Common Genetic Variants in Rare Disorders: Hematology and Beyond

Current Issues in Molecular Biology, Journal Year: 2025, Volume and Issue: 47(1), P. 23 - 23

Published: Jan. 1, 2025

Emerging evidence suggests that common genetic variants play a significant role in various rare but life-threatening hematological and non-hematological conditions [...]

Language: Английский

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Invasive Fungal Disease After Chimeric Antigen Receptor-T Immunotherapy in Adult and Pediatric Patients

Pathogens, Journal Year: 2025, Volume and Issue: 14(2), P. 170 - 170

Published: Feb. 8, 2025

Invasive fungal diseases (IFDs) have been documented among the causes of post-chimeric antigen receptor-T (CAR-T) cell immunotherapy complications, with incidence IFDs in CAR-T therapy recipients being measured between 0% and 10%, globally. are notorious for their potentially life-threatening nature challenging diagnosis treatment. In this review, we searched recent literature aiming to examine risk factors epidemiology post-CAR-T infusion. Moreover, role antifungal prophylaxis is investigated. especially vulnerable due several that contribute patient’s immunosuppression. Those include underlying hematological malignancies, lymphodepleting chemotherapy administered before treatment, existing leukopenia hypogammaglobinemia, use high-dose corticosteroids interleukin-6 blockers as countermeasures immune effector cell-associated neurotoxicity syndrome cytokine release syndrome, respectively. mostly occur within first 60 days following infusion T cells, but cases even a year after described. Aspergillus spp., Candida Pneumocystis jirovecii main cause these infections therapy. More real-world data regarding population essential.

Language: Английский

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Translating biomarker insights into practice: a path forward in TA-TMA management

Frontiers in Medicine, Journal Year: 2025, Volume and Issue: 12

Published: May 8, 2025

Recent advances in the management of transplant-associated thrombotic microangiopathy (TA-TMA) include harmonization diagnostic criteria and identification high-risk disease features. Individual hematologic complement biomarkers show moderate specificity when used alone detection TA-TMA hematopoietic stem cell transplant (HSCT) recipients, but endothelial injury due to microangiopathic process can be enhanced using longitudinal monitoring clinical An increase sC5b-9 level reflects terminal activation, a hallmark pathogenesis that guides therapeutic interventions. In addition, distinguishing physiologic from pathologic activation is essential for timely diagnosis selection targeted Eculizumab therapy, biomarker-guided C5 blocker, significantly improves outcomes severe TA-TMA; however, there lack knowledge on how select second-line inhibitors or combination therapies cases with suboptimal response eculizumab. This article proposes practical approaches increasing attributability by integrating clinically available supportive tests provides insights into potential currently novel inhibitors. These findings help ensure diagnosis, prevent irreversible organ injury, improve HSCT recipients TA-TMA.

Language: Английский

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Soluble Urokinase-Type Plasminogen Activator Receptor (suPAR), Growth Differentiation Factor-15 (GDF-15), and Soluble C5b-9 (sC5b-9) Levels Are Significantly Associated with Endothelial Injury Indices in CAR-T Cell Recipients

International Journal of Molecular Sciences, Journal Year: 2024, Volume and Issue: 25(20), P. 11028 - 11028

Published: Oct. 14, 2024

Endothelial injury indices, such as Activation and Stress Index (EASIX), modified EASIX (m-EASIX), simplified (s-EASIX) scores, have been previously associated with chimeric antigen receptor-T (CAR-T) cell immunotherapy complications. Soluble urokinase-type plasminogen activator receptor (suPAR), growth differentiation factor-15 (GDF-15), soluble C5b-9 (sC5b-9) described markers of endothelial post-hematopoietic stem transplantation. In the current study, we examined whether suPAR, GDF-15, sC5b-9 levels were indices in adult CAR-T recipients. The these measured patients before infusion healthy individuals immunoenzymatic methods. We studied 45 recipients 20 control group. SuPAR, significantly higher patients' group compared to (p < 0.001, all comparisons). SuPAR at baseline m-EASIX scores calculated same time point = 0.020), while suPAR GDF-15 concentrations correlated day 14 post-infusion 0.001 both Moreover, s-EASIX 0.008) 0.005). our sC5b9, found reflect

Language: Английский

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