Publications by authors named "K Nysom"
Background: Due to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.
Methods: FIREFLY-1 investigated the efficacy (arm 1, n=77), safety, and tolerability (arms 1/2) of tovorafenib (420 mg/m2 once weekly; 600 mg maximum) in patients with BRAF-altered relapsed/refractory pediatric low-grade glioma (pLGG).
Article Synopsis
- Diffuse midline glioma (DMG) is a highly aggressive brain cancer in children and young adults, with a poor prognosis of less than 10% survival rate after two years; radiotherapy is a key treatment but only offers short-term benefits.
- The REMIT protocol aims to assess the safety and palliative effectiveness of reirradiation for DMG patients, while monitoring various health indicators, including performance status and quality of life.
- The study will also address patient selection bias and standardization issues, with plans to begin patient inclusion in 2024.
Precision cancer medicine brought the promise of improving outcomes for patients with cancer. High-throughput molecular profiling of tumors at treatment failure aims to direct a patient to a treatment matched to the tumor profile. In this way, improved outcome has been achieved in a small number of patients whose tumors exhibit unique targetable oncogenic drivers.
View Article and Find Full Text PDFPhosphatidylinositol 3-kinase (PI3-K) signalling pathway is a crucial path in cancer for cell survival and thus represents an intriguing target for new paediatric anti-cancer drugs. However, the unique clinical toxicities of targeting this pathway (resulting in hyperglycaemia) difficulties combining with chemotherapy, rarity of mutations in childhood tumours and concomitant mutations have resulted in major barriers to clinical translation of these inhibitors in treating both adults and children. Mutations in PIK3CA predict response to PI3-K inhibitors in adult cancers.
View Article and Find Full Text PDFArticle Synopsis
- Inotuzumab ozogamicin is an approved treatment for relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL) in adults, but its pharmacokinetics in children have not been studied until now.
- This research analyzed serum concentrations from a diverse patient group, utilizing a modified adult pharmacokinetic model to understand how the drug behaves in pediatric patients with BCP-ALL.
- Results indicated that older pediatric patients showed a different drug clearance pattern, with higher drug exposure correlating to better treatment responses among those with relapsed/refractory BCP-ALL.