Psychotropic drug use in intellectual disability and low-functioning autism spectrum disorder: Who, What, When, How, and Why

Friday, 24th September 2021 Meeth the Expert Marco Bertelli 09:00am - 09:45am Room: Humboldt

Intellectual developmental disorder (IDD) and low functioning autism spectrum disorder (LF-ASD) include a wide range of lifelong neurobiological conditions with different clinical features and vulnerabilities, which imply specific psychopharmacological strategies. In the last years many studies are increasingly targeting specific genetic syndromes, such as Fragile X syndrome, Prader-Willi syndrome, Rett syndrome, tuberous sclerosis, and Phelan-McDermid syndrome. Many other aspects, such as co-occurring physical and mental health issues, problem behaviors, or quality of life further enlarge the need of treatment differentiation up to the individual level, entailing tailored psycho-pharmacy and participation of the multi-professional social and health care team.

The correctness and precision of psychiatric diagnoses is a fundamental prerequisite for an effective therapy, especially when targeted at behavioral symptoms, such as irritability, aggression, self-injury, or temper tantrums. If challenging behaviors have not been proven to be symptoms of a psychiatric disorder, their management through psychotropic drugs should be considered only after other non-pharmacological interventions have failed. Drugs should be used at the lowest possible dose and for the minimum duration, with non-medication-based management strategies and the withdrawal of medication being considered at regular intervals.

The most commonly prescribed psychoactive drugs are antipsychotics, especially second-generation antipsychotics (SGA), followed by antidepressants, antiepileptics/mood stabilisers and stimulants. Evidence on efficacy, dosage and safety on adults is scarce and derived mostly from naturalistic studies or case reports, with a main focus on identification of side effects and discontinuation rate. Placebo-controlled or active-controlled studies are limited, with small sample size. Some SGA have been shown to be useful on some behavioural symptoms. Specifically, risperidone and aripiprazole are increasingly reported to be effective on irritability marked by aggression, self-injury, and severe tantrums. More limited data are available for other SGA who are frequently used in daily practice, including clozapine.

Attention deficit and hyperactivity disorder medications may be effective for counteracting the additional features of hyperactivity and short attention span. Antiepileptics, mood stabilizers and selective serotonin reuptake inhibitors have shown promising results, but evidence seems to be even lower and indications even less precise than for the other classes. Some newer compounds, such as asenapine, cariprazine and vortioxetine, have shown few side effects and present a receptor binding profile which is suitable with the characteristics of a large part of this special population.

The usefulness of psychotropic drug use should be judged in terms of effectiveness rather than efficacy (on target symptoms) and safety, with the former including the capability to keep the patient on treatment for the right time and the impact on generic quality of life.

Mooney LN, Dominick KC, Erickson CA. (2019). Psychopharmacology of neurobehavioral disorders. Handb Clin Neurol., 165: 383-390.
National Institute for Health and Care Excellence. Mental health problems in people with learning disabilities: prevention, assessment and management. NICE guideline. 2016. Available from: (l.d. 4/26/2020).
Persico AM, Ricciardello A, Cucinotta F. (2019). The psychopharmacology of autism spectrum disorder and Rett syndrome. Handb Clin Neurol., 165: 391-414.
Royal College of Psychiatrists, Faculty of psychiatry of intellectual disability. Psychotropic drug prescribing for people with intellectual disability, mental health problems and/or behaviours that challenge: practice guidelines. 2016. Available from: HYPERLINK ""

Simple and to the point:

  • In cases of intellectual disability and low-functioning autism, a tailored therapy with psychotropic drugs can be useful
  • Valid diagnostics and the involvement of the multiprofessional social and health care system are the basis for effective pharmacological treatment
  • Medication of challenging behaviour should only be used when non-pharmacological strategies fail, and at the lowest possible dosage for the shortest possible duration
  • The most commonly prescribed psychoactive medications are second-generation antipsychotics and antidepressants
  • Data on the efficacy, dosage and safety of antidepressants and mood stabilisers in people with intellectual disability is sparse; the use of other groups of medication will be discussed
  • In psychopharmacological treatment, it is generally important to consider the necessary duration of treatment and the effects on quality of life