scholarly journals Long-term real-world effectiveness and safety of fingolimod over 5 years in Germany

2022 ◽  
Author(s):  
Tjalf Ziemssen ◽  
Michael Lang ◽  
Stephan Schmidt ◽  
Holger Albrecht ◽  
Luisa Klotz ◽  
...  
Keyword(s):  
Real World ◽  
Expanded Disability Status Scale ◽  
Exclusion Criteria ◽  
Clinical Routine ◽  
Safety Study ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Relapse Rates

Abstract Objective To evaluate the 5-year real-world benefit–risk profile of fingolimod in patients with relapsing–remitting MS (RRMS) in Germany. Methods Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA) is a non-interventional real-world study to prospectively assess the effectiveness and safety of fingolimod in routine clinical practice in Germany. The follow-up period comprised 5 years. Patients were included if they had been diagnosed with RRMS and had been prescribed fingolimod as part of clinical routine. There were no exclusion criteria except the contraindications for fingolimod as defined in the European label. The effectiveness and safety analysis set comprised 4032 and 4067 RRMS patients, respectively. Results At the time of the 5-year follow-up of PANGAEA, 66.57% of patients still continued fingolimod therapy. Annualized relapse rates decreased from baseline 1.5 ± 1.15 to 0.42 ± 0.734 at year 1 and 0.21 ± 0.483 at year 5, and the disability status remained stable, as demonstrated by the Expanded Disability Status Scale mean change from baseline (0.1 ± 2.51), the decrease of the Multiple Sclerosis Severity Score from 5.1 ± 2.59 at baseline to 3.9 ± 2.31 at the 60-months follow-up, and the percentage of patients with ‘no change’ in the Clinical Global Impression scale at the 60-months follow-up (78.11%). Adverse events (AE) occurring in 75.04% of patients were in line with the known safety profile of fingolimod and were mostly non-serious AE (33.62%) and non-serious adverse drug reactions (50.59%; serious AE 4.98%; serious ADR 10.82%). Conclusions PANGAEA demonstrated the sustained beneficial effectiveness and safety of fingolimod in the long-term real-world treatment of patients with RRMS.

scholarly journals A Pilot Study of 24-h Motor Activity Patterns in Multiple Sclerosis: Pre-Planned Follow-Up at 2 Years

2021 ◽  
Vol 3 (3) ◽  
pp. 366-376
Author(s):  
Lorenzo Tonetti ◽  
Federico Camilli ◽  
Sara Giovagnoli ◽  
Vincenzo Natale ◽  
Alessandra Lugaresi
Keyword(s):  
Multiple Sclerosis ◽  
Motor Activity ◽  
Activity Pattern ◽  
Activity Patterns ◽  
Expanded Disability Status Scale ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Edss Score ◽  
Activity Prognosis

Early multiple sclerosis (MS) predictive markers of disease activity/prognosis have been proposed but are not universally accepted. Aim of this pilot prospective study is to verify whether a peculiar hyperactivity, observed at baseline (T0) in early relapsing-remitting (RR) MS patients, could represent a further prognostic marker. Here we report results collected at T0 and at a 24-month follow-up (T1). Eighteen RRMS patients (11 females, median Expanded Disability Status Scale-EDSS score 1.25, range EDSS score 0–2) were monitored at T0 (mean age 32.33 ± 7.51) and T1 (median EDSS score 1.5, range EDSS score 0–2.5). Patients were grouped into two groups: responders (R, 14 patients) and non-responders (NR, 4 patients) to treatment at T1. Each patient wore an actigraph for one week to record the 24-h motor activity pattern. At T0, NR presented significantly lower motor activity than R between around 9:00 and 13:00. At T1, NR were characterized by significantly lower motor activity than R between around 12:00 and 17:00. Overall, these data suggest that through the 24-h motor activity pattern, we can fairly segregate at T0 patients who will show a therapeutic failure, possibly related to a more active disease, at T1. These patients are characterized by a reduced morning level of motor activation. Further studies on larger populations are needed to confirm these preliminary findings.

Pregnancy in women with MS: Impact on long-term disability accrual in a nationwide Danish Cohort

2021 ◽  
pp. 135245852110577
Author(s):  
Johanna Balslev Andersen ◽  
Malthe Faurschou Wandall-Holm ◽  
Per Kragh Andersen ◽  
Finn Sellebjerg ◽  
Melinda Magyari
Keyword(s):  
Multiple Sclerosis ◽  
Clinically Isolated Syndrome ◽  
Time Dependent ◽  
Disease Course ◽  
Expanded Disability Status Scale ◽  
Cox Models ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Edss Score

Background: Pregnancy is considered to influence the disease course in women with multiple sclerosis (MS). Objective: The aim of this study was to investigate the effect of pregnancy on long-term disability accrual in women with MS. Methods: The Danish Multiple Sclerosis Registry (DMSR) was used to identify women diagnosed with clinically isolated syndrome or relapsing-remitting MS. Cox models with pregnancy as a time-dependent exposure and propensity score (PS) models were used to evaluate time to reach confirmed Expanded Disability Status Scale (EDSS) score of 4 and 6. Results: A total of 425 women became parous and 840 remained nulliparous. When including pregnancy as a time-dependent exposure, a non-significant association with time to reach EDSS 4 (hazard ratio (HR) 0.86, 95% confidence interval (CI) 0.61–1.20) and EDSS 6 (HR 0.70, 95% CI 0.40–1.20) was found. Correspondingly, the PS model showed no association with pregnancy on time to reach EDSS 4 (HR 0.85, 95% CI 0.56–1.28). Conclusion: This study concludes that pregnancy does not affect long-term disability accumulation.

Assessing long-term prognosis improvement as a consequence of treatment pattern changes in MS

2017 ◽  
Vol 23 (13) ◽  
pp. 1757-1761 ◽  
Author(s):  
Ruggero Capra ◽  
Cinzia Cordioli ◽  
Sarah Rasia ◽  
Fabio Gallo ◽  
Alessio Signori ◽  
...  
Keyword(s):  
Cox Model ◽  
New Drugs ◽  
Expanded Disability Status Scale ◽  
Disability Progression ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Long Term Prognosis ◽  
Long Term Impact ◽  
Median Interval

Objective: To assess whether the age at which multiple sclerosis (MS) patients reach Expanded Disability Status Scale (EDSS) milestones changed as long as new drugs for the treatment of MS became available. Methods: We evaluated the long-term impact of therapies on disability progression assessing whether there is a detectable delay in the age at which patients reached EDSS milestones in more recent years. We used data collected over more than 30 years in the Center of Brescia, Italy. We compared the age at EDSS = 6 among patients diagnosed with relapsing-remitting MS in different time periods, adjusting for age at diagnosis and median interval among EDSS visits, by a multivariate Cox model. Results: A total of 1324 MS patients were included. Patients diagnosed in more recent periods reached EDSS = 6 at an older age: the rate at which patients reached EDSS = 6 in those diagnosed in 1991–1995 was similar to those diagnosed in 1980–1990 (hazard ratio ( HR) = 1.09, p = 0.68) and to those diagnosed in 1996–2000 ( HR = 0.85, p = 0.44), it was reduced by 37% in patients diagnosed in 2001–2005 ( HR = 0.63, p = 0.05), by 46% in patients diagnosed in 2006–2010 ( HR = 0.54, p < 0.02). Conclusion: A clear modification of MS course is observed after 2000; among other causes, this can be associated to the changes in the treatment patterns experienced in those years.

scholarly journals Real-world persistence and benefit–risk profile of fingolimod over 36 months in Germany

2019 ◽  
Vol 6 (3) ◽  
pp. e548 ◽  
Author(s):  
Tjalf Ziemssen ◽  
Michael Lang ◽  
Björn Tackenberg ◽  
Stephan Schmidt ◽  
Holger Albrecht ◽  
...  
Keyword(s):  
Real World ◽  
Risk Profile ◽  
Disease Modifying Therapy ◽  
Effectiveness Analysis ◽  
Disability Status ◽  
Scale Scores ◽  
First Time ◽  
Previous Disease

ObjectiveTo assess the long-term real-world benefit–risk profile of fingolimod in patients with relapsing MS in Germany.MethodsThis analysis used data from the noninterventional real-world study, Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA), to assess prospectively the persistence, effectiveness, and safety of fingolimod over 36 months (±90 days) in Germany. For inclusion in the effectiveness analysis (n = 2,537), patients were required to have received fingolimod for the first time in PANGAEA, to have at least 12 months of data, and to have completed each 12-month follow-up period. For the safety analysis (n = 3,266), patients were additionally allowed to have received fingolimod before enrollment.ResultsAt baseline, 94.7% of patients in the effectiveness analysis had received a previous disease-modifying therapy. After 36 months, 70.4% of patients were still receiving fingolimod. Over this period, annualized relapse rates decreased to 0.265 (95% CI: 0.244–0.286) from 1.79 (95% CI: 1.75–1.83), and mean Expanded Disability Status Scale scores remained stable (mean change from baseline: +0.049 [95% CI: −0.015 to +0.114]). In total, 16% of patients had 6-month confirmed disability improvement, 12.5% had 6-month confirmed disability worsening, and 52.4% were free from relapses and 6-month confirmed disability worsening. Adverse events (AEs) and serious AEs were experienced by up to 23.4% and 3.9% of patients, respectively, during any of the 12-month follow-up periods. The frequency and nature of AEs were in line with previous findings.ConclusionsUsing systematically collected data from PANGAEA, this analysis demonstrates the sustained effectiveness, high persistence, and manageable safety profile of fingolimod over 36 months.

Clinical follow-up of 304 patients with multiple sclerosis three years after mitoxantrone treatment

2007 ◽  
Vol 13 (5) ◽  
pp. 626-631 ◽  
Author(s):  
M. Debouverie ◽  
L. Taillandier ◽  
S. Pittion-Vouyovitch ◽  
S. Louis ◽  
H. Vespignani
Keyword(s):  
Multiple Sclerosis ◽  
Interferon Beta ◽  
Progressive Multiple Sclerosis ◽  
Expanded Disability Status Scale ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Before And After ◽  
The Mean ◽  
Disease Modifying Treatment

The objectives of this study were to assess the benefits of 1) mitoxantrone after three years of follow-up and 2) disease-modifying treatment (DMT) after stopping mitoxantrone. A retrospective analysis was performed on 304 patients with active relapsing-remitting (RR) or progressive multiple sclerosis (PMS) who were treated with mitoxantrone. After mitoxantrone therapy, some patients received DMT (interferon-beta or glatiramer acetate) while others did not. The disease course of the two groups was evaluated by the Expanded Disability Status Scale (EDSS) before and after mitoxantrone and then every year for three years. The mean EDSS score at starting mitoxantrone and three years after stopping mitoxantrone respectively, were: 3.3 (1.3) and 3.2 (1.7) for the RRMS patients and 5.9 (1.2) and 6.4 (1.4) for the PMS patients. Before starting mitoxantrone, demographic and clinical parameters of predictive disability were not significantly different between patients who received DMT or not. The variation of EDSS between time of stopping mitoxantrone and three years later was significantly different (+0.9 versus +0.3; P=0.03) for patients with RRMS. We found that mitoxantrone treatment induces stable disease up to two years after discontinuation of mitoxantrone therapy. In the third year, patients without DMT deteriorated. Multiple Sclerosis 2007; 13: 626-631. http://msj.sagepub.com

Factors related with treatment adherence to interferon b and glatiramer acetate therapy in multiple sclerosis

2005 ◽  
Vol 11 (3) ◽  
pp. 306-309 ◽  
Author(s):  
Jordi Río ◽  
Joana Porcel ◽  
Nieves Téllez ◽  
Angela Sánchez-Betancourt ◽  
M ar Tintoré ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Side Effects ◽  
Expanded Disability Status Scale ◽  
Early Discontinuation ◽  
Treatment Expectations ◽  
Individualized Care ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Main Factor

Background: Awareness of the factors influencing discontinuation of immunomodulatory drugs (IMD) treatment in multiple sclerosis (MS) can help to find approaches to patient management with the aim of establishing more specific indications and also attaining more optimal patient selection in future clinical trials. Objective: To identify the causes that influence adhesion to IMD therapy within the clinical practice in a large cohort of patients with MS. Patients and methods: We have studied all MS patients who have initiated IMD in our hospital. All patients took part in training sessions where treatment expectations and side effects were explained and they received training in the administration technique. Reasons for stopping therapy were recorded during follow-up. Results: We studied 632 MS patients (mean follow-up was 47.1 (28.7) months). At the time of analysis, 107/632 patients (17%) were no longer receiving IMD. Almost half of the patients who stopped IMD (52/107) did so within the first two years on therapy. Fifty-six patients stopped IMD because of lack of efficacy. Only 27 patients (4.3%) discontinued treatment for reasons other than inefficacy or side effects. The proportion of patients with secondary progressive MS that stopped IMD therapy was 30%, while only 13.5% of the patients with relapsing—remitting MS stopped therapy (P= 0.0001). Expanded Disability Status Scale (EDSS) score at entry was the main factor that predicted interruption of therapy. Conclusions: The proportion of patients interrupting IMD in our centre is low, possibly due to individualized care. Higher EDSS, mainly in the first two years of treatment, is the main factor related with interruption. Close follow-up of these patients would be useful in avoiding early discontinuation of therapy.

scholarly journals Autologous Haematopoietic Stem Cell Transplantation in Active Multiple Sclerosis: a Real-world Case Series

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012449
Author(s):  
Richard S Nicholas ◽  
Elijah Edward Rhone ◽  
Alice Mariottini ◽  
Eli Silber ◽  
Omar Malik ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Real World ◽  
Haematopoietic Stem Cell ◽  
Expanded Disability Status Scale ◽  
Free Survival ◽  
Disability Status

Objective:to examine outcomes in people with multiple sclerosis (PwMS) treated with autologous hematopoietic stem cell transplantation (AHSCT) in a real-world setting.Methods:retrospective cohort study on PwMS treated with AHSCT at two centers in London, UK, consecutively between 2012 and 2019 who had ≥ 6 months of follow-up or died at any time. Primary outcomes were survival free of MS relapses, MRI new lesions and worsening of expanded disability status scale (EDSS). Adverse events rates were also examined.Results:the cohort includes 120 PwMS; 52% had progressive MS (primary or secondary) and 48% had relapsing-remitting MS (RRMS). At baseline, the median expanded disability status scale (EDSS) was 6.0; 90% of the evaluable cases showed MRI activity in the 12 months preceding AHSCT. Median follow-up after AHSCT was 21 months (range 6–85). MS relapse-free survival was 93% at 2 years and 87% at 4 years after AHSCT. No new MRI lesions were detected in 90% of subjects at 2 years and 85% at 4 years. EDSS progression-free survival (PFS) was 75% at 2 years and 65% at 4 years. EBV reactivation and monoclonal paraproteinemia were associated with worse PFS. There were 3 transplant-related deaths within 100 days (2.5%), all following fluid overload and cardiac or respiratory failure.Conclusions:efficacy outcomes of AHSCT in this real-world cohort are similar to those reported in more stringently selected clinical trial populations, although the risks may be higher.Classification of evidence:this study is rated Class IV because of the uncontrolled, open-label design.

scholarly journals Brain atrophy at onset and physical disability in multiple sclerosis

2012 ◽  
Vol 70 (10) ◽  
pp. 765-768 ◽  
Author(s):  
Juan Ignacio Rojas ◽  
Liliana Patrucco ◽  
Cristina Besada ◽  
Laura Bengolea ◽  
Edgardo Cristiano
Keyword(s):  
Multiple Sclerosis ◽  
Magnetic Resonance ◽  
Brain Atrophy ◽  
Physical Disability ◽  
Disease Onset ◽  
First Year ◽  
Expanded Disability Status Scale ◽  
Disability Status

The aim of this study was to investigate if brain atrophy in multiple sclerosis (MS) patients during the disease onset predicts long term disability. METHODS: MS patients with follow-up time of at least 7 years from disease onset and with baseline and second magnetic resonance 12 months later were included to measure brain atrophy. Expanded Disability Status Scale (EDSS) was categorized in three groups, EDSS=0, EDSS=1 and 2.5 and EDSS>2.5, and used as disability measure. RESULTS: Twenty-six patients were included. Mean atrophy during the first year in patients that reached an EDSS≥3 was -0.76±0.45 %, in patients with an EDSS between 1 and 2.5 was -0.59±0.56, while in patients with an EDSS of 0 it was -0.38±0.42 (p=0.003). DISCUSSION: Brain atrophy rates during the first year of disease were predictive of disease progression in our population.

scholarly journals Interferon beta-1b in treatment-naïve paediatric patients with relapsing–remitting multiple sclerosis: Two-year results from the BETAPAEDIC study

2017 ◽  
Vol 3 (4) ◽  
pp. 205521731774762 ◽  
Author(s):  
Jutta Gärtner ◽  
Wolfgang Brück ◽  
Almuth Weddige ◽  
Hannah Hummel ◽  
Christiane Norenberg ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Standard Deviation ◽  
Interferon Beta ◽  
Expanded Disability Status Scale ◽  
Paediatric Patients ◽  
Disability Status ◽  
Relapsing Remitting ◽  
Treatment Naïve ◽  
Relapsing Remitting Multiple Sclerosis

Background and objective Study evaluating Betaferon(R)'s safety and tolerability in paediatric patients with multiple sclerosis (BETAPAEDIC) is a prospective, open-label observational multicentre study to assess the safety and effectiveness of interferon beta-1b in paediatric patients with relapsing–remitting multiple sclerosis. Methods Treatment-naïve patients (12–16 years) scheduled to start interferon beta-1b were enrolled with follow-up visits every six months for two years. Effectiveness was evaluated by annualised relapse rate, Expanded Disability Status Scale progression, cranial magnetic resonance imaging and cognitive testing. Fatigue was assessed by the Fatigue Severity Scale. Results Sixty-eight patients were screened and 67 enrolled, with mean (standard deviation) age 14.2 (1.3) years ( n=65 in the effectiveness analysis). Mean disease duration was 11 months before study enrolment; at baseline, mean (standard deviation) Expanded Disability Status Scale was 0.6 (1.0); T2 lesion number 18.3 (15.1). Mean annualised relapse rate during the study was 0.7 ( n=57), 28/57 patients (49.1%) had no relapses and for 40/52 (76.9%) no Expanded Disability Status Scale progression was observed; 23/56 (41.1%) were relapse- and progression-free to last follow-up. Neuropsychological test and fatigue scores were within normal ranges (baseline and last follow-up). Eighteen patients had fatigue at some point. New T2 and gadolinium-enhancing (Gd+) lesions were seen in 43/55 (66.2%) and 29/55 (52.7%) patients respectively. Most frequent adverse events were influenza-like illness, headache, injection-site reactions and elevated liver enzymes. Conclusion Interferon beta-1b is an effective treatment with a favourable safety profile for paediatric patients.

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