The patient subsequently developed hydrocephalus requiring a ventriculoperitoneal shunt, complicated by refractory ascites that resolved with acetazolamide therapy. Awareness of the potential disease spectrum through early molecular diagnosis, combined with a comprehensive immunologic evaluation, enabled individualized management via closer clinical monitoring and timely interventions to prevent and control neurological and infectious complications. This case highlights the phenotypic heterogeneity of PIK3CA pathogenic variants and the importance of early precision medicine in pediatric care.
The patient was started on a low phosphate diet, sevelamer, and acetazolamide and was instructed to avoid calcium and vitamin D supplements. Intravenous Zoledronic Acid was also given. The patient reported improvement in his symptoms in the follow-up visits.
P1, N=13, Active, not recruiting, University of Minnesota | Trial completion date: Jun 2027 --> Oct 2026 | Trial primary completion date: Jun 2026 --> Oct 2025
2 months ago
Trial completion date • Trial primary completion date
In anticancer assays, compound 4p (SI = 10.55) was about twice as effective as sorafenib (SI = 5.34), and in enzyme inhibition, it showed ~1.5-fold greater potency than acetazolamide. Additionally, pharmacokinetic predictions indicated excellent drug-like properties for 4p, including high permeability, oral absorption, and adherence to Lipinski's rule. These findings highlight compound 4p as a promising candidate for an anticancer agent targeting key enzymes involved in lung cancer progression.