Clinical presentation and management of dyskinetic cerebral palsy

Monbaliu, Elegast; Himmelmann, Kate; Lin, Jean-Pierre; Ortibus, Els; Bonouvrié, Laura A.; Feys, Hilde; Vermeulen, R. Jeroen; Dan, Bernard

doi:10.1016/s1474-4422(17)30252-1

Cited by 173 publications

(206 citation statements)

References 99 publications

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“…2,4 It is characterized by involuntary, uncontrolled, recurring, occasionally stereotyped movements with fluctuating muscle tone. 6 Dystonia and choreoathetosis are frequently present simultaneously in dyskinetic CP, but dystonia is usually more prominent. Dystonia is characterized by slow involuntary movements, distorted voluntary movements, and abnormal postures due to sustained or intermittent muscle contractions.…”

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confidence: 99%

“…5 Dyskinesia is subdivided into dystonia and choreoathetosis. 6 Most treatment options for dyskinetic CP are aimed at decreasing dystonia. Tone is fluctuating but easily increased (hypertonia).…”

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confidence: 99%

“…Dystonia is characterized by slow involuntary movements, distorted voluntary movements, and abnormal postures due to sustained or intermittent muscle contractions. 6 When efficacy of oral medication is insufficient, options requiring neurosurgical intervention such as intrathecal baclofen (ITB) treatment or deep brain stimulation (DBS) are the next step in treatment of dyskinetic CP. 5 Choreoathetosis is featured by fast hyperkinetic movements and tone fluctuation (mainly hypotonia).…”

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confidence: 99%

“…Tone is fluctuating but easily increased (hypertonia). 6,7 DBS is effective in primary dystonia, but results in dyskinetic CP have been conflicting. 5 The majority of dyskinetic CP patients are severely affected and classified in Gross Motor Functioning Classification System (GMFCS) levels IV and V. These GMFCS levels correspond with having no walking ability.…”

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confidence: 99%

“…5 Choreoathetosis is featured by fast hyperkinetic movements and tone fluctuation (mainly hypotonia). 6,7 Although there is some evidence for the effectiveness of ITB in reducing spasticity in CP, mainly from short-term single-bolus trial studies, to date there are only low-quality, noncontrolled studies, producing low-level evidence for ITB in the reduction of dystonia. 6 Dystonia and choreoathetosis are frequently present simultaneously in dyskinetic CP, but dystonia is usually more prominent.…”

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confidence: 99%

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The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial

Bonouvrié

Becher²,

Vles

et al. 2019

Annals of Neurology

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Objective Intrathecal baclofen treatment is used for the treatment of dystonia in patients with severe dyskinetic cerebral palsy; however, the current level of evidence for the effect is low. The primary aim of this study was to provide evidence for the effect of intrathecal baclofen treatment on individual goals in patients with severe dyskinetic cerebral palsy. Methods This multicenter, randomized, double‐blind, placebo‐controlled trial was performed at 2 university medical centers in the Netherlands. Patients with severe dyskinetic cerebral palsy (Gross Motor Functioning Classification System level IV–V) aged 4 to 24 years who were eligible for intrathecal baclofen were included. Patients were assigned by block randomization (2:2) for treatment with intrathecal baclofen or placebo for 3 months via an implanted microinfusion pump. The primary outcome was goal attainment scaling of individual treatment goals (GAS T score). A linear regression model was used for statistical analysis with study site as a covariate. Safety analyses were done for number and type of (serious) adverse events. Results Thirty‐six patients were recruited from January 1, 2013, to March 31, 2018. Data for final analysis were available for 17 patients in the intrathecal baclofen group and 16 in the placebo group. Mean (standard deviation) GAS T score at 3 months was 38.9 (13.2) for intrathecal baclofen and 21.0 (4.6) for placebo (regression coefficient = 17.8, 95% confidence interval = 10.4‐25.0, p < 0.001). Number and types of (serious) adverse events were similar between groups. Interpretation Intrathecal baclofen treatment is superior to placebo in achieving treatment goals in patients with severe dyskinetic cerebral palsy. ANN NEUROL 2019

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confidence: 99%

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confidence: 99%

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The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial

Bonouvrié

Becher²,

Vles

et al. 2019

Annals of Neurology

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Early prediction of adverse outcomes in infants with acute bilirubin encephalopathy

Kang

Xiao

Zhang

et al. 2020

Ann Clin Transl Neurol

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Objective: Acute bilirubin encephalopathy (ABE) remains one of the important causes of neonatal mortality and child disability, early identification, and intervention which could improve outcomes. The purpose of this study was to evaluate early predictors of adverse outcomes in infants with ABE. Methods: Newborns of gestational age ≥ 35 weeks and diagnosed with ABE were included in the study. Bilirubin-induced neurological dysfunction (BIND) score, total serum bilirubin (TSB) peak value, and serum albumin levels were determined. Adverse outcomes were defined as death or survival with auditory dysfunction and/or cerebral palsy. Results: Eighty-two infants were eligible for recruitment in the study. The outcome data from 76 ABE infants (92%) were used for analysis, of which 25 infants got adverse outcomes and 51 live a normal life. Univariate analysis for BIND score, TSB peak value, bilirubin-albumin ratio (B/A), albumin level, abnormal AABR, and neonatal sepsis was performed to elucidate the association with adverse outcomes. Bivariate logistic regression analysis showed B/A (OR 10.48, 95%CI: 1.55-70.81, P = 0.02) and BIND score (OR 3.68, 95%CI: 1.39-9.72, P = 0.01) were correlated with adverse outcomes. ROC curve analysis showed that B/A (≥8.9 mg/g), BIND score (≥6) could predict adverse outcomes of ABE separately; B/A in conjunction with BIND score could increase prediction sensitivity to 100%. Interpretation: Both B/A and BIND score can be used to predict adverse outcomes of ABE, and the combination of the two parameters can increase prediction sensitivity significantly.

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Consensus guidelines for the diagnosis and management of isolated sulfite oxidase deficiency and molybdenum cofactor deficiencies

Schwahn,

van Spronsen,

Misko

et al. 2024

J of Inher Metab Disea

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Sulfite intoxication is the hallmark of four ultrarare disorders that are caused by impaired sulfite oxidase activity due to genetic defects in the synthesis of the molybdenum cofactor or of the apoenzyme sulfite oxidase. Delays on the diagnosis of these disorders are common and have been caused by their unspecific presentation of acute neonatal encephalopathy with high early mortality, followed by the evolution of dystonic cerebral palsy and also by the lack of easily available and reliable diagnostic tests. There is significant variation in survival and in the quality of symptomatic management of affected children. One of the four disorders, molybdenum cofactor deficiency type A (MoCD‐A) has recently become amenable to causal treatment with synthetic cPMP (fosdenopterin). The evidence base for the rational use of cPMP is very limited. This prompted the formulation of these clinical guidelines to facilitate diagnosis and support the management of patients. The guidelines were developed by experts in diagnosis and treatment of sulfite intoxication disorders. It reflects expert consensus opinion and evidence from a systematic literature search.

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Clinical presentation and management of dyskinetic cerebral palsy

Cited by 173 publications

References 99 publications

The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial

The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial

Early prediction of adverse outcomes in infants with acute bilirubin encephalopathy

Consensus guidelines for the diagnosis and management of isolated sulfite oxidase deficiency and molybdenum cofactor deficiencies

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