Study data show treatment with the natural antioxidant quercetin is safe for pediatric and young adult patients with Fanconi anemia (FA) and suggest it reduces reactive oxygen species (ROS) levels in peripheral blood and the bone marrow stem cell compartment.
A phase 1, prospective study of quercetin in patients with FA was conducted at a single center to determine the recommended dose for a subsequent expansion cohort study. Twelve patients, with a median age of 7 years, received twice-daily oral quercetin for 4 months. They had excellent toleration of quercetin at all dose levels, and a weight-adjusted maximum daily dose of 4,000 mg/d was established for the expansion cohort.
The 18-patient expansion cohort received the recommended quercetin dose for 6 months. Treatment was again well-tolerated by patients in the cohort, and they achieved an a priori-defined optimal response of 25% reduction versus baseline in their peripheral blood ROS levels. Over the study period, there was a gradual decrease in the patients’ absolute neutrophil counts (P=0.01) and hemoglobin levels (P=0.001), and their platelet counts either remained stable or slightly improved (P=0.06).
15 of 18 patients in the expansion cohort at baseline had evidence of bone marrow failure, and among them, 53% had a hematological response after receiving quercetin treatment.
The investigators who conducted the study acknowledged that their conclusions are limited by the study’s small size and the fluctuations in blood counts and inflammatory markers that occur in patients with FA. To address these issues, they propose it would be necessary for studies to initiate treatment earlier in patients and have longer follow-up periods spanning several years.
Reference
Mehta PA, et al. Blood Adv. 2025;9(8):1927-1939. doi:10.1182/bloodadvances.2024015053
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