Hematology news.

EMA’s Pharmacovigilance Risk Assessment Committee has begun reviewing injectable iron-containing medicines. The review will assess low blood phosphate levels and related bone problems; the supplied meeting highlights report no findings or prescribing changes.

An observational biopsy study compared 10 patients with enterocolitis after ciltacabtagene autoleucel, seven CAR-T-treated controls without enterocolitis and 26 healthy volunteers. Findings linked enterocolitis to mucosal immune depletion and inflammatory remodeling; two affected patients improved after upadacitinib, providing preliminary support for further study.

A phase 1 trial enrolled 23 adults with relapsed or refractory CD22-positive acute lymphoblastic leukemia or lymphoblastic lymphoma. Venetoclax plus inotuzumab ozogamicin produced complete remission in 21 of 22 evaluable patients, but the small cohort and sinusoidal obstructive syndrome risk warrant caution.

A laboratory study using genetically engineered mice and patient-derived cells linked CALR-mutated myeloproliferative neoplasms to expansion of platelet-biased hematopoietic stem cells. PERK inhibition reduced proliferation of mouse CALRdel52 stem cells and impaired megakaryocytic differentiation of patient CALRdel52-like cells; clinical efficacy was not evaluated.

A translational study combining bone marrow analyses from treatment-naïve stage III breast cancer patients with mouse experiments implicates bone-derived DKK1 in persistent myeloid bias. Targeting DKK1 reduced tumor progression and myeloid output in mice; patient sample size and quantitative effect estimates were not provided in the abstract.

A review in Blood describes how hematologists and oncologists can apply existing expertise to acute radiation syndrome during mass casualty incidents. It emphasizes established clinical resources, rapid training and outpatient management of stable exposed individuals; the supplied abstract reports no patient outcomes.

A longitudinal study of 13 patients with VEXAS syndrome, combined with human monocytic cell-line experiments, identified genotype-dependent inflammatory programs. RNASE1 expression correlated with disease activity, while RIPK3 inhibition attenuated pathological features in cell models.

A mechanistic study in Blood identifies the iron chaperone PCBP2 as a regulator of macrophage iron recycling. Mice lacking macrophage PCBP2 accumulated splenic iron despite reduced bone marrow iron and features of iron-restricted erythropoiesis; sample sizes were not provided in the abstract.

An observational study of 40 patients with VEXAS syndrome found endothelial activation, altered von Willebrand factor profiles, and increased thrombin generation compared with age-matched controls. Twenty-one patients (52.5%) had experienced thrombosis; whether targeting these pathways improves outcomes remains unknown.

An American Heart Association scientific statement examines antithrombotic undertreatment and its consequences in patients with atrial fibrillation, venous thromboembolism, and peripheral artery disease. It highlights gaps in prescribing, dosing, adherence, and access, and calls for interprofessional antithrombotic stewardship.

A Journal of Clinical Oncology review examines treatment selection for relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma in the targeted-therapy era. It emphasizes reassessment of disease biology, prior treatment response, and patient factors when sequencing BTK inhibitors, venetoclax, and cellular therapies.

A prognostic model development and validation study incorporated longitudinal measurable residual disease data to predict outcomes after limited-duration CLL therapy. Validated using CLL14 and CLL13 trial data, CIRI2-CLL outperformed individual prognostic indices for progression-free survival; sample sizes were not provided in the abstract.