The role of ocrelizumab in recommendations and in the Polish therapeutic programme DOI Creative Commons
K Kubicka

Aktualności Neurologiczne, Год журнала: 2023, Номер 23(3), С. 111 - 117

Опубликована: Дек. 28, 2023

Multiple sclerosis is a heterogeneous and chronic disease, the primary goal of treatment to prevent relapses slow progression disability. Ocrelizumab generally well-tolerated disease-modifying therapy for multiple sclerosis, with high efficacy in active relapsing forms, valuable method delaying disease patients progressive form. The activity defined on basis clinical course or radiological findings assessed over year. occurrence and/or magnetic resonance imaging taken into account. In line recommendations European Committee Treatment Research Sclerosis Academy Neurology, as well accordance guidelines American updated 2021, depending early stage choice drug should be motivated by higher efficacy. Recommendations most countries Europe around world are based above-mentioned guidelines. Poland, modifying were developed 2023 experts from Neuroimmunology Section Polish Neurological Society. B.29 programme National Health Fund Poland allows, 1 July 2023, addition escalation, using induction model highly effective therapies first-line treatment.

Язык: Английский

Investigating T cell-derived extracellular vesicles as biomarkers of disease activity, axonal injury, and disability in multiple sclerosis DOI Creative Commons

Jennifer L. Zagrodnik,

Stephanie N. Blandford, Neva J. Fudge

и другие.

Clinical & Experimental Immunology, Год журнала: 2025, Номер unknown

Опубликована: Янв. 8, 2025

Multiple sclerosis (MS) is a chronic immune-mediated demyelinating disease of the CNS, whereby clinical activity primarily monitored by magnetic resonance imaging (MRI). Given limitations associated with implementing and acquiring novel emerging biomarkers in routine practice, discovery biofluid may offer more simple cost-effective measure that would improve accessibility, standardization, patient care. Extracellular vesicles (EVs) are nanoparticles secreted from cells under both homeostatic pathological states, have been recently investigated as MS. The objectives this study were to longitudinally levels specific immune cell-derived EVs MS provide evidence EV sub-populations serve activity, axonal injury, and/or disability. Our results demonstrate rate disability negatively correlates changes circulating CD3+ within plasma. Additionally, numbers CD4+ decrease individuals increasing pNfL overtime magnitude increase plasma CD8+ EVs. Finally, when applying NEDA-3 criteria define active versus stable disease, had significantly elevated compared disease. In summary, analysis subsets method monitor accumulation, injury MS, while also providing insights into pathophysiology cellular/molecular mechanisms influence progression.

Язык: Английский

Процитировано

1
Predicting multiple sclerosis disease progression and outcomes with machine learning and MRI-based biomarkers: a review DOI Creative Commons
Hibba Yousef,

Brigitta Malagurski Tortei,

Filippo Castiglione

и другие.

Journal of Neurology, Год журнала: 2024, Номер unknown

Опубликована: Сен. 12, 2024

Multiple sclerosis (MS) is a demyelinating neurological disorder with highly heterogeneous clinical presentation and course of progression. Disease-modifying therapies are the only available treatment, as there no known cure for disease. Careful selection suitable necessary, they can be accompanied by serious risks adverse effects such infection. Magnetic resonance imaging (MRI) plays central role in diagnosis management MS, though MRI lesions have displayed moderate associations MS outcomes, clinico-radiological paradox. With advent machine learning (ML) healthcare, predictive power improved leveraging both traditional advanced ML algorithms capable analyzing increasingly complex patterns within neuroimaging data. The purpose this review was to examine application MRI-based prediction disease Studies were divided into five main categories: predicting conversion clinically isolated syndrome cognitive outcome, EDSS-related disability, motor disability activity. performance models discussed along highlighting influential MRI-derived biomarkers. Overall, presents promising avenue prognosis. However, integration biomarkers other multimodal patient data shows great potential advancing personalized healthcare approaches MS.

Язык: Английский

Процитировано

4
Unlocking Asthma Remission: Key Insights From an Expert Roundtable Discussion DOI Creative Commons
Dennis Thomas, Hayley Lewthwaite, Peter G. Gibson

и другие.

Respirology, Год журнала: 2025, Номер unknown

Опубликована: Май 23, 2025

ABSTRACT Treatment targets in severe asthma have evolved towards a remission‐focused paradigm guided by precision medicine. This novel concept requires shift from evaluating the efficacy of therapies based on single outcome at time point to an that captures complexity remission involving several domains assessed over sustained period. Since is still emerging, multiple definitions been proposed, ranging symptom control and exacerbation‐free resolution underlying pathobiology, with varying rigour each parameter. Understanding strengths weaknesses current construct needed progress further. We conducted roundtable discussion 27 experts address this issue, discussions were narratively synthesised summarised. The participants observed between one three five people treated targeted biological or macrolides experience low disease activity They unanimously agreed labelling attained clinical state as useful (e.g., facilitating treat‐to‐target approach), policy widening eligibility criteria for biologics), scientific path understanding cure) tool. Current rates vary significantly due definition variability. When assessing remission, it essential consider confounding factors steroid use adrenal insufficiency). More research required reach acceptable definition, including patient's voice such essential. In conclusion, treatment‐induced possible valuable asthma. However, further refinement required. image

Язык: Английский

Процитировано

0
Financial burden of out of pocket nonpharmacological therapies in an Austrian multiple sclerosis cohort DOI Creative Commons

Tiago Lerda Casaccia,

Simon Hefner,

J Strauss

и другие.

Scientific Reports, Год журнала: 2025, Номер 15(1)

Опубликована: Март 19, 2025

Given the financial constraints in public healthcare, we investigated monthly out-of-pocket expenses for non-pharmacological treatments among individuals with multiple sclerosis (MS), a chronic neurological disorder that primarily affects of working age. This cross-sectional study employed an online questionnaire to evaluate and utilization treatments, as well weekly hours 104 relapsing-remitting MS (RRMS, 79%), secondary progressive (SPMS, 12%), primary (PPMS, 10%). Non-pharmacological were used by 82% participants (vitamin D (43%), physiotherapy (31%), massage (21%), magnesium (19%)). The average 136 EUR (SD ± 218) significantly higher PPMS (337 SD 354) compared RRMS (110 195; p = 0.01). 26 lower (11 h/week, 16; 0.008) SPMS (13 0.001) cohort (30 15). Working not related individual costs. reveals substantial incurred Austria, particularly those PPMS, highlighting willingness actively participate their disease management. Physicians should be aware resources inform about available evidence on treatment approaches.

Язык: Английский

Процитировано

0
Advancements in multiple sclerosis DOI Creative Commons
Turlough Montague,

James Drummond,

Karl Ng

и другие.

Internal Medicine Journal, Год журнала: 2025, Номер unknown

Опубликована: Апрель 2, 2025

The global prevalence of multiple sclerosis (MS) is increasing, and early diagnosis treatment essential in mitigating disability. While recent therapeutic advancements have significantly reduced relapse rates, the progressive degenerative aspects MS continue to pose major challenges. This year updates McDonald diagnostic criteria aim enhance sensitivity facilitate earlier use disease-modifying therapies asymptomatic patients. Additionally, novel biomarkers will gain ground clinical practice offer new approaches optimising care. Following widespread cell depleting immunosuppressive agents, innovative directions such as chimeric antigen receptor T-cell therapy Epstein-Barr virus (EBV) vaccination represent promising management MS. perspective provides a comprehensive overview disease while highlighting important shaping future field.

Язык: Английский

Процитировано

0
Comparison of CSF biomarkers in multiple sclerosis patients treated with natalizumab and rituximab DOI Creative Commons
Johan Hellgren,

Isabella Ahlström,

Maria Strandberg

и другие.

Multiple Sclerosis and Related Disorders, Год журнала: 2025, Номер 99, С. 106479 - 106479

Опубликована: Май 1, 2025

Patients with relapsing remitting multiple sclerosis (RRMS) treated the high-efficacy drugs natalizumab (NTZ) or rituximab (RTX) generally show no evidence of disease activity. Currently, there is head-to-head comparison between NTZ and RTX neurodegenerative neuroinflammatory biomarkers in cerebrospinal fluid (CSF). To compare CSF a stable RRMS cohort RTX. A secondary objective was to explore potential associations biomarkers, fatigue, cognition. This Swedish multicentre cross-sectional study assessed kappa-free light chain (k-FLC) index, oligoclonal bands (OCBs), glial fibrillary acidic protein (GFAP) neurofilament (NfL) 30 patients on for at least 24 months. longitudinal performed IgG Indices. Fatigue cognition outcomes were explored relation biomarkers. GFAP level significantly higher group compared (mean difference (CI): 2 716 (155; 5278) ng/L, p=0.047). NfL concentration did not differ groups. OCBs k-FLC index present elevated 97 % 87 participants, respectively. IgG-index reduced only NTZ. No significant found Our results support that intrathecal inflammatory activity still ongoing Cross-sectional might indicate lower risk long-term disability group. data should be interpreted caution because small sample size, making it difficult control confounders.

Язык: Английский

Процитировано

0
An update on the evaluation of treatment switching criteria in multiple sclerosis DOI
Kévin Bigaut,

Judicaelle Didierjean,

de Sèze

и другие.

Expert Review of Neurotherapeutics, Год журнала: 2025, Номер unknown

Опубликована: Май 13, 2025

Multiple sclerosis (MS) is a complex disorder driven by both inflammatory and neurodegenerative processes. While disease-modifying therapies (DMTs) have significantly improved prognosis, robust treatment switching criteria remain essential to balance efficacy safety over the disease course. This review examines historical current for escalating DMTs from moderate- high-efficacy (HET). The authors summarize emerging clinical, imaging, biological markers that inform decision-making explore strategies de-escalation, including DMT discontinuation innovative approaches such as exit bridge therapies. Recent advances in MS management emphasize earlier initiation of HET more stringent criteria. Although monitoring tools - clinical evaluations, markers, patient-reported outcomes (PROs) enhance assessment, they require further validation, standardization, broader accessibility. Similarly, de-escalation need additional research optimize patient selection.

Язык: Английский

Процитировано

0
CONFIDENCE treatment success: long-term real-world effectiveness and safety of ocrelizumab in Germany DOI Creative Commons
Mathias Buttmann, Martin S. Weber, Sven G. Meuth

и другие.

Frontiers in Neurology, Год журнала: 2025, Номер 16

Опубликована: Май 21, 2025

Background Early high-efficacy treatment for people with relapsing multiple sclerosis (pwRMS) may provide better long-term outcomes compared the escalation strategy. In this study, we present an analysis of success and safety from CONFIDENCE study in a real-world cohort pwRMS treated ocrelizumab different lines up to 5.5 years. Methods The ongoing German non-interventional post-authorization (ML39632, EUPAS22951), evaluates effectiveness therapy pwMS newly or other disease-modifying therapies 10 This presents (proportion no clinical disease activity measured by relapse progression discontinuation due adverse event [AE] lack therapeutic effectiveness), confirmed disability (CDP), annualized rates, stratified number previous MS (PMSTs). Results At data cutoff (11 October 2023), full set included 2,261 ≥1 dose ocrelizumab. baseline, mean age (SD) participants was 41.16 (11.39) years (treatment-naïve, 39.19 [12.95] years; ≥3 PMSTs, 42.80 [10.08] years), Expanded Disability Status Scale (EDSS) score 3.08 (1.86) 2.37 [1.54]; 3.57 [1.90]). Overall, 58.4% continuous achieved baseline until year 5 (74.0 50.3% 0 PMSTs). main reasons not achieving were CDP, while AEs played minor role. proportion did increase longer duration tended be higher more PMSTs. spectrum similar across lines, new unexpected observed. Conclusion remained high over treatment, even among RMS Only small discontinued AEs. These results support early intervention optimize pwRMS. Trial registration https://catalogues.ema.europa.eu/node/3142/administrative-details , identifiers ML39632 EUPAS22951.

Язык: Английский

Процитировано

0
In multiple sclerosis patients a single serum neurofilament light chain (sNFL) dosage is strongly associated with 12 months outcome: data from a real-life clinical setting DOI

Simona Malucchi,

Cecilia Irene Bava, Paola Valentino

и другие.

Journal of Neurology, Год журнала: 2024, Номер unknown

Опубликована: Сен. 23, 2024

Язык: Английский

Процитировано

2
Adult and pediatric relapsing multiple sclerosis phase II and phase III trial design and their primary end points: A systematic review DOI Creative Commons
Katsutoshi Hiramatsu, Hideki Maeda

Clinical and Translational Science, Год журнала: 2024, Номер 17(5)

Опубликована: Май 1, 2024

Abstract No systematic review of trial designs in patients with relapsing multiple sclerosis (RMS) was reported. This conducted on the and primary end points (PEs) phase II III trials intended to modify natural course disease RMS. The purpose study is explore trends/topics discussion clinical design PE, comparing them regulatory guidelines expert recommendations. Three registration systems, ClinicalTrials.gov , EU Clinical Trials Register, Japan Registry Trials, were used 60 evaluated. dominant a randomized controlled parallel‐arms other details as follows: adult confirmatory ( n = 32), active‐controlled double‐blind (DBT) (53%) open‐label assessor‐masking (16%); dose‐finding 9), placebo‐ DBT (44%), placebo‐controlled (22%), add‐on (22%); pediatric 8), (38%) non‐masking (25%). most common PEs trials, annual relapse rate (ARR) (56%) no evidence activity‐3 (NEDA‐3) (13%); cumulative number T1 gadolinium‐enhancing lesions (56%), combined unique active overall disability response score ARR time first It suggested that some parts recommendations need be revised.

Язык: Английский

Процитировано

1