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Aim: To gather data on mycophenolate mofetil (MMF) in paediatric autoimmune/immune-mediated central nervous system (CNS) conditions, focusing on safety and factors that may affect MMF efficacy.Method: Retrospective, multicentre study based on four paediatric neurology centres.Results: Forty-four children were included (30 females, 14 males): 19 had proven/suspected autoimmune encephalitis, 14 had inflammatory demyelinating CNS diseases, and 11 had other autoimmune/immune-mediated CNS conditions. Before MMF, all received first-line immune therapies, and 17 had second-line rituximab and/or cyclophosphamide. MMF was started at a median of 9.5 months from disease onset (range 1-127mo) (median age 9y 4mo, range 1y 5mo-16y 5mo), and was used for median 18 months (range 0.3-73mo). On MMF, 31 patients were relapse-free, whereas eight relapsed (excluding patients with chronic-progressive course). Relapses on MMF were associated with medication weaning/cessation, or with suboptimal MMF dosage/duration. Adverse events of MMF occurred in eight patients: six moderate (gastrointestinal, movement disorder, dermatological) and two severe (infectious).Interpretation: MMF use in paediatric neuroimmunology is heterogeneous, although relatively safe. We have identified factors that may affect MMF efficacy and provide recommendations on MMF usage.What This Paper Adds: Mycophenolate mofetil (MMF) use was heterogeneous with relatively common adverse events, although mostly not severe. MMF treatment reduced median annualized relapse rate, although 20% of patients relapsed on MMF. A high relapse rate pre-MMF and late MMF start were associated with higher probability of relapsing on MMF. Most relapses were associated with suboptimal MMF dosage, short MMF duration, or concurrent medication weaning/discontinuation. [ABSTRACT FROM AUTHOR]

Aim: To assess long-term safety and efficacy of stiripentol as an antiepileptic medication for people with Dravet syndrome.Method: A prospective, observational open-label study (2003-2015) of the efficacy and long-term safety of stiripentol in patients with Dravet syndrome and ongoing seizures. Frequency of generalized tonic-clonic seizures, focal seizures, status epilepticus, and adverse events were recorded.Results: Forty-one patients started stiripentol, with median age at enrolment 5 years 7 months (range 11mo-22y) and median duration of treatment 37 months (range 2-141mo). Twenty out of 41 patients had greater than or equal to 50% long-term reduction in frequency of generalized tonic-clonic seizures. Frequency of focal seizures was decreased by greater than or equal to 50% in 11 out of 23 patients over the long-term. Frequency of status epilepticus was decreased by 50% or more in 11 out of 26 patients. The most common adverse events were anorexia, weight loss, sedation, and behavioural changes. One patient had worsening of absence and myoclonic seizures. Another developed recurrent pancreatitis on concurrent valproate.Interpretation: Stiripentol improves long-term seizure frequency in approximately 50% of patients with Dravet syndrome, when used as part of unrestricted polytherapy. Long-term use appears safe. In more than 40% of patients, episodes of status epilepticus markedly decrease after stiripentol initiation. What this paper adds Frequency of status epilepticus is reduced in 40% of patients with Dravet syndrome after stiripentol initiation. Stiripentol is effective for generalized tonic-clonic and focal seizures. Stiripentol can be safely used with a range of antiepileptic drugs. [ABSTRACT FROM AUTHOR]

Aim: To systematically review evidence for pharmacological/neurosurgical interventions for managing dystonia in individuals with cerebral palsy (CP) to inform a care pathway.Method: Searches included studies with a minimum of five participants with dystonia in CP receiving oral baclofen, benzodiazepines (clonazepam, diazepam, lorazepam), clonidine, gabapentin, levodopa, trihexyphenidyl, botulinum toxin, intrathecal baclofen (ITB), or deep brain stimulation (DBS). Evidence was classified according to American Academy of Neurology guidelines.Results: Twenty-eight articles underwent data extraction: one levodopa, five trihexyphenidyl, three botulinum toxin, six ITB, and 13 DBS studies. No articles for oral baclofen, benzodiazepines, clonidine, or gabapentin met the inclusion criteria. Evidence for reducing dystonia was level C (possibly effective) for ITB and DBS; level C (possibly ineffective) for trihexyphenidyl; and level U (inadequate data) for botulinum toxin.Interpretation: For dystonia reduction, ITB and DBS are possibly effective, whereas trihexyphenidyl was possibly ineffective. There is insufficient evidence to support oral medications or botulinum toxin to reduce dystonia. There is insufficient evidence for pharmacological and neurosurgical interventions to improve motor function, decrease pain, and ease caregiving. The majority of the pharmacological and neurosurgical management of dystonia in CP is based on clinical expert opinion.What This Paper Adds: Intrathecal baclofen and deep brain stimulation are possibly effective in reducing dystonia. Current evidence does not support effectiveness of oral medications or botulinum toxin to reduce dystonia. Evidence is inadequate for pharmacological/neurosurgical interventions impact on improving motor function, pain/comfort, and easing caregiving. The majority of the care pathway rests on expert opinion. [ABSTRACT FROM AUTHOR]

Background Alcoholics Anonymous (AA) is a world-wide recovery mutual-help organization that continues to arouse controversy. In large part, concerns persist because of AA's ostensibly quasi-religious/spiritual orientation and emphasis. In 1990 the United States' Institute of Medicine called for more studies on AA's effectiveness and its mechanisms of behavior change (MOBC) stimulating a flurry of federally funded research. This paper reviews the religious/spiritual origins of AA and its program and contrasts its theory with findings from this latest research. Method Literature review, summary and synthesis of studies examining AA's MOBC. Results While AA's original main text ('the Big Book', 1939) purports that recovery is achieved through quasi-religious/spiritual means ('spiritual awakening'), findings from studies on MOBC suggest this may be true only for a minority of participants with high addiction severity. AA's beneficial effects seem to be carried predominantly by social, cognitive and affective mechanisms. These mechanisms are more aligned with the experiences reported by AA's own larger and more diverse membership as detailed in its later social, cognitive and behaviorally oriented publications (e.g. Living Sober, 1975) written when AA membership numbered more than a million men and women. Conclusions Alcoholics Anonymous appears to be an effective clinical and public health ally that aids addiction recovery through its ability to mobilize therapeutic mechanisms similar to those mobilized in formal treatment, but is able to do this for free over the long term in the communities in which people live. [ABSTRACT FROM AUTHOR]

The present paper reports on a 5-year follow-up of a randomized reading intervention in grade 3 in Sweden. An intervention group (n = 57) received daily training for 12 weeks in phoneme/grapheme mapping, reading comprehension and reading speed, whereas a control group (n = 55) participated in ordinary classroom activities. The main aim was to investigate if there were remaining effects of the intervention on reading-related skills. Previous analyses showed that the intervention group performed significantly better than the control group on spelling, reading speed, reading comprehension and phoneme awareness at the immediate post-test with sustained effects 1 year later. Results from the 5-year follow-up show that the only significant difference between the intervention (n = 47) and the control group (n = 37) was on word decoding. There was also a significant interaction effect of group assignment and initial word decoding, in the way that the lowest-performing students benefitted the most from the intervention. Another aim was to examine if the children identified in a screening (n = 2212) as poor readers in grade 2 still performed worse than typical readers. The analyses showed that the typically developing students (n = 66) outperformed the students identified as poor readers in grade 2 on working memory, spelling, reading comprehension and word decoding. Copyright © 2016 John Wiley & Sons, Ltd. [ABSTRACT FROM AUTHOR]