Key takeaways:
- The American Academy of Pediatrics (AAP) issued new guidance advising against the routine use of leucovorin in children with autism, citing limited supporting evidence.
- The statement emphasizes shared decision-making, harm mitigation, and continued reliance on established autism therapies while research on leucovorin continues.
- The interim guidance follows FDA plans to update leucovorin labeling to include cerebral folate deficiency, which can present with developmental delay and autism-like features.
The American Academy of Pediatrics (AAP) has released a guidance statement saying it does not recommend the routine use of leucovorin in children with autism, citing limited evidence.1
In a press release sent on October 31, 2025, the AAP said the prescription form of folinic acid is typically used for the reduction of side effects associated with chemotherapy and to treat a specific kind of anemia caused by vitamin B12 and folate deficiencies.
“Medical decisions, including emerging therapies like leucovorin, must be thoroughly examined before we begin implementing them widely to ensure that children remain safe and healthy," said Kristin Sohl, MD, FAAP, in a statement. Sohl is a member of the AAP Council on Children with Disabilities Executive Committee and immediate past chair of its Autism Subcommittee. "It is important that we take the necessary time to understand the evidence and equip pediatricians with accurate information to navigate these conversations in support of their patients," added Sohl.
In the interim guidance, the AAP expressed support for continued research into promising therapies and provided recommendations for speaking with families about leucovorin.2
In the guidance, the AAP stated:3
"Pediatricians and prescribing pediatric care providers, including other physicians, physician assistants, and nurse practitioners, are encouraged to engage in shared decision-making with families who inquire about or request leucovorin, providing clear information about current evidence and potential risks. Autistic children, adolescents, and young adults benefit from access to robust supports and services based on their individual needs. Pediatricians and other pediatric care providers should work with families to optimize recommended services and emphasize continuation of well-established supports and therapies that are beneficial to the well-being of the child. The AAP does not have prescribing guidelines for leucovorin for the indication of autism. If a pediatrician or other physician, physician assistant, or nurse practitioner prescribes leucovorin for a pediatric patient, they should prioritize harm mitigation, closely monitor for adverse effects, and support families in navigating complex therapeutic decisions grounded in compassion, transparency, and scientific integrity."
“The reason the AAP is not broadly recommending leucovorin is because we know that autistic children deserve the same level of evidence to support interventions for them as anyone else,” added Sohl.
