AAP

AAP: Current data don't support routine leucovorin use in autism

November 4, 2025

The American Academy of Pediatrics (AAP) has issued interim guidance stating that leucovorin (folinic acid) shouldn’t be routinely used for autistic pediatric patients due to insufficient evidence on efficacy and long-term safety. While small studies suggest potential benefit—particularly in children with cerebral folate deficiency or folate receptor autoantibodies—the data are preliminary and don’t support broad clinical recommendations.

The guidance emphasizes that key questions remain unanswered, including which patients may benefit, appropriate dosing and monitoring, and long-term risk profiles. AAP calls for large, well-controlled trials to determine whether leucovorin is a safe and effective option for autism-related symptoms.

Pediatricians are encouraged to engage in shared decision-making with families who inquire about leucovorin, providing clear, science-based information on current evidence and risks. Clinicians should also prioritize established supports and therapies while monitoring emerging research.

Source:

American Academy of Pediatrics. “Interim Guidance from the American Academy of Pediatrics: Use of Leucovorin in Autistic Pediatric Patients.” News release, Oct. 31, 2025. https://www.aap.org/en/patient-care/autism/use-of-leucovorin-in-autistic-pediatric-patients/