PTX3 Genetically Stratified Randomized Double-blinded Allocation Event-driven Clinical Trial for Antifungal Prophylaxis in Patients With Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating the use of posaconazole-based antifungal prophylaxis strategies in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome in transformation (MDSit) who are receiving induction chemotherapy. The trial focuses on genetically stratifying patients based on PTX3 gene single nucleotide polymorphisms (SNPs) to predict their risk for invasive mold infections (IMI), which are serious fungal infections with high incidence and mortality among hematological patients. This is a prospective, randomized, double-blind, multicenter clinical trial assessing the effectiveness of antifungal prophylaxis allocation based on genetic risk. Eligible patients will have their PTX3 SNPs tested through blood samples to identify high or low genetic risk for IMI. Patients with high-risk genotypes will be randomized equally to receive either posaconazole or fluconazole prophylaxis, while those with low-risk genotypes will be randomized mostly to fluconazole. Posaconazole is a broad-spectrum antifungal active against molds, while fluconazole targets mainly Candida species. Randomization occurs within 24 hours after the first day of neutropenia, and patients receive prophylaxis during their neutropenic phase. Participants will be monitored for 180 days after starting prophylaxis for proven, probable, or possible invasive fungal infections using established diagnostic criteria. The study includes regular assessments of infection incidence, mortality, adverse events, treatment success or failure, and use of additional antifungal treatments. Hospital admission is planned for the neutropenic period, and study adherence and safety are monitored throughout. The total involvement lasts at least six months to capture infection outcomes and related health events.

PTX3-targeted Antifungal Prophylaxis