19 research outputs found
Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study
Background
The development and approval of disease modifying treatments have dramatically changed disease progression in patients with spinal muscular atrophy (SMA). Nusinersen was approved in Europe in 2017 for the treatment of SMA patients irrespective of age and disease severity. Most data on therapeutic efficacy are available for the infantile-onset SMA. For patients with SMA type 2 and type 3, there is still a lack of sufficient evidence and long-term experience for nusinersen treatment. Here, we report data from the SMArtCARE registry of non-ambulant children with SMA type 2 and typen 3 under nusinersen treatment with a follow-up period of up to 38 months.
Methods
SMArtCARE is a disease-specific registry with data on patients with SMA irrespective of age, treatment regime or disease severity. Data are collected during routine patient visits as real-world outcome data. This analysis included all non-ambulant patients with SMA type 2 or 3 below 18 years of age before initiation of treatment. Primary outcomes were changes in motor function evaluated with the Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM).
Results
Data from 256 non-ambulant, pediatric patients with SMA were included in the data analysis. Improvements in motor function were more prominent in upper limb: 32.4% of patients experienced clinically meaningful improvements in RULM and 24.6% in HFMSE. 8.6% of patients gained a new motor milestone, whereas no motor milestones were lost. Only 4.3% of patients showed a clinically meaningful worsening in HFMSE and 1.2% in RULM score.
Conclusion
Our results demonstrate clinically meaningful improvements or stabilization of disease progression in non-ambulant, pediatric patients with SMA under nusinersen treatment. Changes were most evident in upper limb function and were observed continuously over the follow-up period. Our data confirm clinical trial data, while providing longer follow-up, an increased number of treated patients, and a wider range of age and disease severity
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Parallel Session 3: Acute/Vascular/Trauma| Wed 18 May, 1445 – 1600|4 Circulating Interleukin-6 predicts carotid study
Background and AimsInterleukin-6 (IL-6) has important roles in atherosclerosis pathophysiology. To determine if anti-IL-6 therapy could be an adjuvant stroke prevention strategy in patients with carotid atherosclerosis, we tested whether circulating IL-6 levels predict carotid plaque severity, vulnerability, and progression in the Cardiovascular Health Study.MethodsCarotid ultrasound was performed at baseline and 5 years. Plaque severity was scored 0 to 5 based on NASCET grade of stenosis. Plaque vulnerability at baseline was the presence of irregular, ulcerated or echolucent plaques. Plaque progression at 5 years was a ≥1 point increase in stenosis severity. Relationship of plasma IL-6 levels with plaque characteristics was modeled using multivariable linear (severity) or logistic (vulnerability and progression) regression. Risk factors of atherosclerosis were included as independent variables.ResultsIn 4334 participants with complete data (58.9% women, 72.7 ± 5.1 years). There were 1267 (29.2%) participants with vulnerable plaque and 1474 (34.0%) with plaque progression. Log IL-6 predicted plaque severity (β = 0.09, p=0.04), vulnerability (OR = 1.22, 95% CI: 1.06-1.40, p=0.006) and progression (OR = 1.44, 95% CI: 1.23-1.69, p50% probability of progression, mean log IL-6 was 0.54 corresponding to 2.0 pg/mL. Dichotomizing IL-6 levels did not affect performance of regression models.ConclusionsPlasma IL-6 predicts carotid plaque severity, vulnerability, and progression. The 2.0 pg/mL cut-off could help select individuals that would benefit from anti-IL-6 drugs for stroke prevention
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Additional Opportunity to Rectify Misconceptions Regarding Current Best Management of Asymptomatic Carotid Stenosis
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