Chronic myelogenous leukemia (CML) in children is relatively rare. Because of a lack of robust clinical study evidence, management of CML in children is not standardized and often follows guidelines developed for adults. Children and young adults tend to have a more aggressive clinical presentation than older adults, and prognostic scores for adult CML do not apply to children. CML in children has been considered to have the same biology as in adults, but recent data indicate that some genetic differences exist in pediatric and adult CML. Because children with CML may receive tyrosine kinase inhibitor (TKI) therapy for many decades, and are exposed to TKIs during a period of active growth, morbidities in children with CML may be distinct from those in adults and require careful monitoring. Aggressive strategies, such as eradication of CML stem cells with limited duration and intensive regimens of chemotherapy and TKIs, may be more advantageous in children as a way to avoid lifelong exposure to TKIs and their associated adverse effects. Blood and marrow transplantation in pediatric CML is currently indicated only for recurrent progressive disease, and the acute and long-term toxicities of this option should be carefully evaluated against the complications associated with lifelong use of TKIs.
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http://dx.doi.org/10.1182/blood-2015-06-648667 | DOI Listing |
ESC Heart Fail
January 2025
Cardiology Unit, University Hospital 'Paolo Giaccone', Palermo Italy and Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (ProMISE) University of Palermo, Palermo, Italy.
Aims: Knowledge of the effects of sex in cardio-oncology is limited, particularly in patients treated with tyrosine kinase inhibitors (TKIs) for chronic myeloid leukaemia (CML). This study aims to evaluate the influence of gender differences on the incidence of cardiovascular toxicity in patients with CML.
Methods: The study population consisted of 148 patients (45% women, mean age: 58 ± 14.
ACS Pharmacol Transl Sci
December 2024
Department of Biology and Biotechnologies "Charles Darwin", Sapienza University of Rome, 00185 Rome, Italy.
FTO, an -methyladenosine (mA) and ,2'--dimethyladenosine (mA) RNA demethylase, is a promising target for treating acute myeloid leukemia (AML) due to the significant anticancer activity of its inhibitors in preclinical models. Here, we demonstrate that the FTO inhibitor FB23-2 suppresses proliferation across both AML and CML cell lines, irrespective of FTO dependency, indicating an alternative mechanism of action. Metabolomic analysis revealed that FB23-2 induces the accumulation of dihydroorotate (DHO), a key intermediate in pyrimidine nucleotide synthesis catalyzed by human dihydroorotate dehydrogenase (DHODH).
View Article and Find Full Text PDFJ Trauma Acute Care Surg
January 2025
From the Department of Surgery (K.M.M.), University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Department of Surgery (E.V.F.), University of Pittsburgh, Pittsburgh, Pennsylvania; Department of Surgery and Critical Care Medicine (P.C.S., C.M.L.), University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; and Department of Surgery (B.A.G.), University of Texas Southwestern, Dallas, Texas.
Background: Trauma-induced coagulopathy is common and associated with poor outcomes in injured children. Our aim was to identify patterns of coagulopathy after injury using endothelial, platelet, and coagulation biomarkers, and associate these phenotypes with relevant patient factors and clinical outcomes in a pediatric trauma cohort.
Methods: Principal component (PC) analysis was performed on data from injured children between 2018 and 2022.
Cancers (Basel)
December 2024
Independent Laboratory of Genetic Diagnostics, Medical University of Lublin, 20-093 Lublin, Poland.
Myeloproliferative neoplasms (MPNs) are clonal hematopoietic cancers characterized by hyperproliferation of the myeloid lineages. These clonal marrow disorders are extremely rare in pediatric patients. MPN is reported to occur 100 times more frequently in adults, and thus research is primarily focused on this patient group.
View Article and Find Full Text PDFPharmacoepidemiol Drug Saf
December 2024
Division of Clinical Pharmacology, Department of Medicine, University of Cape Town, Cape Town, South Africa.
Background: Determining a therapeutic window for maintaining antiretroviral drug concentrations within an appropriate range is required for identifying effective dosing regimens. The limits of this window are typically calculated using predictive models. We propose that target concentrations should instead be calculated based on counterfactual probabilities of relevant outcomes and describe a counterfactual framework for this.
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