Autism Therapy: off-label

definition of off label: For medications, the term “off-label” refers to when a physician prescribes a medication for use in a condition that is not included in FDA-specified uses for that medication. Physicians are allowed to use personal judgment and prescribe medication for a non-indicated or “off-label” use when the physician thinks that the medication may help someone with a different condition than what the FDA has indicated (see indications). Many medications are used off-label to treat conditions that are not indicated by the FDA. Typically, case studies then report if the medications worked in such off-label uses. If they worked, then larger clinical trials are performed to see if the medication is able to be helpful for most people with that specific condition. When there are enough positive clinical studies, the FDA may revise indications for the drug to include the new condition.

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Paediatric and Perinatal Drug Therapy, by Satish, HP, Payne H., Potter F., Nunn AJ, and Brodbelt K., published in 2008, summarized Nov 14, 2008

Overdose of risperidone may make it difficult for a child to breathe.

In the US risperidone may be prescribed to treat behavior problems in children with autism. In the UK, risperidone is not approved for use in children and, therefore, a child using risperidone has to have it prescribed "off-label." This case study describes a 10-year-old boy with mild developmental delay who took ten times more risperidone than he should have been taking. The mistake seemed to have happened because the boy was given a pipette that was made to give the correct dose to an adult, not a child. The dosage was toxic and the boy stopped breathing. The authors point out that 6-8% of white patients take a long time to clear risperidone from their bodies and this may make adverse events more common with risperidone.


Progress in Neuro-Psychopharmacology & Biological Psychiatry, by Zdanys, K., and Tampi RR, published in 2008, summarized Sep 11, 2008

While memantine may help with some of the problem behaviors associated with autism, more research is needed.

This review article describes the use of the brain drug memantine to treat pervasive developmental disorders (PDD). Memantine acts on the brain chemical glutamate, which is known to be involved in brain growth. Some genetic studies point to glutamate as having a role in autism. Memantine drug therapy may be most helpful with regards to routines and rituals that are observed in some people with autism. The authors found five open label studies and one case report on the use of memantine to treat autism in children and adults.


Journal of Child and Adolescent Psychopharmacology, by Valicenti-McDermott, MR, and Demb H., published in 2006, summarized Jan 22, 2007

Aripiprazole may help disruptive behavior in children with autism, but it can also cause significant weight gain.

Earlier studies have found that atypical antipsychotics such as Risperdal can help treat autism. A small study has also found that 12 mg/day of the atypical antipsychotic aripiprazole can help with aggression, self-injurious behavior, and hyperactivity in children with autism. This study of 32 children looked at whether aripiprazole is safe and effective in children with developmental disabilities including autism. The study found that about half of the children given aripiprazole were better behaved. It also found that weight gain was a serious problem, especially for younger children treated with aripiprazole.


Clin Neuropharmacol, by Allain, H., and Bentue-Ferrer D., published in 2006, summarized Oct 18, 2006

This article gives an overview of the implications of the use of galantamine (Reminyl) to treat autism.

Galantamine is an acetylcholinesterase inhibitor that is classified as an anti-dementia drug. Officially, galantamine is indicated only for the treatment of Alzheimer's disease. However, recent clinical trials have encouraged physicians to write off-label prescriptions for acetylcholinesterase inhibitors to treat other forms of brain disorders, including autism. Galantamine is able to help the brain release chemicals, which are critical for sending information between different parts of the brain. In this way, galantamine may be able to improve many brain functions and not just treat problems associated with Alzheimer's disease. The authors concluded by painting an optimistic future of the ability of drugs to improve brain function.


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Citalopram: No Effect in Autism

Dec 3, 2009 by Anonymous

Does citalopram help children with autism? A Citalopram (Cilexa®) study made a big splash in the autism community this month. Citalopram is an antidepressant that has been prescribed off-label for children with autism. Doctors thought it might reduce repetitive behaviors (rocking, hand flapping, etc.) in children with autism. A new study suggests that it probably won’t make a difference.  

Citalopram is a selective serotonin reuptake inhibitor (SSRI) which is a class of drugs that are FDA-approved for depression. SSRIs are said to be widely prescribed off-label for children with autism, but hard numbers are not known because of the off-label use. They are prescribed off-label for other conditions, including obsessive compulsive disorder (OCD) in children.  

King et al (2009) reported results of a large randomized, placebo-controlled, double-blind study of citalopram with 149 children (5-17 years old). The children had autism, Asperger syndrome, or pervasive developmental disorder not otherwise specified (PDD-NOS). All of the children had moderate to severe compulsive or repetitive behaviors. They received citalopram therapy (10-20 mg/day) or placebo for 12 weeks. 

Citalopram therapy did not reduce repetitive behaviors or improve any other behaviors that they measured more than placebo. The authors tested more than 11 behaviors and skills using 3 different standard clinical tests.  

Why is this important? For one thing, citalopram may be commonly prescribed for children with autism, though it is tough to know just how often. Pair that tidbit with the solid design study. The design is quite solid for this kind of study. They had 149 children in the study, placebo controls, double-blinded design, 12 weeks of drug therapy, and 3 standardized tests for 11 different behaviors. And they found no effect.  

The second reason it is important is that this study also showed a noticeable “placebo effect.” The placebo effect is when people who take placebo in the study report having a response (either better or worse). In this study, 34% of children taking placebo showed improvement in the behavioral test scores. The patients and the researchers didn’t know which they were receiving (hence the term “double-blind”). So about 1/3 of the patients showed improvements, whether they took the placebo or the drug



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