
    Hairy Cell Leukemia: Hematological and Immunophenotypic Profile of 13 Patients. (PubMed, Cureus)
    The diagnosis of HCL requires a multipronged approach. The use of clinical features, morphology, and immunophenotyping combined with ancillary techniques provides higher diagnostic accuracy and enables its distinction from other B-cell lymphoproliferative disorders (BCLPDs), leading to better patient management and treatment.
    Journal
    |
    CD20 (Membrane Spanning 4-Domains A1) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CD22 (CD22 Molecule) • IL2RA (Interleukin 2 receptor, alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • ITGAE (Integrin Subunit Alpha E) • ITGAX (Integrin Subunit Alpha X) • ISG20 (Interferon Stimulated Exonuclease Gene 20)
    |
    CD123 positive • CD22 expression • IL3RA positive
    1year
    IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia (clinicaltrials.gov)
    P1b/2, N=292, Recruiting, ImmunoGen, Inc. | Trial completion date: Jun 2024 --> Dec 2024 | Trial primary completion date: Jun 2024 --> Dec 2024
    Trial completion date • Trial primary completion date
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • decitabine • pivekimab sunirine (PVEK)
    1year
    Primary Myeloid Sarcoma of the lung vs BPDCN : a diagnostically challenging case (DGHO 2023)
    She was treated with two induction cycles cytarabine and daunorubicine („7+3“ and „5+2“) achieving partial remission with vital residual pulmonary disease. Classic induction treatment did not result in remission of MS, confirming the poor prognosis and resulting in a salvage treatment before planned allogeneic stem cell transplantation. Further studies are needed to define the optimal therapeutic strategy in this entity.
    Clinical
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CD163 (CD163 Molecule) • CD33 (CD33 Molecule) • CD34 (CD34 molecule) • NCAM1 (Neural cell adhesion molecule 1) • CD14 (CD14 Molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • NRP1 (Neuropilin 1) • CLEC4C (C-Type Lectin Domain Family 4 Member C)
    |
    CD123 positive
    |
    cytarabine • daunorubicin
    over1year
    Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm (clinicaltrials.gov)
    P1, N=31, Active, not recruiting, City of Hope Medical Center | Trial completion date: Jun 2023 --> Dec 2023 | Trial primary completion date: Jun 2023 --> Dec 2023
    Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive
    |
    cyclophosphamide • fludarabine IV • CD123R(EQ)28zeta/EGFRt+ T cells
    over1year
    A Study of the Drug IMGN632 in Children With Leukemia That Has Come Back After Treatment or is Difficult to Treat (clinicaltrials.gov)
    P1/2, N=0, Withdrawn, Children's Oncology Group | N=38 --> 0 | Not yet recruiting --> Withdrawn
    Enrollment change • Trial withdrawal
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    methotrexate • Vyxeos (cytarabine/daunorubicin liposomal formulation) • fludarabine IV • pivekimab sunirine (PVEK) • Starasid (cytarabine ocfosfate)
    over1year
    Safety and Efficacy of CD123-targeted CAR-NK for Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm (clinicaltrials.gov)
    P1/2, N=36, Recruiting, Chongqing Precision Biotech Co., Ltd
    New P1/2 trial
    |
    IL6 (Interleukin 6) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA positive
    |
    cyclophosphamide • CD123 targeted CAR-NK
    over1year
    CADENZA: Study of IMGN632 in Patients With Untreated BPDCN and Relapsed/Refractory BPDCN (clinicaltrials.gov)
    P1/2, N=179, Active, not recruiting, ImmunoGen, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • CD123 expression • IL3RA expression
    |
    pivekimab sunirine (PVEK)
    over1year
    Comparison And Development Of Immunohistochemical Diagnostic Criteria For Blastic Plasmacytoid Dendritic Cell Neoplasm. (PubMed, Mod Pathol)
    We also revealed that CD123-positive AML/MS patients had a significantly poorer outcome than BPDCN and that 12% (24/205) of the cases were non-BPDCN even if all three conventional markers were positive, thus clarifying the risk of diagnosing BPDCN without more specific markers. In addition, histopathological features, such as the reticular pattern, which is not seen in BPDCN and suggests AML/MS, were also identified.
    Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • CD4 (CD4 Molecule) • NCAM1 (Neural cell adhesion molecule 1) • TCF4 (Transcription Factor 4)
    |
    CD123 positive
    over1year
    IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia (clinicaltrials.gov)
    P1b/2, N=242, Recruiting, ImmunoGen, Inc. | Trial completion date: Jun 2022 --> Jun 2024 | Trial primary completion date: Jun 2022 --> Jun 2024
    Trial completion date • Trial primary completion date
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive • IL3RA positive
    |
    Venclexta (venetoclax) • azacitidine • decitabine • pivekimab sunirine (PVEK)
    over1year
    Dose-escalating Trial With UniCAR02-T Cells and CD123 Target Module (TM123) in Patients With Hematologic and Lymphatic Malignancies (clinicaltrials.gov)
    P1, N=90, Recruiting, AvenCell Europe GmbH | N=45 --> 90 | Trial completion date: Jun 2023 --> Sep 2025 | Trial primary completion date: Dec 2022 --> May 2025
    Enrollment change • Trial completion date • Trial primary completion date • Combination therapy
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    CD123 positive
    |
    cyclophosphamide • fludarabine IV • AVC-101
    over1year
    ENGAGING INNATE IMMUNITY: A PHASE 1 DOSE ESCALATION STUDY TO ASSESS SAFETY AND TOLERABILITY OF AFM28 MONOTHERAPY IN PATIENTS WITH RELAPSED/REFRACTORY CD123-POSITIVE ACUTE MYELOID LEUKEMIA (AML) (EHA 2023)
    N/A ADCC, Acute myeloid leukemia, NK cell, Immune therapy
    Clinical • P1 data
    |
    FLT3 (Fms-related tyrosine kinase 3) • CD123 (Interleukin 3 Receptor Subunit Alpha) • FCGR3A (Fc Fragment Of IgG Receptor IIIa)
    |
    CD123 positive • CD123 expression
    |
    AFM28