Efficacy of oral pharmacological treatments in dyskinetic cerebral palsy: a systematic review

Abstract

Evidence to prove (or disprove) the efficacy of oral drugs in dyskinetic cerebral palsy is low. The most investigated drugs, trihexyphenidyl and levodopa, show contradictory results. Tetrabenazine, levetiracetam, and gabapentin efficacy should be studied in more detail. Lack of evidence is partially due to the inconsistency of classifications and outcome measures used. Outcome measures should be selected within the framework of the International Classification of Functioning, Disability and Health in next clinical trials.

Current therapies and therapeutic decision making for childhood‐onset movement disorders

“…Complete dopa responsiveness is suggestive of a primary neurotransmitter condition and sometimes other monogenic disorders such as PARKIN deletions; however, varying dopamine responsiveness can be observed in some other genetic nonprogressive disorders (DNAJC12, DYT11, paroxyxmal kinesigenic and nonkinesigenic dyskinesia, and pyruvate dehydrogenase complex deficiency), genetic‐progressive disorders (ataxia telangiectasia, chorea acanthocytosis, DJ‐1, FBX07, Kufor‐Rakeb disease, nuclear inclusion body disease, PINK1, pantothenate kinase associated neurodegeneration, PLA2G6 associated neurodegeneration, pontocerebellar hypoplasia type 2, SCA2, SCA3, SOX6, SPG11, and xeroderma pigmentosum), and, to a lesser extent, in acquired disorders such as movement disorders with lupus, organophosphate poisoning, subacute sclerosing panencephalitis, postencephalitic dystonia, or extrapontine myelinolysis. The role of l ‐dopa in management of dystonic cerebral palsy (CP) is questionable and results from previous studies are contradictory …”

“…In some genetic dystonias, such as DYT1 (and personal experience) or DYT11, trihexiphenidyl can be highly effective. Adverse events are common and include anticholinergic side effects such as constipation, ileus, dry mouth, blurred vision, urinary retention, and cardiovascular alterations . In addition, anticholinergics can cause confusion, depression, personality change, and cognitive issues …”

“…The role of L-dopa in management of dystonic cerebral palsy (CP) is questionable and results from previous studies are contradictory. 87 Given the chance of variable benefits in some cases with different underlying disorders, an L-dopa trial is still commonly practised. Though relatively safe, families need to be counseled about commonly reported adverse events with dopamine (nausea, vomiting, and sometimes headache, constipation, somnolence, or insomnia).…”

“…Adverse events are common and include anticholinergic side effects such as constipation, ileus, dry mouth, blurred vision, urinary retention, and cardiovascular alterations. 87,108 In addition, anticholinergics can cause confusion, depression, personality change, and cognitive issues. 106,114 Benztropine seems to find more favor in psychiatric settings compared to trihexiphenidyl for treatment of drug-related dystonia.…”

Medical treatment of dyskinetic cerebral palsy: translation into practice

“…The systematic review by Masson et al demonstrates that the current evidence does not strongly suggest efficacy of any oral medications in dyskinetic CP . It is important not to misinterpret this result as a lack of efficacy.…”