Use of Medicines in the management of autism

Use of medicines in the management of ASD



  • Medicines do not treat the core deficits of autism.
  • Do a thorough clinical evaluation of maladaptive behaviour and establish its severity and pattern
  • Identify any environmental factors that may be causing the behaviour or making it worse.
  • Consider health problems that may be causing the behaviour or making it worse.
  • Consider the child’s and the family’s needs.
  • Only use medicines to target specific symptoms indicated for the medicine.
  • FIRST DO NOHARM: All medicines listed below have side effects, that may adversely affect the child. Know the side effects, inform parents about side effects and monitor the child for the side effects. IF YOU START THE MEDICINE, MONITORING THE SIDE EFFECTS IS YOUR RESPONSIBILITY.
  • Monitor the target symptom. Stop the medicine if there is no effect.
  • Do not prescribe more than one of the medicines listed below at any one time.


  • Only prescribe supplements, such as multivitamins and iron in the recommended daily dosages. THERE IS NO VALUE IN OVERPRESCRIBING SUPPLEMENTS, AND IT MAY CAUSE HARM.



About 40% of children with ASD have Attention deficit and hyperactivity disorder (ADHD). If the ADHD has been diagnosed, using appropriate tools, and is causing significant functional problems for the child’s learning, socialising and day-to-day life, then consider methylphenidate (Davis & Kollins, 2012).


Lower doses (range 2.5–10mg or 0.14–0.58 mg/kg for both the morning and noon doses) may be effective and avoid side effects associated with higher doses.


Atypical antipsychotic drugs


A larger multicenter trial conducted by RUPP Autism Network reported that treatment of 101 ASD children and adolescents (5–17 years old) with risperidone (mean dose 1.8 mg/kg) for 8 weeks resulted in 57% reduction in irritability vs. 14% reduction in the placebo group.


Risperidone treatment also significantly improved aggression, social withdrawal, repetitive behaviours, and hyperactivity (McCracken et al., 2002).



Risperidone treatment was associated with significant weight gain as well as fatigue, drowsiness and dizziness.


Low doses of risperidone (0.5–1.5 mg/d) are effective in reducing irritability and agitation in very young ASD children (aged 2–9 years) (Luby et al., 2006; Posey, Walsh, Wilson, & McDougle, 1999).



Two double-blind RCTs evaluated the efficacy and safety of aripiprazole in ASD children. The first study included 218 autistic children and adolescents (aged 6–17 years) who were randomized to receive either one of three different doses of aripiprazole (5, 10 or 15 Mg/d) or placebo for 8 weeks. All three doses significantly reduced irritability

compared with placebo at week 8. The three most common side effects were sedation, drooling and tremor,  and this contributed to a higher mean of discontinuation rate of aripiprazole-treated patients (10.3%) vs. placebo (7.7%) (Marcus et al., 2009).


These trials led to FDA approval of aripiprazole as the second atypical antipsychotic drug to treat irritability in children and adolescents with autistic disorder aged 6–17 years (Blankenship, Erickson, Stigler, Posey, & McDougle, 2010).


Dose (2.5 mg a day for children age 6 years and older, 5 mg in age 17 years and older).



An 8-week double-blind RCT reported that olanzapine improved response rates in 11 young ASD patients (Hollander et al., 2006). Open-label treatment of 8 children, adolescents, and adults (age range, 5– 42 years)with olanzapine (mean dose 7.8 mg/d) significantly improved many symptoms including irritability, anger, anxiety, hyperactivity, social withdrawal, and language usage (Potenza, Holmes, Kanes, & McDougle, 1999).


In spite of the superior efficacy of olanzapine in managing symptoms of ASD, its clinical use has been limited due to its well-known metabolic side effects

including increased appetite, excessive weight gain and impaired insulin sensitivity (Tschoner et al., 2007).


Antidepressant drugs


Few trials have evaluated the efficacy and tolerability of sertraline in ASD patients. A small open-label clinical trial found that 2–8 weeks of treatment with sertraline (25–50 mg) significantly improved anxiety and irritability in 8 out of 9 pediatric ASD patients (Steingard, Zimnitzky, DeMaso, Bauman,& Bucci, 1997). A larger open-label trial reported that 12-week treatment with higher doses of sertraline (50–200 mg) reduced aggressive and repetitive behaviours in adult ASD patients (McDougle et al., 1998). The medication was well tolerated with minimal adverse events.


Alpha-2 adrenergic receptor agonists


The use of alpha-2 adrenergic receptor agonists is associated with the treatment of aggressive behaviour, sleep disturbances and anxiety, which are prominent symptoms in ASD patients. These medications inhibit norepinephrine neurotransmission in the brainstem, leading to a decrease in sympathetic outflow and peripheral resistance thereby diminishing states of hyper-arousal, anxiety, and/or motor spasms (Newcorn et al., 1998).

A third open-labelled retrospective clinical trial found that clonidine was  effective in reducing sleep initiation latency and night awakening, and to a

lesser degree, in also improving hyperactivity and aggressiveness in ASD children, with a fairly benign tolerability profile (Ming, Gordon, Kang, & Wagner, 2008).


Dose: 0.1 mg once at bedtime for children age 6years and older.


Administration of clonidine may be accompanied by drowsiness, dry mouth, bradycardia and orthostatic hypotension. Abrupt withdrawal of the drug could lead to rebound hypertension resulting in a hypertensive crisis. 


2 to 3 mg at bedtime to improve sleep initiation. It is relatively free of side effects, except for some mood changes.