Baclofen as an adjuvant therapy for autism: a randomized, double-blind, placebo-controlled trial

Mahdavinasab, Seyedeh-Mahsa; Saghazadeh, Amene; Motamed-Gorji, Nogol; Vaseghi, Salar; Mohammadi, Mohammad-Reza; Alichani, Rosa; Akhondzadeh, Shahin

doi:10.1007/s00787-019-01333-5

Cited by 26 publications

(12 citation statements)

References 53 publications

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“…A randomized, placebocontrolled, phase 2 study of 150 participants with ASD showed there was no difference in the primary outcome measure between the arbaclofen group and control group, while there was improvement on the clinician-rated clinical global impression of severity in a specified secondary analysis (Veenstra-VanderWeele et al, 2017). Baclofen as an adjuvant therapy can enhance the therapeutical effect of antipsychotic drugs, risperidone, in children with ASD, including improvement of irritability, lethargy, stereotypic behavior, hyperactivity, and inappropriate speech (Mahdavinasab et al, 2019). A study with a small sample found that treatment with bumetanide decreased the autistic behaviors in autistic infants (Lemonnier and Ben-Ari, 2010).…”

Section: Potential Therapeutic Efficacy Of Gaba R Modulators In Asdmentioning

confidence: 99%

GABAergic System Dysfunction in Autism Spectrum Disorders

Zhao

Mao

Zhu

et al. 2022

Front. Cell Dev. Biol.

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Autism spectrum disorder (ASD) refers to a series of neurodevelopmental diseases characterized by two hallmark symptoms, social communication deficits and repetitive behaviors. Gamma-aminobutyric acid (GABA) is one of the most important inhibitory neurotransmitters in the central nervous system (CNS). GABAergic inhibitory neurotransmission is critical for the regulation of brain rhythm and spontaneous neuronal activities during neurodevelopment. Genetic evidence has identified some variations of genes associated with the GABA system, indicating an abnormal excitatory/inhibitory (E/I) neurotransmission ratio implicated in the pathogenesis of ASD. However, the specific molecular mechanism by which GABA and GABAergic synaptic transmission affect ASD remains unclear. Transgenic technology enables translating genetic variations into rodent models to further investigate the structural and functional synaptic dysregulation related to ASD. In this review, we summarized evidence from human neuroimaging, postmortem, and genetic and pharmacological studies, and put emphasis on the GABAergic synaptic dysregulation and consequent E/I imbalance. We attempt to illuminate the pathophysiological role of structural and functional synaptic dysregulation in ASD and provide insights for future investigation.

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Section: Potential Therapeutic Efficacy Of Gaba R Modulators In Asdmentioning

confidence: 99%

GABAergic System Dysfunction in Autism Spectrum Disorders

Zhao

Mao

Zhu

et al. 2022

Front. Cell Dev. Biol.

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“…[32][33][34] Other off-label uses. There are case reports or small studies describing the off-label use of baclofen for the treatment of several clinical entities, including muscle spasm/musculoskeletal pain, 2 persistent/chronic hiccups, [35][36][37] autism spectrum disorders, 38 chronic post-traumatic stress disorder (PTSD), 39 narcolepsy, 40 persistent speech stuttering, 41 post-hemorrhoidectomy pain, 42 trigeminal neuralgia, 43 and low back pain. 44…”

Section: Off-label Usesmentioning

confidence: 99%

Baclofen therapeutics, toxicity, and withdrawal: A narrative review

et al. 2021

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Baclofen is an effective therapeutic for the treatment of spasticity related to multiple sclerosis, spinal cord injuries, and other spinal cord pathologies. It has been increasingly used off-label for the management of several disorders, including musculoskeletal pain, gastroesophageal reflux disease, and alcohol use disorder. Baclofen therapy is associated with potential complications, including life-threatening toxicity and withdrawal syndrome. These disorders require prompt recognition and a high index of suspicion. While these complications can develop following administration of either oral or intrathecal baclofen, the risk is greater with the intrathecal route. The management of baclofen toxicity is largely supportive while baclofen withdrawal syndrome is most effectively treated with re-initiation or supplementation of baclofen dosing. Administration of other pharmacologic adjuncts may be required to effectively treat associated withdrawal symptoms. This narrative review provides an overview of the historical and emerging uses of baclofen, offers practical dosing recommendations for both oral and intrathecal routes of administration, and reviews the diagnosis and management of both baclofen toxicity and withdrawal.

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“…McCracken et al (2002) reported a placebo controlled, double-blind study of the use of risperidone in the treatment of 101 children (82 boys and 19 girls) aged 5 to 17 years, having autism disorder associated with severe tantrums, aggression, or self-injurious behavior. The study found that risperidone was beneficial for the treatment of tantrums, aggression, or selfinjurious behavior in children, and was well tolerated [26].…”

Section: Discussionmentioning

confidence: 98%