Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.
IntroductionOpsoclonus myoclonus syndrome (OMS) in childhood still remains unexplored from the perspective of clinical psychology.Objectives and aimsThe research was aimed at defining types of psychological development in the group of children with OMS. The study included 18 children between ages 2 years 4 months and 9 years with OMS (8 boys, 10 girls).MethodsThe following methods were used: analysis of patient's development and medical record, psychological interview with parents, neurological state assessment and pathopsychological assessment.ResultsPatients were divided into three groups according to cognitive development level:– group with normal intelligence level (n = 4);– group with developmental delay (n = 11);– group with intellectual disability in different forms (n = 3).Several children in group 1 (n = 3) and in group 2 (n = 4) expressed neurotic behavior with increased levels of anxiety and fear of trying unusual actions during psychological assessment. Children with delay in psychoverbal development (n = 7) and with developmental delay (n = 1) expressed psychopathy-like states with verbal aggression and had difficulties controlling their emotions. In some cases (n = 2), the psychological state of the patient was defined as borderline state with emotional instability, especially in mother–child interaction.ConclusionPsychological features of children with OMS indicate heterogeneity of their development types that implies different prognosis and developmental dynamics for each type. The results point out the necessity of detailed psychological examination aimed at correlation of psychological help to children with OMS and their families with their psychological state.Disclosure of interestThe authors have not supplied their declaration of competing interest.
Cortexin as add-on to AEDs reduced for more than two times the number of seizures, along with improvement of motor function, in 31 (36.9%) patients. The improvement of motor function, but without a significant decrease in epileptic seizures, was achieved in 15 (17.8%) of the patients. Reduction of epileptic seizures frequency (>2 times), but without a significant effect on motor function, was observed in 14 cases (16.7%). Twenty-three patients (27.4%) did not respond the therapy. The aggravation of epileptic seizures during cortexin therapy was observed in only 1 girl with West syndrome (1.2%), and this was significantly lower than the probability of seizures aggravation on AED. Polypeptide nootropic medication cortexin demonstrated efficacy and safety as adjunctive therapy in children with CP combined with epilepsy.
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