Leucovorin and Autism Spectrum Disorders
Benjamin N. Rome, MD, MPH, et al has succinctly covered the topic of leucovorin and its use for the treatment of Autism Spectrum Disorders. He stated as much in his article that appeared in Pediatrics, vol 158, issue 2, Aug. 2026, pg. 73. I took the liberty of lifting his own words from the article:
"The use of leucovorin among children increased substantially in 2025 following media coverage and a press conference attended by FDA leaders discussing the drug's use for ASD, including a 14-fold increase among those aged 0 to 10 years."
"There is limited evidence supporting leucovorin to treat ASD, including observational studies linking prenatal folic acid supplementation with lower risk of ASD and a few small studies showing modest improvements for children treated with leucovorin compared with placebo."
"The publication of one such study was retracted in January 2026. Following the FDA announcement and press conference, professional societies including the American Academy of Pediatrics issued statements not recommending the routine use of folinic acid to treat ASD. Ultimately, in March 2026, the FDA approved leucovorin for treating cerebral folate transport deficiency but not for treating ASD."
"The premature and inaccurate messaging around leucovorin resulted in many young children with ASD being exposed to a treatment with unknown effectiveness and may have perpetuated public distrust in a regulatory process unmoored to scientific evidence. Additionally, after the rise in off-label use, shortages of leucovorin were reported that may have made it more difficult for patients with cancer to obtain the medication for uses with well-established evidence."
Once again, the therapeutic gun went off "half cocked". Let's not subject our children to therapeutic misadventures.
Vitamin K Refusal for Newborns
I would have thought the neonatal use of vitamin K to prevent hemorrhagic disease of the newborn was no longer in question. Well, I guess not.
In the August edition of AAP News, pg. 11, Melissa Jenco wrote Members of Congress Urge Tracking of Rising Vitamin K Refusal for Newborns. She then quoted the AAP President, Andrew D. Racine, MD, PhD, FAAP:
"...false and misleading claims have led to increased rates of refusal, putting the youngest and most vulnerable babies at unnecessary risk, and now we're seeing the devastating impacts."
Why must we risk our most vulnerable babies, the neonates, because of undocumented claims regarding their treatment with vitamin K? Please parents, wake up.
On this topic I must refer you to an article that appeared in Pediatrics, vol 157, issue 5, May 2026, pg. 1, authored by Jeremy W. Jacobs, MD, MHS, et al, entitled Vitamin K Deficiency Bleeding After Refusal: A Sentinel Event in a Misinformation Era.
This article spells out the problem clearly and the author summarizes the situation perfectly:
"Pediatrics has a long tradition of preventing rare but devastating outcomes with simple, low-cost, high-value prophylactic measures. The case described here is a sentinel harm that should prompt 2 actions: (1) continued surveillance of rising IM vitamin K nonreceipt and (2) a systems-level response that treats refusal as a high-risk condition with standardized mitigation, just as we do for other predictable perinatal hazards. If refusal continues to rise, the question is not whether we will see more late-onset VKDB, but whether health systems will be prepared to prevent the preventable."
