BackgroundEvidence on the use of fingolimod in real-world clinical practice and data on patient-reported health-related quality of life (HRQoL) in countries such as the Middle East are sparse. The Prospective Evaluation of Treatment with Fingolimod for Multiple Sclerosis (PERFORMS) study assessed HRQoL and effectiveness and safety of fingolimod in patients with relapsing-remitting multiples sclerosis (RRMS), primarily in Middle Eastern countries.MethodsThis 12-month, observational, multicentre, prospective, real-world study was conducted in patients with RRMS who initiated fingolimod or another approved disease-modifying treatment (DMT) within 4 weeks before study entry. Patients were enrolled in a 2:1 ratio to obtain more data in fingolimod and parallel in other DMTs cohort by physicians during routine medical care. Key study outcomes included HRQoL assessed using MS International QoL (MusiQoL), MS relapses and disability. Safety was assessed throughout the study period. Due to the observational nature of the study, no neuroimaging assessments were mandated and central reading was not performed.ResultsOf 249 enrolled patients, 247 were included in the analysis (fingolimod cohort 172; other DMTs cohort 75). Overall, the mean age of patients was 36.5 years, 64.4% were women and ~90% were Caucasians. At baseline, mean MS duration since diagnosis was 7.2 years in the fingolimod and 4.8 years in the other DMTs cohorts. Overall, mean changes in MusiQoL index scores were −2.1 in the fingolimod cohort and −0.7 in the other DMTs cohort at Month 12, but improvement was not significant vs. baseline in both cohorts. Proportion of relapse-free patients increased significantly during the study vs. 0–12 months before the study in the fingolimod cohort (80.2% vs. 24.4%; p < 0.0001). Proportion of patients free from disability progression was 86.5% in the fingolimod cohort. The incidences of AEs were 59.9% and 50.6% in the fingolimod and other DMTs cohorts, respectively. First-dose monitoring of fingolimod observed no cases of symptomatic bradyarrhythmia. Three cases of bradycardia were reported in the fingolimod cohort: one after the first dose and two during the study. No cases of macular oedema were observed during the study.ConclusionsFingolimod treatment maintained QoL over 12 months and was effective in reducing relapse rate and disability progression. No new safety findings were observed in this real-world observational study in Middle Eastern countries.Electronic supplementary materialThe online version of this article (doi:10.1186/s12883-017-0913-3) contains supplementary material, which is available to authorized users.

Introduction Eptinezumab is a humanized IgG1 immunoglobulin monoclonal antibody administered intravenously as a preventative migraine treatment. Previously conducted randomized, double-blind, placebo-controlled trials exhibited significant reductions in monthly migraine frequency among adults experiencing episodic and chronic migraine. The present study seeks to expand upon the current findings and to evaluate eptinezumab’s efficacy as a preventative treatment for chronic and episodic migraine patients in the United Arab Emirates. This study is intended to represent the first real-world evidence and will hopefully serve as a valuable complement to the existing literature on the subject. Methods This was a retrospective exploratory study. The participants included within the study were adult (≥ 18 years) patients diagnosed with either episodic or chronic migraine. Patients were categorized according to their history of previous preventative treatment failure. For the final assessment of treatment efficacy, we included only patients with a minimum of 6 months of clinical follow-up data. Patients were assessed at baseline for their monthly migraine frequency and assessed again at months 3 and 6. The primary objective was to evaluate the efficacy of eptinezumab in reducing migraine frequency among chronic and episodic migraine patients. Results A total of 100 participants were identified, of whom 53 completed the study protocol at month 6. Of the total, 40 (75.47%) were female, 46 (86.79%) were Emirati locals, and 16 (30.19%) were pharmaceutically naïve, having never tried any prior preventative therapy. Additionally, 25 (47.17%) patients met the criteria for chronic migraine (CM), whereas the remaining 28 (52.83%) were diagnosed with episodic migraine (EM). The baseline monthly migraine frequency (MMD) was 12.23 (4.97) days across all participants, 15.56 (3.97) for CM patients, and 9.25 (3.76) for EM patients; by month 6, these frequencies reduced to 3.66 (4.21), 4.76 (5.32), and 2.68 (2.61), respectively. Overall, 58.49% of those enrolled experienced > 75% reduction in MMD frequency by month 6. Conclusion Patients enrolled in this trial experienced clinically significant reductions in MMD by month 6. Eptinezumab was well tolerated and with one AE of significance that led to discontinuation from the study.

Background Eptinezumab-jjmr is a humanized IgG1 immunoglobulin monoclonal antibody administered intravenously as a preventative migraine treatment. Previously conducted randomized, double-blind, placebo-controlled trials exhibited significant reduction in monthly migraine frequency among adults experiencing episodic and chronic migraine. The present study seeks to expand upon the current findings and evaluate Eptinezumab-jjmr’s efficacy as a preventative migraine treatment for chronic and episodic migraine patients in the United Arab Emirates. This study is intended to represent the first real-world evidence study and will hopefully serve as a valuable complement to the existing literature on the subject.Methods This was a retrospective, observational, and exploratory study. The participants included within the study were adult (≥ 18 years) patients diagnosed with either episodic or chronic migraine. Patients were categorized according to their history of previous preventative treatment failure. For the final assessment of treatment efficacy, we included only patients with a minimum of 6 months of clinical follow-up data. Patients were assessed at baseline for their monthly migraine frequency and assessed again at months 3 and 6. The primary objective was to evaluate the efficacy of Eptinezumab-jjmr in reducing migraine frequency among chronic and episodic migraine patients.Results 100 participants were identified, of whom 53 patients completed the study protocol to month 6. 40 (75.5%) were female, 46 (86.8%) were Emirati locals, and 16 (30.2%) were pharmaceutically naïve, having never tried any prior preventative therapy. Additionally, 25 (47.2%) patients met the criteria for chronic migraine (CM), whereas the remaining 28 (52.8%) were diagnosed with episodic migraine (EM). The baseline monthly migraine frequency (MMD) was 12.2 (4.97) days across all participants, 15.6 (3.97) for CM patients, and 9.25 (3.76) for EM patients; by month 6, these frequencies reduced to 3.66 (4.21), 4.76 (5.32), and 2.68 (2.61), respectively. Overall, 58.5% of those enrolled experienced > 75% reduction in MMD frequency by month 6.Conclusion Patients enrolled in this trial experienced clinically significant reductions in MMD by month 6. Eptinezumab-jjmr was well tolerated with only one AE of note.

The authors present a case of refractory multiple myeloma in an elderly patient with heterozygosity for the G20210A mutation of factor II and for the C677T mutation of methylenetetrahydrofolate reductase. This patient developed acute, multiple cerebral infarctions 5 months into the treatment with thalidomide. The patient improved on oral anticoagulation. The authors presume that thalidomide increased the risk of cerebral infarction in this predisposed patient. The presence of increased risk for thrombosis should alert clinicians to screen patients presenting with systemic thrombosis for inherited thrombophilia. This risk increases with the presence of diseases and therapies that predispose for a hypercoagulable state. They suggest that patients required to be on thalidomide should be screened for inherited thrombophilia and should be considered for prophylactic anticoagulation therapy.

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