248 research outputs found
First-line therapies in Late Onset Multiple Sclerosis: an Italian registry study
No full textBackground: The diagnosis of late onset (≥50 years) relapsing remitting multiple sclerosis (LORRMS) has been increasingly described in clinical practice, at the same time, data focusing on the specific therapeutic management of LORRMS are scarce.
Objective: To compare effectiveness of injectable and oral first line disease modifying therapies (DMTs) in a cohort of LORRMS patients with for time to first relapse, time to confirmed disability progression (CDP), and, additionally, for time to discontinuation.
Methods: This is a multicenter, observational, retrospectively acquired cohort study on LORRMS-naïve patients from the Italian MS Register who started either injectable or oral first line DMTs between January 1, 2013 and December 31, 2017. LORRMS were divided into two groups, namely the injectable group (IG) and oral group (OG). Cox models adjusted with inverse probability weighted propensity score were built for the investigated outcomes.
Results: Of a cohort of 3,989 patients, 302 were enrolled (203 in the IG and 99 in the OG). The two cohorts did not differ for baseline characteristics. Time to first relapse did not show any difference between the two groups (HR = 1.10; CI 95% 0.50-2.46, p=.797). Furthermore, no differences were found between the two groups with respect to the risk of CDP (HR = 1.04; CI 95% 0.35-3.06, p = .939), nor for the risk of DMT discontinuation (HR = 0.90; CI 95% 0.17-2.08, p =.425).
Conclusions: Real-world data from the Italian MS Register suggested that both injectables and oral first line DMTs controlled similarly the investigated outcomes in LORRMS
Long-term effectiveness in patients previously treated with cladribine tablets: a real-world analysis of the Italian multiple sclerosis registry (CLARINET-MS)
No full textBackground: The CLARINET-MS study assessed the long-term effectiveness of cladribine tablets by following patients with multiple sclerosis (MS) in Italy, using data from the Italian MS Registry.Methods: Real-world data (RWD) from Italian MS patients who participated in cladribine tablets randomised clinical trials (RCTs; CLARITY, CLARITY Extension, ONWARD or ORACLE-MS) across 17 MS centres were obtained from the Italian MS Registry. RWD were collected during a set observation period, spanning from the last dose of cladribine tablets during the RCT (defined as baseline) to the last visit date in the registry, treatment switch to other disease-modifying drugs, date of last Expanded Disability Status Scale recording or date of the last relapse (whichever occurred last). Time-to-event analysis was completed using the Kaplan-Meier (KM) method. Median duration and associated 95% confidence intervals (CI) were estimated from the model.Results: Time span under observation in the Italian MS Registry was 1-137 (median 80.3) months. In the total Italian patient population (n = 80), the KM estimates for the probability of being relapse-free at 12, 36 and 60 months after the last dose of cladribine tablets were 84.8%, 66.2% and 57.2%, respectively. The corresponding probability of being progression-free at 60 months after the last dose was 63.7%. The KM estimate for the probability of not initiating another disease-modifying treatment at 60 months after the last dose of cladribine tablets was 28.1%, and the median time-to-treatment change was 32.1 (95% CI 15.5-39.5) months.Conclusion: CLARINET-MS provides an indirect measure of the long-term effectiveness of cladribine tablets. Over half of MS patients analysed did not relapse or experience disability progression during 60 months of follow-up from the last dose, suggesting that cladribine tablets remain effective in years 3 and 4 after short courses at the beginning of years 1 and 2
Long-term effectiveness in patients previously treated with cladribine tablets: a real-world analysis of the Italian multiple sclerosis registry (CLARINET-MS)
No full textBackground: The CLARINET-MS study assessed the long-term effectiveness of cladribine tablets by following patients with multiple sclerosis (MS) in Italy, using data from the Italian MS Registry. Methods: Real-world data (RWD) from Italian MS patients who participated in cladribine tablets randomised clinical trials (RCTs; CLARITY, CLARITY Extension, ONWARD or ORACLE-MS) across 17 MS centres were obtained from the Italian MS Registry. RWD were collected during a set observation period, spanning from the last dose of cladribine tablets during the RCT (defined as baseline) to the last visit date in the registry, treatment switch to other disease-modifying drugs, date of last Expanded Disability Status Scale recording or date of the last relapse (whichever occurred last). Time-to-event analysis was completed using the Kaplan–Meier (KM) method. Median duration and associated 95% confidence intervals (CI) were estimated from the model. Results: Time span under observation in the Italian MS Registry was 1–137 (median 80.3) months. In the total Italian patient population (n = 80), the KM estimates for the probability of being relapse-free at 12, 36 and 60 months after the last dose of cladribine tablets were 84.8%, 66.2% and 57.2%, respectively. The corresponding probability of being progression-free at 60 months after the last dose was 63.7%. The KM estimate for the probability of not initiating another disease-modifying treatment at 60 months after the last dose of cladribine tablets was 28.1%, and the median time-to-treatment change was 32.1 (95% CI 15.5–39.5) months. Conclusion: CLARINET-MS provides an indirect measure of the long-term effectiveness of cladribine tablets. Over half of MS patients analysed did not relapse or experience disability progression during 60 months of follow-up from the last dose, suggesting that cladribine tablets remain effective in years 3 and 4 after short courses at the beginning of years 1 and 2
The Swiss Multiple Sclerosis Registry (SMSR): study protocol of a participatory, nationwide registry to promote epidemiological and patient-centered MS research
No full textAbstract Background Multiple sclerosis (MS) is one of the most frequently observed neurological conditions in Switzerland, but data sources for country-wide epidemiological trend monitoring are lacking. Moreover, while clinical and laboratory MS research are generally well established, there is a gap in patient-centered MS research to inform care management, or treatment decisions and policy making not only in Switzerland but worldwide. Methods In light of these research gaps, the Swiss Multiple Sclerosis Society initiated and funded the Swiss Multiple Sclerosis Registry (SMSR) an open-ended, longitudinal and prospective, nationwide, patient-centered study. The SMSR recruits adult persons with a suspected or confirmed MS diagnosis who reside or receive care in Switzerland. The SMSR has established a governance structure with clear rules and guidelines. It follows a citizen-science approach with direct involvement of persons with MS (PwMS), who contribute actively to registry development, operations, and research. Main scientific goals entail the study of MS epidemiology in Switzerland, health care access and provision, as well as life circumstances and wellbeing of persons with MS. The innovative study design (“layer model”) offers several participation options with different time commitments. Data collection is by means of regular surveys and medical record abstraction. Survey participation is offered in different modes (web, paper & pencil) and in the three main national languages (German, French, Italian). Participants also receive regular data feedbacks for personal use and self-monitoring, contextualized in the whole population of study participants. Data feedbacks are also used to solicit data corrections of key variables from participants. Discussion The SMSR combines the advantages of traditional and novel research methods in medical research and has recruited over 1600 PwMS in its first year. The future-oriented design and technology will enable a response not only to future technological innovations and research trends, but also to challenges in health care provision for MS. Trial registration ClinicalTrials.gov NCT02980640; December 6, 2016; retrospectively registered
Relapse-associated worsening in a real-life multiple sclerosis cohort: The role of age and pyramidal phenotype
No full textThe Italian Dystonia Registry: rationale, design and preliminary findings
No full textThe Italian Dystonia Registry is a multicenter data collection system that will prospectively assess the phenomenology and natural history of adult-onset dystonia and will serve as a basis for future etiological, pathophysiological and therapeutic studies. In the first 6 months of activity, 20 movement disorders Italian centres have adhered to the registry and 664 patients have been recruited. Baseline historical information from this cohort provides the first general overview of adult-onset dystonia in Italy. The cohort was characterized by a lower education level than the Italian population, and most patients were employed as artisans, builders, farmers, or unskilled workers. The clinical features of our sample confirmed the peculiar characteristics of adult-onset dystonia, i.e. gender preference, peak age at onset in the sixth decade, predominance of cervical dystonia and blepharospasm over the other focal dystonias, and a tendency to spread to adjacent body parts, The sample also confirmed the association between eye symptoms and blepharospasm, whereas no clear association emerged between extracranial injury and dystonia in a body site. Adult-onset dystonia patients and the Italian population shared similar burden of arterial hypertension, type 2 diabetes, coronary heart disease, dyslipidemia, and hypothyroidism, while hyperthyroidism was more frequent in the dystonia population. Geographic stratification of the study population yielded no major difference in the most clinical and phenomenological features of dystonia. Analysis of baseline information from recruited patients indicates that the Italian Dystonia Registry may be a useful tool to capture the real world clinical practice of physicians that visit dystonia patients
The Italian Natalizumab Registry: a 1-year follow-up
No full textBackground: Natalizumab is labelled by EMEA (A) as second line in relapsing remitting multiple sclerosis (RRMS) in non-responders to -interferon, and (B) as first line in more aggressive RRMS. Italian Medicines Agency (AIFA), accordingly to a neurological expert panel, established more restrictive criteria to dispense and reimburse
natalizumab, and organised a national registry for monitoring
its safety.
Objectives: To assess risks associated with natalizumab after 1 year since its marketing in Italy, and to compare the Italian Registry data with the characteristics of RCT AFFIRM patients.
Methods: Patients under treatment with natalizumab were enrolled into a web-based registry available on AIFA website. It contains electronic forms on: patient eligibility, follow-up during the therapy (to fill every 3 months), drug withdrawal, ADR report, and follow-up after drug withdrawal (to fill every 6 months). It has been active since January 2007. Data up to January 2008 were collected and analysed.
Results: Ninety-six MS Centres have enrolled at least one patient. Overall, 696 pts (67% females) were treated: 91% as non-responders to -interferon (A), and 9% with aggressive RRMS (B). Patient baseline disease was more serious compared to AFFIRM: annual relapses were 2.31.0 (average SD) in A pts and 3.51.3 in B pts vs 1.50.9 in AFFIRM pts; EDSS disability was 3.71.5 in A pts and 3.71.4 in B pts vs 2.31.2 in AFFIRM pts. Main pts (53%) received 1–4 drug doses. For 371 pts, at least 1 follow-up was recorded and 78 pts had at least 1 ADR. Hundred ADRs were recorded and 29% were infections. Drug effectiveness in terms of EDSS on 270 pts with 4 to 9 months therapy showed improvements in 49% of cases.
Conclusions: Data on ADRs were consistent with available evidences. The national registry appears a useful tool to closely monitor a drug with critical safety profile
Predictors of treatment switching in the Big Multiple Sclerosis Data Network
No full textBackgroundTreatment switching is a common challenge and opportunity in real-world clinical practice. Increasing diversity in disease-modifying treatments (DMTs) has generated interest in the identification of reliable and robust predictors of treatment switching across different countries, DMTs, and time periods.ObjectiveThe objective of this retrospective, observational study was to identify independent predictors of treatment switching in a population of relapsing-remitting MS (RRMS) patients in the Big Multiple Sclerosis Data Network of national clinical registries, including the Italian MS registry, the OFSEP of France, the Danish MS registry, the Swedish national MS registry, and the international MSBase Registry.MethodsIn this cohort study, we merged information on 269,822 treatment episodes in 110,326 patients from 1997 to 2018 from five clinical registries. Patients were included in the final pooled analysis set if they had initiated at least one DMT during the relapsing-remitting MS (RRMS) stage. Patients not diagnosed with RRMS or RRMS patients not initiating DMT therapy during the RRMS phase were excluded from the analysis. The primary study outcome was treatment switching. A multilevel mixed-effects shared frailty time-to-event model was used to identify independent predictors of treatment switching. The contributing MS registry was included in the pooled analysis as a random effect.ResultsEvery one-point increase in the Expanded Disability Status Scale (EDSS) score at treatment start was associated with 1.08 times the rate of subsequent switching, adjusting for age, sex, and calendar year (adjusted hazard ratio [aHR] 1.08; 95% CI 1.07-1.08). Women were associated with 1.11 times the rate of switching relative to men (95% CI 1.08-1.14), whilst older age was also associated with an increased rate of treatment switching. DMTs started between 2007 and 2012 were associated with 2.48 times the rate of switching relative to DMTs that began between 1996 and 2006 (aHR 2.48; 95% CI 2.48-2.56). DMTs started from 2013 onwards were more likely to switch relative to the earlier treatment epoch (aHR 8.09; 95% CI 7.79-8.41; reference = 1996-2006).ConclusionSwitching between DMTs is associated with female sex, age, and disability at baseline and has increased in frequency considerably in recent years as more treatment options have become available. Consideration of a patient's individual risk and tolerance profile needs to be taken into account when selecting the most appropriate switch therapy from an expanding array of treatment choices
Multiple sclerosis and neuromyelitis optica spectrum disorders in Argentina: comparing baseline data from the Argentinean MS Registry (RelevarEM)
No full textThe objective of this study was to describe and compare the baseline epidemiological data of multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD) patients included in RelevarEM (Clinical Trials registry number NCT03375177).
Methods: RelevarEM is a longitudinal, strictly observational MS and NMOSD registry in Argentina. Epidemiological and comorbidity data from MS and NMOSD patients were described and compared. For comorbidities, the Charlson comorbidity index (CCI) was used to calculate the burden at entry. CCI was stratified in 0 and ≥ 1 and described for the entire cohort.
Results: A total of 1588 and 75 MS and NMOSD patients (respectively) were included. For MS patients, the mean age was 42 ± 7 years, female sex 65.3%, mean EDSS 2, and mean disease duration 8 ± 6 years. In NMOSD, the mean age was 40 ± 7 years, female sex 78.7%, mean disease duration 5 ± 3.5 years, and mean EDSS 2.5. The most frequent MS phenotype was RRMS in 82.4%. In MS, the CCI was 0 in 85.8.2% while ≥ 1 was in 14.2% of patients. Regarding phenotype stratification, CCI ≥ 1 was 3.9% in CIS, 13.5% in RRMS, 28.7% in SPMS, and 17.4% in PPMS (p < 0.001 between groups). In NMOSD, the CCI was 0 in 64% while ≥ 1 was in 36%. The MS/NMOSD ratio found was 21/1.
Conclusions: This is the first analysis of the longitudinal Argentinean registry of MS and NMOSD describing and comparing conditions that contributes to provide reliable real-world data in the country.Fil: Correale, Jorge. Fleni. Departamento de Neurología. Servicio de Neuroinmunología y Enfermedades Desmielinizantes; Argentina.Fil: Gaitán, María Inés. Fleni. Departamento de Neurología. Servicio de Neuroinmunología y Enfermedades Desmielinizantes; Argentina.Fil: Marrodán, Mariano. Fleni. Departamento de Neurología. Servicio de Neuroinmunología y Enfermedades Desmielinizantes; Argentina.Fil: Negrotto, Laura. Fleni. Departamento de Neurología. Servicio de Neuroinmunología y Enfermedades Desmielinizantes; Argentina.Fil: Ysrraelit, Maria. Fleni. Departamento de Neurología. Servicio de Neuroinmunología y Enfermedades Desmielinizantes; Argentina.Fil: Rojas, Juan Ignacio. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina. Centro de Esclerosis Múltiple de Buenos Aires; Argentina.Fil: Alonso Serena, Marina. Hospital Italiano de Buenos Aires. Área de Investigación en Medicina Interna; Argentina.Fil: Garcea, Orlando. Universidad de Buenos Aires. Facultad de Medicina. Centro Universitario de Esclerosis Múltiple; Argentina. Hospital Dr. J. M. Ramos Mejía; Argentina.Fil: Patrucco, Liliana. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina.Fil: Carrá, Adriana. Hospital Británico. Sección de Enfermedades Desmielinizantes; Argentina. Fundación Favaloro/INECO; Argentina.Fil: Vrech, Carlos. Sanatorio Allende. Departamento de Enfermedades desmielinizantes; Argentina.Fil: Pappolla, Agustín. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina.Fil: Miguez, Jimena. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina.Fil: Doldan, María L. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina. Centro de Esclerosis Múltiple de Buenos Aires, Buenos Aires, Argentina.Fil: Silveira, Facundo. Hospital Italiano de Buenos Aires. Servicio de Neurología; Argentina.Fil: Alonso, Ricardo. Universidad de Buenos Aires. Facultad de Medicina. Centro Universitario de Esclerosis Múltiple; Argentina. Hospital Dr. J. M. Ramos Mejía; Argentina. Sanatorio Güemes; Argentina.Fil: Cohen, Leila. Universidad de Buenos Aires. Facultad de Medicina. Centro Universitario de Esclerosis Múltiple; Argentina. Hospital Dr. J. M. Ramos Mejía; Argentina.Fil: Pita, Cecilia. Universidad de Buenos Aires. Facultad de Medicina. Centro Universitario de Esclerosis Múltiple; Argentina. Hospital Dr. J. M. Ramos Mejía; Argentina.Fil: Silva, Berenice Anabel. Universidad de Buenos Aires. Facultad de Medicina. Centro Universitario de Esclerosis Múltiple; Argentina. Hospital Dr. J. M. Ramos Mejía; Argentina
- …
