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    ASD—About Medications and Integrative, Complementary, and Alternative Treatments

    Updated at January 17th, 2023

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    Treating children with autism spectrum disorder (ASD) often involves a comprehensive program that addresses the education, development, and behavior of the child. Read on to learn more from the American Academy of Pediatrics about the different treatments for ASD including medications and alternative treatments.

    Medicines

    Medicines have not been shown to change or improve the core symptoms of ASD; however, they may be helpful in addressing symptoms that affect daily functioning and a child’s ability to make developmental progress.

    Medicines may be helpful when behaviors interfere with progress in a child’s intervention program. Such behaviors may include hyperactivity, inattention, irritability, aggression, self-injury, repetitive behaviors, mood disturbances, anxiety, and behaviors related to GI issues or sleep problems. The use of medicine is sometimes considered when these behavioral symptoms are judged to be interfering with learning, socialization, health and safety, or quality of life and in addition are not responding enough to behavioral treatments. Occasionally, medicine may be needed as a first-line intervention when the safety of others or the child’s safety is at risk. Sometimes a child may have an additional diagnosis, such as depression or a seizure disorder, that may be treated with medicine.

    Commonly used classes of medicines include atypical or second-generation antipsychotics (such as risperidone and aripiprazole), stimulants (such as methylphenidate and dextroamphetamine), selective serotonin reuptake inhibitors (such as fluoxetine), α 2 -adregneric agonists (such as clonidine and guanfacine), sleep-inducing medicines (such as melatonin and trazodone), and certain antiseizure medicines.

    Risperidone and aripiprazole are the only 2 medicines approved by the US Food and Drug Administration (FDA) for treatment of irritability (aggression, tantrums, or self-injury) in children with ASD. Both medicines are in the class known as atypical antipsychotics. Risperidone has been approved for use in children and adolescents with ASD aged 5 to 16 years, and aripiprazole has been approved for use in children and adolescents with ASD aged 6 to 17 years. These medicines have not been shown to improve core symptoms of autism, including social communication and repetitive or stereotypic behavior. Children treated with these medicines should be monitored closely for potential side effects such as sedation, excessive weight gain, hyperglycemia (high blood glucose [“blood sugar”] level), and abnormal twisting movements of their face or upper body.

    Before starting a child on a medicine, it is important to look for medical factors that might cause or contribute to the behavior. For example, the child may have a hidden medical source of discomfort, such as constipation or an ear infection, that is causing agitation. There may also be environmental factors, such as changes in school routines, that are upsetting to the child and causing disruptive outbursts. An FBA may help determine the cause of new disruptive behaviors and with making decisions about medicines.

    In recent years, larger, better-designed studies have been done to determine which medicines are helpful for children with ASD and associated behavior problems. For example, several studies have shown risperidone to be very effective for the treatment of tantrums, aggression, or self-injurious behavior in children with ASD. Melatonin may be helpful in regulating sleep. Doctors are guided in the use of medicine by research studies not only involving children and adults with ASD but also on related disorders such as attention-deficit/hyperactivity disorder (ADHD), obsessive-compulsive disorder, depression, and anxiety. If symptoms of these disorders are also present in a child with ASD, stimulants, antidepressants, antianxiety agents, and other medicines might be considered if behavioral strategies are not successful alone.

    It is always important to consider the potential benefits and side effects when making a decision about treatment. Such decisions are best made in partnership with doctors, therapists, teachers, and, most important, parents. It is best to rely on more than one source for information concerning the effectiveness of various medicines commonly used in children with ASD because no medicine has been universally helpful in all patients.

    Target behaviors must be measured or assessed to determine what effect the medicine is having, and monitoring for side effects is very important. Only one medicine or treatment change should be made at a time. The medicine dose often has to be adjusted depending on how well it is working and whether there are any side effects. A medicine is continued only if benefits outweigh any negative effects. Although few medicines will directly enhance language and social skills, the goals of most medicines are to allow the child to benefit more fully from educational and behavioral interventions, be included in settings with typically developing children, improve functional independence, and experience a higher quality of life.

    Integrative, Complementary, and Alternative Medicine (ICAM) Treatments

    The most effective treatments of ASD include a comprehensive, intensive program of educational, developmental, and behavioral therapies. However, progress may be slow and because researchers have not been able to explain what causes ASD, many families may try treatments that may not have been scientifically studied. These types of treatments are called integrative, complementary and alternative medicine (ICAM) treatments. When ICAM treatments enter conventional medical care, they are often called integrative medicine.

    Many families learn about these treatments on the internet, in books and magazines, and from other parents of children with ASD. It may be difficult to tell which treatments have scientific support and which don’t. It is important to understand all the potential benefits and risks of any chosen treatment. Some natural treatments can have serious side effects. Parents should discuss any use of ICAM treatments with their child’s pediatrician.

    Popularity of ICAM interventions varies over time depending on the availability of practitioners in a given region and the occasional coverage by insurance or provision by schools. Here are some treatments that are currently popular.

    Biological ICAM Treatments

    Nutritional (dietary) supplements. Supplements are used for many disorders in addition to ASD because families assume that they have fewer side effects than prescription medicine and are a natural treatment. Nutritional supplements are not monitored by the US FDA, so the concentration of the active ingredient may differ from brand to brand or batch to batch. Also, labels on supplements do not always include information on the proper dosages for children. An adult dosage may cause side effects in a child.

    There could be potential side effects from supplements, including side effects that could be toxic. Examples of side effects include hand tingling (vitamin B 6), GI symptoms, heart irregularities (magnesium), rash, and increased pressure around the brain (vitamin A). No scientific studies have been done to look for toxic effects from long-term vitamin supplement use in young children. A child’s pediatrician should be informed if the child is taking nutritional supplements.

    Diet changes. Changes to diet are another natural approach to treating many chronic conditions. Some families believe a gluten (wheat)-free and casein (milk)-free diet can help with symptoms of ASD. This is based on the theory that some children with ASD may have a “leaky gut” that allows some partially digested proteins to worsen symptoms of ASD. There is no evidence to support the leaky gut theory or that this diet improves the symptoms of ASD. However, if GI symptoms are improved by removal of certain foods, behavior in general might also improve. Children with ASD and GI symptoms might have celiac disease (gluten intolerance) or lactose intolerance (inability to digest milk sugars). Children who have diarrhea or other significant GI symptoms should be evaluated by their pediatrician. Before trying this dietary intervention, the family should talk with their child’s pediatrician and perhaps a dietitian. Adequate levels of calcium, vitamin D, and protein need to be provided in other foods if dairy products are eliminated. Vegetable- or nut-based milk substitutes may not be nutritionally equal to milk.

    Prescription or over-the-counter medicines. Sometimes, novel treatments include medicines (prescription or over-the-counter) ordinarily used for other purposes. For prescription medicines, this is called off-label use, or a use not approved by the US FDA.

    One example is secretin, an intestinal hormone, which came to attention in 1998 as a possible treatment of ASD behaviors. Many scientific studies have been done and have failed to prove that secretin is effective as a treatment of ASD.

    The immune system is another focus of medical treatment, with off-label use of medicines to decrease inflammation or overgrowth of bacteria, viruses, or yeast. Each of these treatments has its own risk of side effects that must be considered in view of the limited proof to support their use. While probiotics may be beneficial for some GI problems, the use of probiotics or other treatments to increase the microflora of the intestines has not been studied specifically for treatment of behavioral symptoms. Parents should tell their child’s pediatrician if their child is taking any of these medicines or supplements. Although there may be immunologic factors involved in the cause of some cases of ASD, there is not enough scientific proof at this time to support these modalities to treat ASD.

    It has been suggested that mercury from the preservative thimerosal in some vaccines may cause ASD. Thimerosal was taken out of standard childhood vaccines in the United States in 2000. In 2004, the Institute of Medicine concluded that there is no proof that thimerosal causes ASD, as there were no differences in the rate of ASD among children who were and were not given vaccines with thimerosal. Studies since that time have not identified an association between vaccines and ASD.

    Despite the lack of proof, some practitioners have still supported the use of certain medicines to chelate (extract) metals such as mercury. Medicines used for chelation are not approved by the US FDA for treatment of ASD and are not recommended for general use at this time. They can also remove metals needed for body function. Chelation can be dangerous and may even result in serious harm or death.

    Another controversial treatment of ASD is hyperbaric oxygen therapy. The scientific support for this intervention remains weak.

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    Nonbiological ICAM Treatments

    Auditory integration training. Children with ASD who receive AIT are conditioned to tolerate certain sounds by listening to filtered music in a sound booth twice a day over a period of time (usually 2–4 weeks). There is no evidence that AIT improves core symptoms of autism.

    Facilitated communication. Facilitated communication enlists a second person (facilitator) to help a child with ASD point to letters or words on a communication board or type by holding the child’s hand or supporting the child’s arm. Scientific studies have shown that facilitated communication is largely a function of the thoughts and activities of the facilitator and does not actually represent the thoughts or feelings of a child with ASD.

    Facilitated communication is different from alternative and augmentative communication, during which picture books or lapboards containing words, numbers, and letters or electronic devices (some speech-generating) are introduced with a goal of independent use for communication by an individual with ASD. Augmentative communication may be helpful in learning to communicate.

    Treatment, whether conventional or ICAM, should be adequately monitored. Parents should inform their child’s pediatrician about all treatments their child is using. Families should work closely with their child’s pediatrician and intervention team to

    • Determine the specific symptoms or behaviors that they hope will change or improve with any treatment.
    • Determine a method to monitor these symptoms or behaviors (for example, daily ratings).
    • Collect baseline data before treatment is started.
    • Continue data collection when treatment is started.
    • Continue data collection for long enough to account for outside changes (for example, sick child, change in family schedule).
    • Have a “blind” observer who also collects data (such as a therapist who does not know about the treatment change) if possible.
    • Make only one treatment change at a time.
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    Visit HealthyChildren.org for more information.


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