Acute Myelogenous Leukemia

P1, N=10, Active, not recruiting, Baylor College of Medicine | Trial completion date: Jul 2026 --> Jul 2027

P1, N=46, Recruiting, City of Hope Medical Center | Trial completion date: Feb 2030 --> May 2028 | Trial primary completion date: Feb 2030 --> May 2028

Ruxolitinib-based regimens, administered in 19 patients (40%) as EMH-directed therapy, yielded rapid palliation (dyspnea/ascites resolution), complemented by radiotherapy (11%) and splenectomy; HSCT achieved complete remission in one case...Atypical EMH signals advanced myelofibrosis with dismal prognosis, responsive to JAK inhibitors/locoregional therapies but highlighting sanctuary biology. Systematic surveillance and transplant referral are imperative; prospective registries are needed to refine risk models and test novel antifibrotics.

Pharmacologic inhibition of IGF2BP1-RNA interactions with the small-molecule BTYNB induced potent and selective cytotoxicity in patient-derived AMKL models. Together, these findings uncover a developmental co-dependency between SNHG29 and IGF2BP1 that defines a lineage-restricted oncogenic circuit and an actionable therapeutic vulnerability in AMKL.

P1, N=48, Recruiting, Sawa Ito, MD | Not yet recruiting --> Recruiting

P2, N=108, Active, not recruiting, City of Hope Medical Center | Trial completion date: May 2026 --> May 2027 | Trial primary completion date: May 2026 --> May 2027

It delves into its regulation of core cellular processes, its integration into oncogenic signaling networks, and its recently discovered functions in epigenetic and metabolic reprogramming. By consolidating this knowledge, we aim to highlight the multifaceted nature of DHX33 in cancer biology and firmly establish its potential as a viable and promising therapeutic target for future anticancer strategies.

Genetic analysis identified a previously unreported heterozygous splice-site variant in GATA2 (c.1017 + 1 G > A), confirmed in stored DNA from her deceased father. Despite supportive care and allogeneic hematopoietic stem cell transplantation from a matched unrelated donor, she developed severe graft-versus-host disease and died from transplant-related complications.This report identifies a previously unreported GATA2 splice-site variant with clinical and familial evidence supporting pathogenicity, contributing to the expanding mutational spectrum and enhancing understanding of genotype-phenotype correlations in GATA2 deficiency.

The histopathologic, immunohistochemical, and molecular findings as well as the current diagnostic criteria for BPDCN are reviewed. In addition, the differential diagnosis with acute myeloid leukemia with CD4/CD56/CD123 positivity and reactive plasmacytoid dendritic cell hyperplasia is discussed.

While A-1331852 was cytotoxic yet weakly immunogenic, its combination with OT-55 enhanced DAMP release, increased CD8⁺ effector functions, and, with PD-1/Tim-3 blockade, achieved local and distant tumor control with low toxicity. These findings identify OT-55 as an immunogenic adjuvant converting tolerogenic BH3 mimetic-driven apoptosis into ICD, providing a proof-of-concept immunogenic treatment for myelomonocytic AML.

P2, N=39, Recruiting, University of Maryland, Baltimore | Trial completion date: Feb 2027 --> Feb 2028 | Trial primary completion date: Jun 2026 --> Jun 2027