methotrexate

P4, N=12, Recruiting, BioMarin Pharmaceutical | Not yet recruiting --> Recruiting

Tumor tissue and serial plasma samples were collected from 15 patients with aUC treated with dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin (ddMVAC). While the NOIR-SS-based assay proved sensitive and informative, limitations include the cost and time required for sequencing, potential temporal discordance between tissue and plasma sampling, and the absence of correction for clonal hematopoiesis of indeterminate potential. Overall, ctDNA profiling using this targeted panel and NOIR-SS suggested the feasibility of sensitive, non-invasive molecular monitoring in aUC, and may have future clinical applicability if validated prospectively in larger cohorts.

Systemic therapy comprised methotrexate and tumor necrosis factor-alpha (TNF-α) inhibitors. In this patient, systemic inflammatory markers did not reliably reflect intraocular disease activity, and infliximab achieved durable remission of Blau-associated uveitis refractory to adalimumab. Although limited to a single observation, the long-term outcome supports infliximab as a salvage option in refractory pediatric Blau-associated uveitis and underscores the value of ophthalmologic-rather than serological-monitoring of disease activity.

P2, N=90, Not yet recruiting, SWOG Cancer Research Network

The control group received oral methotrexate (10 mg/week) combined with folic acid (10 mg/week)...Its mechanism involves suppressing key inflammatory mediators TNF-α and IL-17A, thereby activating and regulating the Wnt/β-catenin signaling pathway. This modulates serum levels of OPG and β-CTX, leading to systemic improvement in bone metabolic balance and exerting a multi-target therapeutic effect.

In 2022, acitretin was commenced and uptitrated to 25 mg once daily...Other immunosuppressive agents, such as methotrexate, cyclosporine, and azathioprine, have been described in the literature with varying efficacy...Naltrexone is postulated to work due to its analgesic and anti-inflammatory effects. It is a well-tolerated treatment for severe HHD that has drastically improved both our patient's skin and his quality of life.

P2, N=25, Recruiting, University of Chicago | Suspended --> Recruiting | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2027 --> Mar 2028

While HPE treatment with MTX improved body weight, biochemical, ultrastructural and biophysical changes comparing to the MTX group. Human placental extract can reduce MTX-induced nephrotoxicity in rats through boosting oxidative stress/anti-oxidant balance as it is rich with essential elements.

P2/3, N=132, Not yet recruiting, Singapore General Hospital

Among the pharmacogenetic factors influencing toxicity of commonly used B-ALL treatments, variants in the TPMT and NUDT15 genes, both involved in the metabolism of 6-mercaptopurine, represent the most robust and clinically validated predictors. Emerging evidence also links additional genetic variants to toxicities associated with other key agents used in ALL treatment regimens, including variants in SLCO1B1 associated with methotrexate-related gastrointestinal toxicity and variants in CEP72 associated with vincristine-induced neuropathy. The integration of pharmacogenomic biomarkers into clinical protocols, enabling genotype-guided dose adjustment, may represent a valuable strategy to avoid toxicity and improve overall cancer outcomes. However, further studies and innovative approaches are required to validate emerging biomarkers and facilitate their translation into routine clinical practice.

Traditional IT agents such as methotrexate or cytarabine were generally associated with modest survival outcomes, whereas more recent studies evaluating IT pemetrexed and molecularly guided regimens reported longer survival in selected cohorts, particularly in EGFR-mutant NSCLC-LM. Ommaya reservoir-based delivery may offer practical advantages for repeated treatment and CSF monitoring in appropriately selected patients, with acceptable toxicity and manageable device-related risks. Emerging data on pemetrexed-based intrathecal regimens and other molecularly informed approaches suggest potential benefit in selected subgroups, but prospective, multicenter, mutation-stratified studies are needed to refine patient selection, optimize dosing strategies, and define the comparative role of different intrathecal delivery routes.

This case highlights the diagnostic challenge of primary gastrointestinal ENKL in patients presenting with abdominal pain and hematochezia in the absence of computed tomography (CT) abnormalities. Persistent unexplained abdominal pain should prompt consideration of rare ulcerative small bowel diseases, including ENKL, particularly when perforation is a potential complication. Early multidisciplinary intervention is essential to improve the outcomes of this devastating condition.