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Year : 2017  |  Volume : 15  |  Issue : 1  |  Page : 28-37

Medication in autism

Department of Psychiatry, Child and Adolescent Psychiatry Unit, Christian Medical College, Vellore, Tamil Nadu, India

Date of Web Publication17-Feb-2017

Correspondence Address:
Sherab Tsheringla
Department of Psychiatry, Child and Adolescent Psychiatry Unit, Christian Medical College, Vellore - 632 002, Tamil Nadu
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Source of Support: None, Conflict of Interest: None

DOI: 10.4103/0973-4651.200308

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Autism affects up to 1 in 250 children. Early intervention programs combining behavioral and developmental training are the current standard of care. In this article, the role of medication in children and adolescents with autism spectrum disorders (ASDs) is discussed. We highlight the key symptoms that can be targeted with medication-based treatments. An approach to treatment is based on available guidelines which includes clinical assessments, addressing comorbid conditions, planning investigations prior to starting pharmacotherapy, choosing suitable pharmacological agents, and subsequent monitoring while on medication. Risperidone and aripiprazole continue to be the only approved medications. Continued support and counseling of caregivers after starting medication includes discussions on monitoring behavioral change along with any adverse effect and discussing further referral when needed. Primary care physicians in India are an important resource in the absence of developed systems for the management of developmental disabilities. Early referral to specialists for initial diagnosis and developmental interventions cannot be overemphasized. However, a community-based follow-up model for continued care of children and adolescents with ASD that includes primary care physicians can be implemented.

Keywords: Aripiprazole, autism, medication in autism, risperidone

How to cite this article:
Tsheringla S. Medication in autism. Curr Med Issues 2017;15:28-37

How to cite this URL:
Tsheringla S. Medication in autism. Curr Med Issues [serial online] 2017 [cited 2022 Aug 13];15:28-37. Available from: https://www.cmijournal.org/text.asp?2017/15/1/28/200308

Case discussion

A 6-year-old Jacob is brought to the primary health center by his parents. Jacob has previously seen specialists in Delhi who have diagnosed him to have autism spectrum disorder. His parents were taught behavioral management strategies and asked to follow up for therapy for developmental needs. However, living in rural Odisha, they are unable to continue training and cannot follow up in the tertiary clinic in Delhi. He is currently brought by his parents who are concerned that his behavior has worsened. They complain of increased restlessness, repeatedly rocking his body and flapping his hands, biting his fingers, not reading his school books, not playing with other children, and no improvement in his speech. They request the primary health center physicians to start him on some medication to improve these symptoms.

For discussion in primary care setting

  • Which of Jacob's symptoms would be targeted by medication?
  • What are the key points you will discuss with Jacob's parents before starting medication?
  • What will you monitor for after starting medication?

  Introduction Top

The first line of treatment for children with autism spectrum disorders (ASDs) is not medication.[1],[2] Early intensive behavioral training is the evidence-based treatment to be recommended for all children with autism. Parents usually come to clinicians with expectations of curing their child from the developmental disorder. They may sometimes be willing to try a variety of medications without understanding the indications or the possible harm from starting these drugs. Medicines can help with certain behaviors.[3] However, it is equally important to understand the harmful effects in children with developmental disorders, who may be more prone to side effects than typically developing children. It may also be prudent to start one intervention at a time to better monitor improvement or worsening of behavior. The decision to start medication involves the parent, child's therapists (if available), and physicians working together.[4]

Using medicines for children with autism spectrum disorders: What a physician needs to know

Autism presents with problems in three domains which are:

  1. Reciprocal social interaction
  2. Communication
  3. Repetitive or stereotyped patterns of behavior.

Current scientific literature suggests that drug therapy is effective primarily for the domain of repetitive or stereotyped patterns of behavior.[3] In addition, aggressive behavior in children with autism also responds to medication. In a primary care setting, caregivers may seek advice on which symptoms of Autism respond to medication or for follow-up after starting medication in a tertiary care clinic. These are summarized in [Table 1].
Table 1: Symptoms of autism and medication

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It is also important to remember that children with autism are likely to have comorbid conditions which also require pharmacotherapy, which is discussed later in the article.

The decision to prescribe medication involves both the parents/caregivers and the clinician [Figure 1]. The target behavior that requires medication, their beliefs about the effect of medication have to be identified and clarified. The parents/caregivers may then decide to either start medication or wait. If they decide to wait, the child must be followed up and reviewed by the clinician regularly and the decision to start medication may be taken later based on the clinical picture.
Figure 1: Deciding on medication to treat autism.

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Approach to prescribing medication for children with autism

The management of autism is multimodal and the strategies include (a) behavioral interventions, (b) educational interventions, (c) caregiver training and family support, and (d) medical management. It should be emphasized again that medical management is not the first line of management, it is only a part of the management of the problem and is effective only for some of the symptoms of autism. Parent training programs which use parents as behavioral change agents to promote skill acquisition and generalization of acquired skills to home and community have the most empirical evidence.[5]

The decision to prescribe medication involves both the parents/caregivers and the clinician [Figure 1]. The target behavior that requires medication, their beliefs about the effect of medication have to be identified and clarified. The parents/caregivers may then decide to either start medication or wait. If they decide to wait, the child must be followed up and reviewed by the clinician regularly and the decision to start medication may be taken later based on the clinical picture.

The approach to prescribing medication involves the following steps.

  Clinical Assessment Top

Clinical assessment involves initial evaluation after establishing a diagnosis of autism, identification of target symptoms, and quantification of these symptoms.

Initial evaluation

Initial evaluation before starting medication for children who have ASD may reveal important information about the child's condition. Once a diagnosis of ASD has been established, the clinician should also look for other neurodevelopment disorders such as intellectual disability (ID). ID cannot be treated with medication but is a significant prognostic factor in the improvements shown by children with ASD. Diagnosis of ID will also require holistic developmental interventions to address the multiple skill deficits and disabilities that the child is presenting with. For example, very often children with both ASD and ID are referred only for speech therapy, which disregards the need for other interventions such as occupational therapy and behavioral strategies.

Identify target symptoms

Once the clinician and caregivers have identified target symptoms [Table 1] for which medication use is being considered, the following steps should be followed:

  • Collect information about symptoms from multiple sources as well as other parent, family members, and school teachers
  • Corroborate historical information with observations if possible - observing the child at home, while playing or in classroom settings. The child's behavior in the clinician's office can often be very different from the presentation at home or school
  • Consider differential diagnosis for behavioral change [Table 2] and rule them out before initiating treatment
  • The parents' understanding of target behavior must be clarified because the behavior that they would prefer treatment for may differ from the target behavior identified by the clinician.
Table 2: Differential diagnosis for behavioral change

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Quantify the behavioral problems

Psychologists use various rating scales to quantify problem behavior. If available, these are useful instruments in clinical practice. The Childhood Autism Rating Scale (CARS) is an instrument used to diagnose and assess the severity of autism. However, medication use is not solely decided on the scores in this scale. Similarly, the Child Behaviour Checklist (CBCL) identifies the various behavioral problems in the child and has the advantage of providing a more comprehensive assessment of the child's behavioral needs. In primary care practice, where trained assessors may not be available, an alternate method that can be used is to rate target symptoms on a 0–10 Likert scale for severity at baseline and follow-up [Appendices 1 and 2 for CARS and Likert scale].

  Comorbidity Top

The behavioral problems seen in children with ASD can also either be worsened by the presence of comorbidities or be the presentation of a comorbid condition by itself. Autism is understood to be caused by neurobiological changes in the developing brain, have multiple genetic mechanisms and epi-genetic influences, and a multitude of postulated environmental factors. With shared biological and environmental factors, comorbid conditions are commonly seen in children with ASD. The following comorbidities should be identified and treated in children with ASD.


Up to 40% of children with ASD have an underlying seizure disorder. Seizures can worsen behavioral problems. Sometimes, anti-epileptics can also exacerbate behavior problems, for example, worsening hyperactivity on phenobarbitone and phenytoin. There is also a high prevalence of electroencephalogram (EEG) abnormalities in children with ASD. Clinical correlation of EEG findings should be considered before initiating anti-epileptic treatments

Nutritional deficiencies

Studies from low-income countries have shown that children with developmental disorders should be screened for malnutrition. Selective eating practices and other feeding difficulties can affect the nutritional status of these children. Many children also have pica or nonnutritive eating habits which require appropriate treatment

Gastrointestinal disorders

The “brain-gut hypothesis” is often cited, stating gastrointestinal problems in children with ASD. From sensitivity to certain foods to malabsorption syndromes and structural abnormalities, any child with ASD with suspected gastrointestinal disease should undergo further evaluation.

Sleep disorders

Before diagnosing a sleep disorder, it is advisable to elicit the history of child's sleeping patterns. Often, poor sleep hygiene with chaotic sleep timings and excess napping in day time or watching television till late in night are responsible for sleep problems. In children who do have sleep disorder in spite of good sleep hygiene, medication may be considered. As a word of caution, benzodiazepines should not be prescribed as they can cause paradoxical worsening of behavior and also develop a dependence syndrome. Medications such as risperidone and aripiprazole which are started for autism-related behavioral concerns can improve sleep. For specific sleep onset-related problems, melatonin, which is usually prescribed for up to 6 months duration, can be prescribed


Hyperactivity, poor motor co-ordination, lack of environmental awareness, poor understanding of safety concepts, sensory issues, and behaviors such as repetitive movements, self-injurious behavior, and aggression make children with ASD prone to sustain physical injuries. Head injuries would require further evaluation with neuro-imaging (computed tomography brain, NICE guidelines 2014)

Mental health conditions

Anxiety and depression are common mental health disorders which are seen in this population. Other mood disorders such as bipolar disorder and psychosis should also be considered in the presence of new-onset abnormal behavior. Appropriate referral for management of psychiatric disorders should be advised to the families.

  Investigations Top

Laboratory investigations are required for diagnosing physiological etiology of behavioral symptom and as a baseline before initiating pharmacotherapy. The following investigations are suggested where available [Table 3].
Table 3: Laboratory investigations

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  Prescribing Medication Top

”Pharmacotherapy may be offered to children with ASD when there is a specific target symptom or co-morbid condition” (American Academy of Child and Adolescent Psychiatry practice parameters 2014). Risperidone [6],[7],[8] and aripiprazole [9] are the only two US FDA approved medication for use in children with ASD. Both these drugs are second-generation, atypical antipsychotics, and serotonin dopamine antagonists.

Medications available in India

In a primary care setting in India, risperidone is currently more widely available. A syrup formulation of risperidone is also marketed in India, which some parents might find easier to administer. Caregivers of children with ASD often complain of the children being very restless, fidgety, and inattentive. The first-line drug to treat these symptoms which may suggest a comorbid Attention Deficit Hyperactivity Disorder (ADHD) is a stimulant. Methylphenidate [10],[11],[12] is currently the only stimulant available in India. Atomoxetine,[13],[14] a norepinephrine re-uptake inhibitor, is the next line of treatment for ADHD which is also commonly prescribed. The alpha-agonist clonidine [15] can also be prescribed for children with significant restlessness and is also found to be effective in treating irritability in these children. Selective serotonin re-uptake inhibitors such as fluoxetine [16] are effective in treating comorbid anxiety and depression. They may also have a role in reducing repetitive behavior and self-injurious behavior. Valproate, naltrexone,[17],[18] and other medications have been used for treating autism [Table 4],[Table 5],[Table 6].
Table 4: Medications used for autism

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Table 5: Other medication used for autism and their indications

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Table 6: Evidence base for commonly used medication for children with autism spectrum disorder

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  Monitoring Top

Children who have been started on medication require monitoring of physical parameters. Along with the baseline investigations, clinical assessment should also include the following before starting medication:

  • Height
  • Weight
  • Body mass index
  • Pulse
  • Blood pressure.

These parameters should also be documented at every subsequent visit to the clinician. Monitoring should include the following at least for every 6 months:

  • Fasting and postprandial blood sugars
  • Fasting lipid profile
  • Liver function tests.

Specific psychotropics may require additional tests to be done on a periodic basis. Clinical monitoring is required depending on the adverse effects of the medication chosen [Table 7].
Table 7: Common adverse effects of medications

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  Counseling Caregivers About Medication Use Top

Caregivers, who are usually the parents or other family members, bear a considerable burden during the treatment of a child with autism. Counseling and training of these individuals is, therefore, vital, especially when medications are planned.

Caregivers may require to be reminded of the reasons why the medication was started and discuss change in target behavior over time. They should also be educated about the common adverse effects seen with the prescribed medication. Families should be told when and where to seek help if any concern related to the use of medication arises. Caregivers may be concerned that the child will get “addicted” to the medication. Though tolerance to the dosage prescribed may occur, dependence syndromes are uncommon with drugs such as risperidone and aripiprazole. Caregivers are sometimes worried about the child taking medication “life long.” Many behavioral problems show remission with increasing age, for example, reduction in hyperactivity in adolescence. Behavioral modification also occurs with improvement in skill deficits and behavioral therapy. Thus, it is possible that medication can be stopped on review consultations. It is also advisable to ask families to check with their general pediatricians when prescribing medication for other physical illnesses for potential drug interactions. The importance of continuing behavioral interventions while on medication has to be emphasized to the caregivers. At the same time, a therapeutic alliance has to be maintained with the family to ensure compliance with drug therapy when needed.

Complementary and alternative medicine

Many caregivers consider complementary and alternative medicine (CAM) practices, even prior to medical consultation. The current evidence base does not support the use of CAM in autism. However, families who consider CAM should be provided adequate guidance in their choice of therapies, especially with the possibility of significant pharmaco-kinetic interactions.

  Referral Top

Children who do not show improvement or worsen after starting first-line drugs should be referred for further care. A lower threshold for referral should be applied for children with significant aggression or self-injurious behavior. The presence of multiple comorbidities requiring poly-pharmacy should also be done under the supervision of a specialist dealing with psychopharmacology. Common adverse effects may require only symptomatic treatment. However, if the child develops severe adverse effects such as acute or tardive dystonias and dyskinesias, referral to a tertiary care facility is suggested. The caregivers may often also have significant mental health-related issues which should be referred to mental health professionals, especially if they affect the functioning of the child.

  Conclusions Top

  • Medicines are not the first line of treatment in autism. They are usually given as an adjunct to behavioral therapy. Drug therapy is effective primarily for the domain of repetitive or stereotyped patterns of behavior. Hence, it is important to delineate the target symptoms before starting therapy
  • Work with families in making medication plan. The family or caretaker must be educated about the intended effect of medications, adverse effects, and the importance of behavioral therapy
  • Each child with autism can respond differently to medicines. Therapy should, therefore, be tailored for each individual based on observation of the child. This takes time and a concerted effort of the physician, behavioral therapist, occupational therapist, and the parents/caregivers.

Financial support and sponsorship


Conflicts of interest

There are no conflicts of interest.

  References Top

Volkmar F, Siegel M, Woodbury-Smith M, King B, McCracken J, State M; American Academy of Child and Adolescent Psychiatry (AACAP) Committee on Quality Issues (CQI). Practice parameter for the assessment and treatment of children and adolescents with autism spectrum disorder. J Am Acad Child Adolesc Psychiatry 2014;53:237-57.  Back to cited text no. 1
Autism in Under 19s; Support and Management, NICE Guidelines; Clinical guideline Published: 2013. Available from: https://www.nice.org.uk/guidance/cg170/ nice.org.uk/guidance/cg170. [Last accessed on 2017 Jan 18].  Back to cited text no. 2
McPheeters ML, Warren Z, Sathe N, Bruzek JL, Krishnaswami S, Jerome RN, et al. A systematic review of medical treatments for children with autism spectrum disorders. Pediatrics 2011;127:e1312-21.  Back to cited text no. 3
ATN/AIR-P Medication Decision Aid. Available from: https://www.autismspeaks.org/science/resources-programs/autism-treatment-network/tools-you-can-use/medication-guide. [Last accessed on 2017 Jan 15].  Back to cited text no. 4
Aman MG, McDougle CJ, Scahill L, Handen B, Arnold LE, Johnson C, et al. Medication and parent training in children with pervasive developmental disorders and serious behavior problems: Results from a randomized clinical trial. J Am Acad Child Adolesc Psychiatry 2009;48:1143-54.  Back to cited text no. 5
McCracken JT, McGough J, Shah B, Cronin P, Hong D, Aman MG, et al. Risperidone in children with autism and serious behavioral problems. N Engl J Med 2002;347:314-21.  Back to cited text no. 6
Shea S, Turgay A, Carroll A, Schulz M, Orlik H, Smith I, et al. Risperidone in the treatment of disruptive behavioral symptoms in children with autistic and other pervasive developmental disorders. Pediatrics 2004;114:e634-41.  Back to cited text no. 7
McDougle CJ, Scahill L, Aman MG, McCracken JT, Tierney E, Davies M, et al. Risperidone for the core symptom domains of autism: Results from the study by the autism network of the research units on pediatric psychopharmacology. Am J Psychiatry 2005;162:1142-8.  Back to cited text no. 8
Marcus RN, Owen R, Kamen L, Manos G, McQuade RD, Carson WH, et al. A placebo-controlled, fixed-dose study of aripiprazole in children and adolescents with irritability associated with autistic disorder. J Am Acad Child Adolesc Psychiatry 2009;48:1110-9.  Back to cited text no. 9
Quintana H, Birmaher B, Stedge D, Lennon S, Freed J, Bridge J, et al. Use of methylphenidate in the treatment of children with autistic disorder. J Autism Dev Disord 1995;25:283-94.  Back to cited text no. 10
Pearson DA, Santos CW, Aman MG, Arnold LE, Casat CD, Mansour R, et al. Effects of extended release methylphenidate treatment on ratings of attention-deficit/hyperactivity disorder (ADHD) and associated behavior in children with autism spectrum disorders and ADHD symptoms. J Child Adolesc Psychopharmacol 2013;23:337-51.  Back to cited text no. 11
Research Units on Pediatric Psychopharmacology Autism Network. Randomized, controlled, crossover trial of methylphenidate in pervasive developmental disorders with hyperactivity. Arch Gen Psychiatry 2005;62:1266-74.  Back to cited text no. 12
Arnold LE, Aman MG, Cook AM, Witwer AN, Hall KL, Thompson S, et al. Atomoxetine for hyperactivity in autism spectrum disorders: Placebo-controlled crossover pilot trial. J Am Acad Child Adolesc Psychiatry 2006;45:1196-205.  Back to cited text no. 13
Harfterkamp M, van de Loo-Neus G, Minderaa RB, van der Gaag RJ, Escobar R, Schacht A, et al. A randomized double-blind study of atomoxetine versus placebo for attention-deficit/hyperactivity disorder symptoms in children with autism spectrum disorder. J Am Acad Child Adolesc Psychiatry 2012;51:733-41.  Back to cited text no. 14
Jaselskis CA, Cook EH Jr., Fletcher KE, Leventhal BL. Clonidine treatment of hyperactive and impulsive children with autistic disorder. J Clin Psychopharmacol 1992;12:322-7.  Back to cited text no. 15
Hollander E, Phillips A, Chaplin W, Zagursky K, Novotny S, Wasserman S, et al. A placebo controlled crossover trial of liquid fluoxetine on repetitive behaviors in childhood and adolescent autism. Neuropsychopharmacology 2005;30:582-9.  Back to cited text no. 16
Hellings JA, Weckbaugh M, Nickel EJ, Cain SE, Zarcone JR, Reese RM, et al. A double-blind, placebo-controlled study of valproate for aggression in youth with pervasive developmental disorders. J Child Adolesc Psychopharmacol 2005;15:682-92.  Back to cited text no. 17
Campbell M, Anderson LT, Small AM, Adams P, Gonzalez NM, Ernst M. Naltrexone in autistic children: Behavioral symptoms and attentional learning. J Am Acad Child Adolesc Psychiatry 1993;32:1283-91.  Back to cited text no. 18


  [Figure 1]

  [Table 1], [Table 2], [Table 3], [Table 4], [Table 5], [Table 6], [Table 7]


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