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Background: Rett syndrome (RS) is a severe neurodevelopmental disorder for which there is no approved therapy. This study aimed to assess safety and efficacy of oral fingolimod in children with RS using a pre-post and case-control design.Methods: At the University of Basel Children's Hospital, Basel, Switzerland, children with RS were included if they were older than 6 years and met the established diagnostic criteria of RS, including a positive MeCP2 mutation. Participants were observed 6 months before and after treatment and received 12 months of fingolimod treatment. Serum samples of 50 children without RS served as reference for brain-derived neurotrophic factor (BDNF) measurements. Primary outcome measures were safety and efficacy, the latter measured by change in levels of BDNF in serum/CSF (cerebrospinal fluid) and change in deep gray matter volumes measured by magnetic resonance imaging (MRI). Secondary outcome measure was efficacy measured by change in clinical scores [Vineland Adaptive Behaviour Scale (VABS), Rett Severity Scale (RSSS) and Hand Apraxia Scale (HAS)].Results: Six children with RS (all girls, mean and SD age 11.3 ± 3.1 years) were included. Serum samples of 50 children without RS (25 females, mean and SD age 13.5 ± 3.9 years) served as reference for BDNF measurements. No serious adverse events occurred. Primary and secondary outcome measures were not met. CSF BDNF levels were associated with all clinical scores: RSSS (estimate - 0.04, mult.effect 0.96, CI [0.94; 0.98], p = 0.03), HAS (estimate - 0.09, mult.effect 0.91, CI [0.89; 0.94], p <  0.01) and VABS (communication: estimate 0.03, mult.effect 1.03, CI [1.02; 1.04], p < 0.01/daily living: estimate 0.03, mult.effect 1.03, CI [1.02; 1.04], p < 0.01/social skills: estimate 0.07, mult.effect 1.08, CI [1.05; 1.11], p < 0.01/motoric skills: estimate 0.04, mult.effect 1.04, CI [1.03; 1.06], p = 0.02).Conclusions: In children with RS, treatment with fingolimod was safe. The study did not provide supportive evidence for an effect of fingolimod on clinical, laboratory, and imaging measures. CSF BDNF levels were associated with clinical scores, indicating a need to further evaluate its potential as a biomarker for RS. This finding should be further validated in independent patient groups.Trial Registration: Clinical Trials.gov NCT02061137, registered on August 27th 2013, https://clinicaltrials.gov/ct2/show/study/NCT02061137 . [ABSTRACT FROM AUTHOR]

Background: The Swiss label of oral fingolimod (0.5 mg once daily) requires a 6-hour first dose observation (FDO) including an ECG prior to and 6 hours after the first intake but in comparison to other countries such as Austria, Australia and Canada there are no restrictions regarding the clinical settings of the FDO procedure in Switzerland. We present here our real-world experience of the 6 hour FDO procedure in three different clinical settings, following fingolimod treatment initiation. This is the first report on the FDO of fingolimod in these real-world clinical settings in Swiss patients with multiple sclerosis (MS). Methods: This was a retrospective, multi-clinic, observational study of 136 patients with relapsing-remitting multiple sclerosis. Summary statistics have been used to present the data. Results: Only two patients (<1.5% [2/136]) experienced symptoms after the first dose of fingolimod. Atrioventricular conduction abnormalities were reported in 3% (4/136) of patients, which resolved spontaneously within 24 hours of treatment initiation. During the average 6.8 months follow-up, 96% (131/136) of the patients remained on therapy. Conclusions: These findings support the safety and feasibility of FDO and tolerability of fingolimod in real-world clinical settings. [ABSTRACT FROM AUTHOR]

Background: Fingolimod is an oral multiple sclerosis drug that is considered a specialty drug due to its high cost and safety issues. The Fingolimod Patient Support Program (F-PSP) is a specialty pharmacy service developed to ensure the responsible use of fingolimod by promoting patient safety and medication adherence. This study aims to explore the satisfaction, experiences and perceptions regarding the F-PSP among patients currently involved in this program or recently withdrawn., Methods: A qualitative study was conducted via individual, face-to-face semistructured interviews with patients involved in the F-PSP. The interviews were audio-recorded, transcribed verbatim, coded and analyzed via thematic content analysis., Results: The main themes identified from the interviews (n = 17) were overall perception of the F-PSP, perception of the pharmacist-led consultations, perception of the tools (electronic monitor and drug intake graph), reasons to participate or potentially withdraw, and suggestions for improvements. Participants perceived the F-PSP as a reassuring support that complemented their medical care, providing a more human, personalized and person-centered approach than usual pharmacy care. Pharmacist-led consultations were valued for the medication-related and holistic support they provided. The importance of the pharmacist's attitude was emphasized. The electronic monitor was valued for promoting daily medication adherence and allowing the involvement of relatives, which reassured participants and their relatives. The participants appreciated the drug intake graph because it provided an objective overview of medication adherence, thereby reassuring, rewarding, and motivating them. The main reason to join the program was to be supported, especially with respect to medication adherence., Conclusions: Participants were satisfied with the F-PSP, each for different reasons. Their feedback enabled the identification of measures for the optimization of the F-PSP and should facilitate its dissemination and transfer to other drugs/diseases/populations. Essential elements of generic pharmacist-led patient support programs considered valuable from the patients' perspective were identified.

Background and Purpose: In 2011, fingolimod was approved in Switzerland for the treatment of relapsing-remitting multiple sclerosis (RRMS). The aim of the present study was to assess the effectiveness and retention of fingolimod in a real-life Swiss setting, in which patients can receive fingolimod as both first- and second-line treatment for RRMS., Methods: This cross-sectional, observational study with retrospective data collection was performed at 19 sites that comprised both hospitals and office-based physicians across Switzerland. Sites were asked to document eligible patients in consecutive chronological order to avoid selection bias. Demographic and clinical data from 274 consenting adult patients with RRMS who had received treatment with fingolimod were analyzed., Results: Mean treatment duration with fingolimod was 32 months. Under fingolimod, 77.7% of patients remained free from relapses and 90.3% did not experience disability progression. The proportion of patients who were free from any clinical disease activity, i.e. without relapses and disability progression, was 72.1%. A total of 28.5% of patients had been RRMS treatment-naïve prior to fingolimod therapy. High long-term treatment retention rates ranging between 95.7% at 24 months and 87.8% at 36 months were observed., Conclusion: In this Swiss cohort of naïve and pre-treated subjects with RRMS, the majority of patients under fingolimod treatment showed freedom from relapses and disability progression. In addition, treatment retention rate over 2 and 3 years was high, irrespective of previous treatment., (© 2018 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.)