Publications by authors named "F M Balis"
Article Synopsis
- * Diagnosis often relies on specific clinical features, but atypical presentations can lead to missed or delayed diagnoses, as seen in the five patients described.
- * Three patients with these atypical presentations developed osteosarcoma, emphasizing the need for vigilance in recognizing unusual signs of Rothmund-Thomson syndrome to ensure timely cancer monitoring.
Article Synopsis
- Clinical trials by the Intergroup Rhabdomyosarcoma Study Group and the Children's Oncology Group have set the standards for diagnosing and treating rhabdomyosarcoma (RMS), but new biological insights complicate these processes.* -
- The rarity of RMS makes it difficult to conduct large phase 3 clinical trials, highlighting the need for careful planning to explore drug effectiveness, response markers, treatment toxicity, and patient quality of life.* -
- The Children's Oncology Group Soft Tissue Sarcoma Committee proposes a strategic plan for future RMS trials that includes identifying new agents, improving trial efficiency, expanding knowledge opportunities, reducing treatment toxicity, and enhancing patient engagement.*
Population modeling analyses of crizotinib in pediatric patients with ALK-positive advanced cancers.
Pediatr Blood Cancer
September 2024
Article Synopsis
- Alterations in the ALK gene are crucial for the development of anaplastic large cell lymphoma (ALCL), and crizotinib is an approved treatment for pediatric patients with this type of cancer.
- The study included data from pediatric patients treated with crizotinib, focusing on how the drug is processed in the body and its safety and effectiveness at different doses.
- Results showed that crizotinib dosing appropriately adjusted for patient size led to similar drug exposure across different ages and tumor types, with a notable relationship between higher doses and better treatment responses, except for severe cases of neutropenia.
Background: MYC genes regulate ornithine decarboxylase (Odc) to increase intratumoral polyamines. We conducted a Phase I trial [NCT02030964] to determine the maximum tolerated dose (MTD) of DFMO, an Odc inhibitor, with celecoxib, cyclophosphamide and topotecan.
Methods: Patients 2-30 years of age with relapsed/refractory high-risk neuroblastoma received oral DFMO at doses up to 9000 mg/m/day, with celecoxib (500 mg/m daily), cyclophosphamide (250 mg/m/day) and topotecan (0.