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Doctors say evidence lacking to prescribe the drug broadly

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Gaps in data leave physicians confused about dosing,

safety,

expert says

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Group urges shared decisionmaking, focus on safety

By Julie Steenhuysen

CHICAGO, Oct 31 (Reuters) - The American Academy of

Pediatrics on Friday said it does not recommend the routine use

of leucovorin - a form of vitamin B9 - in children with autism,

citing a lack of evidence on the treatment's benefits and risks

to support widespread use in this population.

The announcement follows a flood of demand from parents

eager to try the treatment after U.S. health officials touted

the decades-old drug in a White House press conference on

September 22 that focused on autism causes and treatments.

The generic drug, originally developed by GSK, is a

form of folinic acid currently used in people undergoing

chemotherapy but can be prescribed off-label for other uses.

The FDA said it has started the process of approving the

drug for people with a rare genetic condition called cerebral

folate deficiency that can cause autism-like symptoms, based on

an analysis of published data on some 40 cases showing a

benefit.

The agency also mentioned the potential use of the treatment

in individuals with autism, but cautioned that available data

for this population was limited and said additional studies were

needed to assess its safety and efficacy.

Dr Kristin Sohl, an autism expert at the University of

Missouri who worked on the guidance, said that while the FDA

intends to approve leucovorin for a rare neurological condition,

there is "very limited evidence" for its use in autism, leading

the AAP to advise against a broad recommendation for its use.

Sohl said the AAP's goal is to help pediatricians speak with

parents about the evidence behind the treatment and urged shared

decision-making, but acknowledged that there are many unknowns.

"We don't know who this medicine is supposed to work for. We

don't know whether it's safe. We don't know what dose to start

with," she said.

"If this is something that can be supportive of an autistic

child's developmental progress, then great, but we also don't

want to just throw things at someone because we don't know what

else to do," she said.

If a practitioner decides to prescribe leucovorin for

autism, the group recommends that clinicians prioritize reducing

the risk of harm and closely monitoring for harmful side

effects.

Sohl said she has faced many requests for the treatment in

her own practice since the announcement, and so far has only

prescribed it once, and only in the context of a controlled

study agreed upon by the parent and the patient.