The patient achieved complete remission following the standard 7 + 3 induction chemotherapy regimen for AML with gemtuzumab ozogamicin. This case illustrates the diagnostic challenges posed by concomitant class-defining alterations in hematologic neoplasms and underscores the importance of integrated genomic assessment.

16 days ago

Journal

PDGFRB (Platelet Derived Growth Factor Receptor Beta) • WT1 (WT1 Transcription Factor) • GOLGA4 (Golgin A4) • CDKN1A (Cyclin-dependent kinase inhibitor 1A)

CBFB-MYH11 fusion

Mylotarg (gemtuzumab ozogamicin)

22d

APL-like subset within NPM1-mutated AML: A distinct immunophenotype correlating with early vascular complications. (PubMed, Hemasphere)

We validated these clinical and molecular findings in an independent validation cohort of 302 NPM1 mut patients enrolled in the acute myeloid leukemia study group (AMLSG) 09-09 clinical trial, which included the administration of all-trans retinoic acid (ATRA) to all patients and a randomization for gemtuzumab ozogamicin. In this cohort, the APL-like immunophenotype was associated with events occurring within the first 15 days but did not influence mortality, likely due to protocol-driven patient selection. Our findings have important clinical implications that warrant the development of studies exploring disease-tailored clinical measures to mitigate the risk of early vascular events, as in current APL management.

22 days ago

Clinical • Journal

IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1)

NPM1 mutation

Mylotarg (gemtuzumab ozogamicin)

24d

MyeChild 01: International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia (clinicaltrials.gov)

P3, N=700, Active, not recruiting, University of Birmingham | Recruiting --> Active, not recruiting

24 days ago

Enrollment closed

cytarabine • cyclophosphamide • Mylotarg (gemtuzumab ozogamicin) • mitoxantrone • fludarabine IV • busulfan

28d

Targeting BCL-2 and PI3K signaling pathways enhances cytotoxicity of gemtuzumab ozogamicin against acute myeloid leukemia cells. (PubMed, Biochem Biophys Res Commun)

Such inhibition caused cell line-dependent changes in the expression of apoptotic regulators, including BCL-2, MCL-1 and p53 protein, suggesting that cellular context shapes responses to combined treatments. In conclusion, our findings identify BCL-2 and PI3K inhibition as a promising new approach to sensitize AML cells to GO therapy, and highlight the importance of these pathways in determining the cellular response to gemtuzumab ozogamicin.

28 days ago

Journal • IO biomarker

BCL2 (B-cell CLL/lymphoma 2) • MCL1 (Myeloid cell leukemia 1)

Venclexta (venetoclax) • Mylotarg (gemtuzumab ozogamicin)

2ms

CA235-0001: A Phase I Study of BMS-986497 alone or in combination with Azacitidine (Aza) and Azacitidine plus Venetoclax (AZA+VEN) in relapsed or refractory (R/R) AML or MDS (2024-519537-29-00)

P1, N=35, Recruiting, Bristol-Myers Squibb Services Unlimited Company | Not yet recruiting --> Recruiting

2 months ago

Enrollment open

Venclexta (venetoclax) • azacitidine

2ms

A Study To Learn About the Safety Medicine (Called Mylotarg) In People With Acute Myeloid Leukemia (clinicaltrials.gov)

P=N/A, N=165, Recruiting, Pfizer | Trial completion date: Apr 2027 --> Jul 2027 | Trial primary completion date: Apr 2027 --> Jul 2027

2 months ago

Trial completion date • Trial primary completion date

CD33 (CD33 Molecule)

CD33 positive

Mylotarg (gemtuzumab ozogamicin)

2ms

A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia (clinicaltrials.gov)

P2/3, N=216, Not yet recruiting, Sichuan Baili Pharmaceutical Co., Ltd. | N=80 --> 216 | Initiation date: Dec 2025 --> Mar 2026

2 months ago

Enrollment change • Trial initiation date

Venclexta (venetoclax) • cytarabine • azacitidine • daunorubicin • BL-M11D1

2ms

A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients (clinicaltrials.gov)

P1, N=32, Not yet recruiting, M.D. Anderson Cancer Center | Initiation date: Dec 2025 --> Dec 2027

2 months ago

Trial initiation date

KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)

cytarabine • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • Revuforj (revumenib)

3ms

MM1YA-A04: Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MyeloMATCH Treatment Trial) (clinicaltrials.gov)

P2, N=162, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Feb 2026 --> Jun 2026

3 months ago

Trial initiation date

ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RUNX1 (RUNX Family Transcription Factor 1) • RUNX1T1 (RUNX1 Partner Transcriptional Co-Repressor 1)

Venclexta (venetoclax) • cytarabine • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • Starasid (cytarabine ocfosfate)