Neurological SciencesPub Date : 2025-04-01Epub Date: 2024-12-12DOI: 10.1007/s10072-024-07940-8
Shanwen Chen, Lu Wang, Quanli Kang
{"title":"Herniation secondary to critical cerebrospinal fluid hypovolemia after supratentorial craniotomy: a single-center case series.","authors":"Shanwen Chen, Lu Wang, Quanli Kang","doi":"10.1007/s10072-024-07940-8","DOIUrl":"10.1007/s10072-024-07940-8","url":null,"abstract":"<p><strong>Background: </strong>Critical cerebrospinal fluid hypovolemia (CCSFH) is a rare postcraniotomy condition in patients with acute supratentorial brain injury, often mistaken for intracranial hypertension. This article aims to enhance awareness of CCSFH by describing its clinical and radiological characteristics.</p><p><strong>Methods: </strong>Between January 2019 and November 2023, 330 consecutive patients with acute critical brain injury underwent supratentorial craniotomy. CCSFH diagnosis was based on three criteria: a decline in consciousness, head CT scans revealing midline shift of ≥ 5 mm, and rapid clinical or imaging presentation improvement within three days after implementing various treatments to increase CSF volume. Clinical and imaging features, treatment courses, and responses were analyzed. Midline shift on head CT scans was measured at four time points before and after surgery and compared using repeated measures ANOVA.</p><p><strong>Results: </strong>Fifteen patients (4.5%) developed CCSFH within 1 to 13 days post-surgery. Of them, seven patients exhibited a decline in consciousness or mental status, with three presenting anisocoria. The remaining eight had normal pupil sizes, complicating consciousness assessment due to postoperative sedation and analgesia. The average midline shift was 10.84 ± 2.83 mm during the CCSFH presentation, which showed a statistically significant difference from the initial postoperative measurement (p = 0.005) but not from the preoperative measurement (p = 0.536). Intracranial pressure ranged from 1-11 mmHg in five cases. The first four cases underwent an unplanned decompressive craniectomy as their conditions progressed to severe cingulate or transtentorial herniation, attributable to unawareness of cerebrospinal fluid hypovolemia before the second surgery. Drawing from the accumulated experiences, the subsequent eleven cases of CCSFH were promptly identified upon onset, and appropriate treatments were administered, with the supine position serving as the primary intervention modality. The CCSFH condition was successfully reversed in all patients.</p><p><strong>Conclusions: </strong>CCSFH after craniotomy should be considered, and prompt identification and intervention are required in cases of clinical deterioration. The primary management strategy is placing the patient supine, along with stopping cerebrospinal fluid drainage, halting hyperosmotic diuretics, and administering intravenous hydration, often leading to favorable outcomes.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":"1765-1775"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142813745","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurological SciencesPub Date : 2025-04-01Epub Date: 2025-01-14DOI: 10.1007/s10072-025-07997-z
Matteo Betti, Cristina Giubbilei, Simona D'angelo, Emilio Portaccio, Maria Pia Amato, Valentina Carrai
{"title":"Multiple sclerosis and sickle cell anemia: when two worlds collide.","authors":"Matteo Betti, Cristina Giubbilei, Simona D'angelo, Emilio Portaccio, Maria Pia Amato, Valentina Carrai","doi":"10.1007/s10072-025-07997-z","DOIUrl":"10.1007/s10072-025-07997-z","url":null,"abstract":"<p><strong>Background: </strong>The coexistence of sickle cell anemia and multiple sclerosis in a single patient presents a rare and challenging clinical scenario, possibly favoured by the interplay between chronic inflammatory states and autoimmune processes.</p><p><strong>Methos/results: </strong>We present the case of a 36-year-old woman with sickle cell anemia who developed progressive neurological symptoms leading to frequent falls and paraparesis; magnetic resonance imaging showed many periventricular, infratentorial, and both cervical and dorsal spinal cord lesions, leading to a diagnosis of multiple sclerosis. After a multidisciplinary approach the patient was successfully started on ofatumumab.</p><p><strong>Conclusion: </strong>This case report raises awareness among clinicians to consider the possibility of multiple sclerosis in patients with sickle cell anemia who present with neurological symptoms, ensuring timely diagnosis and intervention, and emphasizes the need for a personalized, multidisciplinary approach to care.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":"1897-1900"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurological SciencesPub Date : 2025-04-01Epub Date: 2024-12-02DOI: 10.1007/s10072-024-07894-x
Elena Anghileri, Paola Gaviani, Anna Amato, Bianca Pollo, Rosina Paterra, Marcello Marchetti, Fabio M Doniselli, Francesco Restelli, Marica Eoli, Ludmila de Oliveira Muniz Koch, Veronica Redaelli, Andrea Giorgio Botturi, Francesco DiMeco, Paolo Ferroli, Mariangela Farinotti, Antonio Silvani
{"title":"Choroid plexus tumors in adults: a retrospective mono-institutional study.","authors":"Elena Anghileri, Paola Gaviani, Anna Amato, Bianca Pollo, Rosina Paterra, Marcello Marchetti, Fabio M Doniselli, Francesco Restelli, Marica Eoli, Ludmila de Oliveira Muniz Koch, Veronica Redaelli, Andrea Giorgio Botturi, Francesco DiMeco, Paolo Ferroli, Mariangela Farinotti, Antonio Silvani","doi":"10.1007/s10072-024-07894-x","DOIUrl":"10.1007/s10072-024-07894-x","url":null,"abstract":"<p><strong>Purpose: </strong>Choroid plexus tumors (CPT) are rare entities, and even rarer in adulthood.</p><p><strong>Methods: </strong>A retrospective consecutive series of 24 adult CPT patients was reviewed.</p><p><strong>Results: </strong>We described 24 adult CPTs. Clinical onset included cerebellar signs (n = 11, 45.8%), intracranial hypertension signs (n = 8, 33.4%), cranial nerves impairment (n = 5, 20.8%), incidental findings (n = 4, 16.6%), seizures (n = 1, 4.2%), spinal signs (n = 1, 4.2%). At first diagnosis, CPT was mostly located in the ventricular system, but other locations can occur, including the spine (one case); meningeal involvement was present in one, pre-surgical hydrocephalus in one case only. CPT histological grade ranged from grade 1 (n = 17), grade 2 (n = 6), and grade 3 (n = 1). TERTp mutation was detected in 17.6% (n = 3/17). TP53 mutation in 5.9% (n = 1/17). Gross Total, Subtotal, Partial resection and Biopsy were achieved in 17 (70.8%), 3 (12.5%), 3 (12.5%) and 1 (4.2%) of patients, respectively. 76% of cases (n = 16/21) experienced clinical worsening suddenly after surgery for different reasons, and mostly gradually recovered. For three cases no data was available. Adjuvant therapy was performed only for grades 2 and 3. At recurrence, surgery, radiosurgery, radiotherapy and chemotherapy were considered. The median Overall Survival from surgery was 219.25 months (95% CI, 188.83-249.67).</p><p><strong>Conclusions: </strong>We confirm that CPT can occur in adults and are mostly grade 1 tumors located in the ventricular system. The surgical approach is the gold standard, although 76% of clinical worsening occurred, often transient. Adjuvant treatment was limited to higher grade CPT; however, no consensus has already been achieved about adjuvant therapy.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":"1859-1866"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11920377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142770373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Federico Mainardi, Ferdinando Maggioni, Giorgio Zanchin
{"title":"Eponymous of migraine spectra of fortification: Vauban or Sanmicheli?","authors":"Federico Mainardi, Ferdinando Maggioni, Giorgio Zanchin","doi":"10.1007/s10072-025-08130-w","DOIUrl":"https://doi.org/10.1007/s10072-025-08130-w","url":null,"abstract":"<p><p>The most frequent type of aura preceding the migraine headache is the visual one. The visual perception of the migraineur can be variegated, the most common being a zig-zag pattern of luminous lines, the complete denomination of this phenomenon being \"fortification spectra of Vauban\". We investigated on original sources about the origin of this complex denomination and more in detail about the eponym and the priority on the construction of the Reinassance fortification. This kind of visual aura was compared to Reinassance fortifications by John Fothergill. (1712-1780). In 1870 Hubert Airy, reporting on his own attack of migraine with aura, compared the visual disturbance to a fortified town with its bastions of a colourful appearance. Moreover, realising the illusory nature of these images, he called them \"fortification spectra\". Later on, William Gowers (1845-1915), with reference to \".its projecting and reintrant angles bearing resemblance to the plan which the French engineer Vauban first described as the most effective for the defence of a fortress.\", introduced the term \"fortification of Vauban\", that since then has been largely used. Historically, these kinds of fortifications were first developed in early sixteen century in Italy by Michele Sanmicheli (1484-1559), a Venetian military engineer, when medieval tall courtains and circular towers were substituted with low and thicker walls and polygonal bastions, to face the destroying power of the new mobile siege guns. Only later Sébastien Le Prestre De Vauban (1633-1707), at the service of Louis XIV, the Sun King, greatly contributed to the development of this branch of military engineering. With reference to the visual aura, the eponym \"fortifications spectra of Vauban\" should be substituted for that \"of Sanmicheli\".</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"How far are we from bringing intensive care bundle for intracerebral hemorrhage into the real-world setting? A 5-year population based-study.","authors":"Paola Colantuono, Lucio D'Anna, Matteo Foschi, Michela Adipietro, Stefania Lancia, Leondino Mammarella, Simona Sacco, Raffaele Ornello","doi":"10.1007/s10072-025-08113-x","DOIUrl":"https://doi.org/10.1007/s10072-025-08113-x","url":null,"abstract":"<p><strong>Introduction: </strong>Comprehensive care bundles including rapid blood pressure management, anticoagulation reversal, neurosurgical consultation, control of blood glucose and body temperature, can improve short- and medium-term outcomes in patients with intracerebral hemorrhage (ICH). This study assessed how the acute management of ICH practices evolved in a real-world setting over five years characterized by global changes in ICH care.</p><p><strong>Methods: </strong>This study analysed ICH cases from a population-based stroke registry between 2018 and 2022. We collected demographic and clinical data, focusing on key parameters of ICH management, such as systolic blood pressure, anticoagulation reversal, neurosurgical referrals, blood glucose, and body temperature. We also examined yearly trends in control of parameters over time.</p><p><strong>Results: </strong>We included 460 patients with ICH (55.4% male, median age 79 years, interquartile range 69-85). At onset, 266 patients (57.8%) had high SBP (SBP ≥ 140 mmHg), 286 (70.3%) hyperglycemia (blood glucose ≥ 108 mg/dL), and 63 (17.3%) hyperpyrexia (body temperature ≥ 37.0*C). Anticoagulation was reversed in 21.4% of anticoagulated patients within 24 h. Neurosurgical referrals were made for 84.6% of patients while only 12.4% underwent surgery. From 2018 to 2022, anticoagulation reversal rates increased from 0 to 88.9% (p < 0.001), while neurosurgical referrals not followed by surgery decreased from 79.5 to 55.7% (p < 0.001).</p><p><strong>Conclusions: </strong>This real-world study demonstrates suboptimal management of key factors associated with ICH prognosis; nevertheless, it highlights improvement over time. There is a need for structured interventions to improve the timely and consistent application of simple yet effective measures yielding the potential to improve patients' outcomes.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lena Sauthoff, Maider Iza Achutegui, Lan Ye, Clara Niesmann, Florian Wegner, Matthias Höllerhage, Martin Klietz
{"title":"Systematic review of pharmacological treatment options for orthostatic tremor in prospective patient cohorts and randomized controlled trials.","authors":"Lena Sauthoff, Maider Iza Achutegui, Lan Ye, Clara Niesmann, Florian Wegner, Matthias Höllerhage, Martin Klietz","doi":"10.1007/s10072-025-08129-3","DOIUrl":"https://doi.org/10.1007/s10072-025-08129-3","url":null,"abstract":"<p><strong>Introduction: </strong>Orthostatic tremor is an infrequent movement disorder characterized by a high-frequency tremor manifesting primarily in the standing position. This condition can lead to relevant restrictions of mobility in everyday life and adversely affect the quality of life. The etiology has not been conclusively clarified. To date, there are few therapy studies of sufficient quality.</p><p><strong>Aim: </strong>The aim of the present literature analysis is to systematically evaluate the existing evidence of pharmacological therapies for orthostatic tremor.</p><p><strong>Materials and methods: </strong>This study searched for publications via PubMed and Google Scholar using the terms \"orthostatic tremor\" AND \"therapy\" and \"shaky legs syndrome\" AND \"therapy\". The main inclusion criteria were a subject number ≥ 5, pharmacological treatment approaches and the presence of a prospective experimental design.</p><p><strong>Results: </strong>The evaluation of the results indicated the most positive evidence for therapy with gabapentin. Additionally, other drugs such as perampanel and levodopa also showed positive outcomes regarding specific endpoints. In contrast, there is a lack of evidence supporting the efficacy of levetiracetam and botulinum toxin in the context of primary orthostatic tremor.</p><p><strong>Discussion and conclusion: </strong>According to current evidence, gabapentin is the drug with the most robust data. However, further studies are needed to support the evidence for different pharmacological therapeutic approaches for orthostatic tremor. Future investigations should emphasize larger sample sizes, placebo-controlled, double-blinded methodologies and a longer follow-up to be able to make more precise recommendations with greater generalizability.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
D Duran, P Arpaia, G D'Errico, L Grazzi, P Lanteri, N Moccaldi, A Raggi, R Robbio, E Visani
{"title":"State of the art in EEG signal features of mindfulness-based treatments for chronic pain.","authors":"D Duran, P Arpaia, G D'Errico, L Grazzi, P Lanteri, N Moccaldi, A Raggi, R Robbio, E Visani","doi":"10.1007/s10072-025-08145-3","DOIUrl":"https://doi.org/10.1007/s10072-025-08145-3","url":null,"abstract":"<p><p>A systematic review of electroencephalographic (EEG) correlates of Mindfulness- based treatment for chronic pain is presented. Recent technological advances have made EEG acquisition more accessible and also reliable. EEG monitoring before, during, and after treatment might support efficacy assessment and enable real- time adaptive intervention. The preliminary research extracted 131 papers from 6 scientific search engines. The application of the exclusion criteria led to the selection of 4 papers, indicating that the topic is still unexplored and further investigations are required. The collected papers exhibited great variability making challenging the comparison, nevertheless promising EEG correlates emerged. In particular, pain-related evoked potentials correlate with Mindfulness-Based treatment. EEG source analysis revealed the prevalent involvement of regions modulating emotional responses. In addition, higher baseline theta power was associated with greater improvement in depression when Mindfulness-based treatments are administered. This last result makes EEG also suitable for evaluating which patients can benefit most from mindfulness-based treatments.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Xindi Song, Yanan Wang, Wen Guo, Meng Liu, Yilun Deng, Ming Liu
{"title":"Time-varying association between blood pressure and malignant brain edema after large hemispheric infarction: a prospective cohort study.","authors":"Xindi Song, Yanan Wang, Wen Guo, Meng Liu, Yilun Deng, Ming Liu","doi":"10.1007/s10072-025-08147-1","DOIUrl":"https://doi.org/10.1007/s10072-025-08147-1","url":null,"abstract":"<p><strong>Aim: </strong>To investigate the time-varying association between blood pressure (BP) and malignant brain edema (MBE) risk after large hemispheric infarction (LHI).</p><p><strong>Methods: </strong>We prospectively enrolled LHI patients (CT hypodensity > 1/3 middle cerebral artery territory within 6 h or > 1/2 within 6-48 h of onset) from registry cohort. BP was recorded hourly and analyzed for 24 h and in two 12-hour epochs after onset: 1-12 h and 13-24 h. MBE was defined as neurological deterioration symptoms with midline shift ≥ 5 mm. Generalized estimating equation (GEE) compared BP patterns by MBE occurrence. Logistic regression and restricted cubic splines (RCS) assessed dose-response associations.</p><p><strong>Results: </strong>Among 414 included LHI patients (mean age 69 ± 14y, median onset-to-admission interval 3 h [interquartile range, IQR 2-5]), 117(28.3%) developed MBE with a median post-onset interval of 29 h (IQR 15-56). No significant difference in BP level was observed during 1-12 h. Systolic BP (SBP) was significantly higher in MBE group over time during 13-24 h (GEE P = 0.027). The 24-hour mean SBP exhibited a U-shaped association with MBE risk (nonlinear test, P = 0.029), with no significance in 1-12 h and a positive correlation between mean SBP in 13-24 h and MBE risk (aOR 1.02[1.00-1.04]). A threshold effect for 24-hour mean diastolic BP (DBP) was identified (non-linear test, P = 0.039), with DBP increase above 70 mmHg associated with higher MBE risk (aOR 1.03 [1.00-1.07]). Similarly, elevated mean DBP > 75mmHg during 1-12 h was associated with higher MBE risk.</p><p><strong>Conclusion: </strong>In LHI patients, 24-hour mean SBP demonstrated a U-shaped association with MBE risk, with no significant effect during 1-12 h and a positive linear correlation during 13-24 h after onset. DBP exhibited a threshold effect, primarily influencing MBE risk during 1-12 h after onset.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743530","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Emilio Albamonte, Adrea Lizio, Giorgia Coratti, Lorenzo Maggi, Elena Pegoraro, Marika Pane, Sonia Messina, Riccardo Masson, Adele D'Amico, Enrico Bertini, Antonella Pini, Federica Ricci, Tiziana Mongini, Claudio Bruno, Katia Patanella, Maria Sframeli, Claudia Dosi, Silvia Bonanno, Gaia Scarpini, Noemi Brolatti, Alice Zanolini, Chiara Bravetti, Maria Carmela Pera, Eugenio Maria Mercuri, Valeria Ada Sansone
{"title":"Patients on treatment with risdiplam in Italy: challenges in the interpretation of the real-world data.","authors":"Emilio Albamonte, Adrea Lizio, Giorgia Coratti, Lorenzo Maggi, Elena Pegoraro, Marika Pane, Sonia Messina, Riccardo Masson, Adele D'Amico, Enrico Bertini, Antonella Pini, Federica Ricci, Tiziana Mongini, Claudio Bruno, Katia Patanella, Maria Sframeli, Claudia Dosi, Silvia Bonanno, Gaia Scarpini, Noemi Brolatti, Alice Zanolini, Chiara Bravetti, Maria Carmela Pera, Eugenio Maria Mercuri, Valeria Ada Sansone","doi":"10.1007/s10072-025-08125-7","DOIUrl":"https://doi.org/10.1007/s10072-025-08125-7","url":null,"abstract":"<p><strong>Aims: </strong>(i) provide a snapshot from a large cohort of Italian patients with SMA on risdiplam in the real-world setting; (ii) identify any differences in the cohorts before and after commercial drug approval considering the different eligibility access criteria (iii) describe preliminary data on adherence to treatment and reasons for shifting from nusinersen to risdiplam.</p><p><strong>Methods: </strong>Charts from patients on risdiplam were retrospectively reviewed. Results were then compared between patients accessing the drug during an initial restricted compassionate use program (cohort 1) and those after commercial approval, with no restrictions (cohort 2). Side effects and adherence were recorded for both cohorts as well as data on shifters.</p><p><strong>Results: </strong>283 patients (median age: 22 years) were included. Only a minority were walkers. Respiratory and bulbar comorbidities were more severe in cohort 1 (58% non sitters) than in cohort 2 (52% sitters). 35% and 46% of patients from cohorts 1 and 2 shifted from nusinersen to risdiplam respectively. Adherence and safety profile were good in both cohorts.</p><p><strong>Conclusions: </strong>This is the largest cohort described so far providing insights on the characteristics of patients on risdiplam in the real world. The disability level and age were very different from those that had driven efficacy results in the trials. This may at least in part produce some evidence to account for the variable results reported so far in the realworld. Importantly, the safety profile was confirmed even in these more severely disabled and older patients compared to those in the trials.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143736068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}