Pub Date : 2025-01-01Epub Date: 2024-02-16DOI: 10.1017/cjn.2024.27
Archana B Netto, Niveditha Chandrahasa, Sheril S Koshy, Arun B Taly
Guillain-Barre syndrome (GBS) is the commonest cause of acute polyradiculoneuropathy that requires hospitalization. Many of these patients experience systemic and disease-related complications during its course. Notable among them is hyponatremia. Though recognized for decades, the precise incidence, prevalence, and mechanism of hyponatremia in GBS are not well known. Hyponatremia in GBS patients is associated with more severe in-hospital disease course, prolonged hospitalization, higher mortality, increased costs, and a greater number of other complications in the hospital and worse functional status at 6 months and at 1 year. Though there are several reports of low sodium associated with GBS, many have not included the exact temporal relationship of sodium or its serial values during GBS thereby underestimating the exact incidence, prevalence, and magnitude of the problem. Early detection, close monitoring, and better understanding of the pathophysiology of hyponatremia have therapeutic implications. We review the complexities of the relationship between hyponatremia and GBS with regard to its pathophysiology and treatment.
{"title":"Hyponatremia in Guillain-Barre Syndrome: A Review of Its Pathophysiology and Management.","authors":"Archana B Netto, Niveditha Chandrahasa, Sheril S Koshy, Arun B Taly","doi":"10.1017/cjn.2024.27","DOIUrl":"10.1017/cjn.2024.27","url":null,"abstract":"<p><p>Guillain-Barre syndrome (GBS) is the commonest cause of acute polyradiculoneuropathy that requires hospitalization. Many of these patients experience systemic and disease-related complications during its course. Notable among them is hyponatremia. Though recognized for decades, the precise incidence, prevalence, and mechanism of hyponatremia in GBS are not well known. Hyponatremia in GBS patients is associated with more severe in-hospital disease course, prolonged hospitalization, higher mortality, increased costs, and a greater number of other complications in the hospital and worse functional status at 6 months and at 1 year. Though there are several reports of low sodium associated with GBS, many have not included the exact temporal relationship of sodium or its serial values during GBS thereby underestimating the exact incidence, prevalence, and magnitude of the problem. Early detection, close monitoring, and better understanding of the pathophysiology of hyponatremia have therapeutic implications. We review the complexities of the relationship between hyponatremia and GBS with regard to its pathophysiology and treatment.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"9-19"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139742824","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}
Pub Date : 2025-01-01Epub Date: 2024-03-01DOI: 10.1017/cjn.2024.30
Christine Van Winssen, Andrea V Andrade, Danielle M Andrade, Jorge G Burneo, Sandrine de Ribaupierre, Elizabeth Donner, Ayman Hassan, George Ibrahim, Kevin C Jones, Lysa Boissé Lomax, Katherine Muir, Maryam N Nouri, Nikki Porter, Rajesh Ramachandrannair, Paul Raymond, James Rutka, Michelle J Shapiro, David A Steven, Darryl Swain, Taufik Valiante, Sharon Whiting, Robyn Whitney, Ivanna Yau, Tadeu A Fantaneanu
There are numerous challenges pertaining to epilepsy care across Ontario, including Epilepsy Monitoring Unit (EMU) bed pressures, surgical access and community supports. We sampled the current clinical, community and operational state of Ontario epilepsy centres and community epilepsy agencies post COVID-19 pandemic. A 44-item survey was distributed to all 11 district and regional adult and paediatric Ontario epilepsy centres. Qualitative responses were collected from community epilepsy agencies. Results revealed ongoing gaps in epilepsy care across Ontario, with EMU bed pressures and labour shortages being limiting factors. A clinical network advising the Ontario Ministry of Health will improve access to epilepsy care.
{"title":"Evaluating the Current State of Epilepsy Care in the Province of Ontario.","authors":"Christine Van Winssen, Andrea V Andrade, Danielle M Andrade, Jorge G Burneo, Sandrine de Ribaupierre, Elizabeth Donner, Ayman Hassan, George Ibrahim, Kevin C Jones, Lysa Boissé Lomax, Katherine Muir, Maryam N Nouri, Nikki Porter, Rajesh Ramachandrannair, Paul Raymond, James Rutka, Michelle J Shapiro, David A Steven, Darryl Swain, Taufik Valiante, Sharon Whiting, Robyn Whitney, Ivanna Yau, Tadeu A Fantaneanu","doi":"10.1017/cjn.2024.30","DOIUrl":"10.1017/cjn.2024.30","url":null,"abstract":"<p><p>There are numerous challenges pertaining to epilepsy care across Ontario, including Epilepsy Monitoring Unit (EMU) bed pressures, surgical access and community supports. We sampled the current clinical, community and operational state of Ontario epilepsy centres and community epilepsy agencies post COVID-19 pandemic. A 44-item survey was distributed to all 11 district and regional adult and paediatric Ontario epilepsy centres. Qualitative responses were collected from community epilepsy agencies. Results revealed ongoing gaps in epilepsy care across Ontario, with EMU bed pressures and labour shortages being limiting factors. A clinical network advising the Ontario Ministry of Health will improve access to epilepsy care.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"132-134"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139998348","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}
Pub Date : 2025-01-01Epub Date: 2024-04-01DOI: 10.1017/cjn.2024.46
Galit Kleiner, Farooq Ismail, Omar Khan, Christopher Pilieci, Linda Truong, Chris Boulias
Paratonia is a form of hypertonia characterized by an inability to relax muscles in the setting of cognitive impairment. Paratonia results in pain, refusal of care, and caregiver burden. We sent surveys to 67 Canadian physiatrists and neurologists regarding their experience treating paratonia with botulinum toxin A (BoNT-A). Twenty-seven survey respondents were included in the analysis. Thirteen percent of survey respondents treating paratonia with BoNT-A reported a significant clinically relevant improvement; 74% endorsed a moderately clinically relevant improvement; 13% endorsed a slight clinically relevant improvement. Ninety percent of survey respondents endorsed significant barriers in treating paratonia with BoNT-A.
{"title":"Survey of Canadian Physician Use of Botulinum Toxin A (BoNT-A) for Paratonia: Real World Insights.","authors":"Galit Kleiner, Farooq Ismail, Omar Khan, Christopher Pilieci, Linda Truong, Chris Boulias","doi":"10.1017/cjn.2024.46","DOIUrl":"10.1017/cjn.2024.46","url":null,"abstract":"<p><p>Paratonia is a form of hypertonia characterized by an inability to relax muscles in the setting of cognitive impairment. Paratonia results in pain, refusal of care, and caregiver burden. We sent surveys to 67 Canadian physiatrists and neurologists regarding their experience treating paratonia with botulinum toxin A (BoNT-A). Twenty-seven survey respondents were included in the analysis. Thirteen percent of survey respondents treating paratonia with BoNT-A reported a significant clinically relevant improvement; 74% endorsed a moderately clinically relevant improvement; 13% endorsed a slight clinically relevant improvement. Ninety percent of survey respondents endorsed significant barriers in treating paratonia with BoNT-A.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"141-144"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140332351","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}
Pub Date : 2025-01-01Epub Date: 2024-02-20DOI: 10.1017/cjn.2024.18
Ava Mehdipour, Lizabeth Teshler, Vanina Dal Bello-Haas, Vanessa Bouchard, Ayse Kuspinar
The objective of this study was to translate the Preference-Based Amyotrophic Lateral Sclerosis Scale to French-Canadian. After the scale underwent forward and back translations, the expert committee examined the translated versions and found minor grammatical errors and suggested idioms to be changed to better represent French-Canadian language. Cognitive debriefing interviews were carried out to assess the pre-final version for clarity, and minor changes were made. Consensus from the expert committee and people with amyotrophic lateral sclerosis on the measure's clarity, word choice, and meaning were achieved, resulting in the final French version of the Preference-Based Amyotrophic Lateral Sclerosis Scale.
{"title":"Translation of the Preference-Based Amyotrophic Lateral Sclerosis Scale into French.","authors":"Ava Mehdipour, Lizabeth Teshler, Vanina Dal Bello-Haas, Vanessa Bouchard, Ayse Kuspinar","doi":"10.1017/cjn.2024.18","DOIUrl":"10.1017/cjn.2024.18","url":null,"abstract":"<p><p>The objective of this study was to translate the Preference-Based Amyotrophic Lateral Sclerosis Scale to French-Canadian. After the scale underwent forward and back translations, the expert committee examined the translated versions and found minor grammatical errors and suggested idioms to be changed to better represent French-Canadian language. Cognitive debriefing interviews were carried out to assess the pre-final version for clarity, and minor changes were made. Consensus from the expert committee and people with amyotrophic lateral sclerosis on the measure's clarity, word choice, and meaning were achieved, resulting in the final French version of the Preference-Based Amyotrophic Lateral Sclerosis Scale.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"129-131"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139906970","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}
Pub Date : 2025-01-01Epub Date: 2024-03-11DOI: 10.1017/cjn.2024.34
Emma Stefanie Campisi, Omar Khan, Anne Marie Reet Agur, Rajiv Reebye
{"title":"An Anatomical Perspective to Botulinum Toxin Injection of Flexor Digitorum Profundus.","authors":"Emma Stefanie Campisi, Omar Khan, Anne Marie Reet Agur, Rajiv Reebye","doi":"10.1017/cjn.2024.34","DOIUrl":"10.1017/cjn.2024.34","url":null,"abstract":"","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"145-146"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140095225","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}
Pub Date : 2025-01-01Epub Date: 2024-03-11DOI: 10.1017/cjn.2024.17
Aqeel Almutairi, Tommy Lik Hang Chan
{"title":"Cough Headache Responsive to Occipital Nerve Blockade.","authors":"Aqeel Almutairi, Tommy Lik Hang Chan","doi":"10.1017/cjn.2024.17","DOIUrl":"10.1017/cjn.2024.17","url":null,"abstract":"","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"154-155"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140095227","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}
Pub Date : 2025-01-01Epub Date: 2024-03-06DOI: 10.1017/cjn.2024.29
Nabeela Nathoo, Kevin D Chodnicki, Jamie J Van Gompel, Karl N Krecke, Aivi T Nguyen, Michel Toledano, Orhun H Kantarci
{"title":"Atypical Bilateral Idiopathic Inflammatory Cavernous Sinus Syndrome Responsive to Cyclophosphamide.","authors":"Nabeela Nathoo, Kevin D Chodnicki, Jamie J Van Gompel, Karl N Krecke, Aivi T Nguyen, Michel Toledano, Orhun H Kantarci","doi":"10.1017/cjn.2024.29","DOIUrl":"10.1017/cjn.2024.29","url":null,"abstract":"","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"159-161"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140041042","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}
Pub Date : 2025-01-01Epub Date: 2024-03-22DOI: 10.1017/cjn.2024.42
Lalith Satkunam, Sean P Dukelow, Jaime Yu, Stephen McNeil, Huong Luu, Karen J B Martins, Khanh Vu, Phuong Uyen Nguyen, Lawrence Richer, Tyler Williamson, Scott W Klarenbach
Background: Limited evidence exists regarding care pathways for stroke survivors who do and do not receive poststroke spasticity (PSS) treatment.
Methods: Administrative data was used to identify adults who experienced a stroke and sought acute care between 2012 and 2017 in Alberta, Canada. Pathways of stroke care within the health care system were determined among those who initiated PSS treatment (PSS treatment group: outpatient pharmacy dispensation of an anti-spastic medication, focal chemo-denervation injection, or a spasticity tertiary clinic visit) and those who did not (non-PSS treatment group). Time from the stroke event until spasticity treatment initiation, and setting where treatment was initiated were reported. Descriptive statistics were performed.
Results: Health care settings within the pathways of stroke care that the PSS (n = 1,079) and non-PSS (n = 22,922) treatment groups encountered were the emergency department (86 and 84%), acute inpatient care (80 and 69%), inpatient rehabilitation (40 and 12%), and long-term care (19 and 13%), respectively. PSS treatment was initiated a median of 291 (interquartile range 625) days after the stroke event, and most often in the community when patients were residing at home (45%), followed by "other" settings (22%), inpatient rehabilitation (18%), long-term care (11%), and acute inpatient care (4%).
Conclusions: To our knowledge, this is the first population based cohort study describing pathways of care among adults with stroke who subsequently did or did not initiate spasticity treatment. Areas for improvement in care may include strategies for earlier identification and treatment of PSS.
{"title":"Poststroke Care Pathways and Spasticity Treatment: A Retrospective Study in Alberta.","authors":"Lalith Satkunam, Sean P Dukelow, Jaime Yu, Stephen McNeil, Huong Luu, Karen J B Martins, Khanh Vu, Phuong Uyen Nguyen, Lawrence Richer, Tyler Williamson, Scott W Klarenbach","doi":"10.1017/cjn.2024.42","DOIUrl":"10.1017/cjn.2024.42","url":null,"abstract":"<p><strong>Background: </strong>Limited evidence exists regarding care pathways for stroke survivors who do and do not receive poststroke spasticity (PSS) treatment.</p><p><strong>Methods: </strong>Administrative data was used to identify adults who experienced a stroke and sought acute care between 2012 and 2017 in Alberta, Canada. Pathways of stroke care within the health care system were determined among those who initiated PSS treatment (PSS treatment group: outpatient pharmacy dispensation of an anti-spastic medication, focal chemo-denervation injection, or a spasticity tertiary clinic visit) and those who did not (non-PSS treatment group). Time from the stroke event until spasticity treatment initiation, and setting where treatment was initiated were reported. Descriptive statistics were performed.</p><p><strong>Results: </strong>Health care settings within the pathways of stroke care that the PSS (<i>n</i> = 1,079) and non-PSS (<i>n</i> = 22,922) treatment groups encountered were the emergency department (86 and 84%), acute inpatient care (80 and 69%), inpatient rehabilitation (40 and 12%), and long-term care (19 and 13%), respectively. PSS treatment was initiated a median of 291 (interquartile range 625) days after the stroke event, and most often in the community when patients were residing at home (45%), followed by \"other\" settings (22%), inpatient rehabilitation (18%), long-term care (11%), and acute inpatient care (4%).</p><p><strong>Conclusions: </strong>To our knowledge, this is the first population based cohort study describing pathways of care among adults with stroke who subsequently did or did not initiate spasticity treatment. Areas for improvement in care may include strategies for earlier identification and treatment of PSS.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"75-84"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140186407","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}
Pub Date : 2025-01-01Epub Date: 2024-03-25DOI: 10.1017/cjn.2024.43
Pooja Mailankody, Nitish Kamble, Amitabh Bhattacharya, G S Shubha Bhat, Thamodharan Arumugam, K Thennarasu, Rashmi Arasappa, Shivarama Varambally, Ravi Yadav, Pramod Kumar Pal
Objective: We aimed to evaluate the effect of yoga on motor and non-motor symptoms and cortical excitability in patients with Parkinson's disease (PD).
Methods: We prospectively evaluated 17 patients with PD at baseline, after one month of conventional care, and after one month of supervised yoga sessions. The motor and non-motor symptoms were evaluated using the Unified Parkinson's disease Rating Scale (motor part III), Hoehn and Yahr stage, Montreal Cognitive Assessment, Hamilton depression rating scale, Hamilton anxiety rating scale, non-motor symptoms questionnaire and World Health Organization quality of life questionnaire. Transcranial magnetic stimulation was used to record resting motor threshold, central motor conduction time, ipsilateral silent period (iSP), contralateral silent period (cSP), short interval intracortical inhibition (SICI), and intracortical facilitation.
Results: The mean age of the patients was 55.5 ± 10.8 years, with a mean duration of illness of 4.0 ± 2.5 years. The postural stability of the patients significantly improved following yoga (0.59 ± 0.5 to 0.18 ± 0.4, p = 0.039). There was a significant reduction in the cSP from baseline (138.07 ± 27.5 ms) to 4 weeks of yoga therapy (116.94 ± 18.2 ms, p = 0.004). In addition, a significant reduction in SICI was observed after four weeks of yoga therapy (0.22 ± 0.10) to (0.46 ± 0.23), p = 0.004).
Conclusion: Yoga intervention can significantly improve postural stability in patients with PD. A significant reduction of cSP and SICI suggests a reduction in GABAergic neurotransmission following yoga therapy that may underlie the improvement observed in postural stability.
{"title":"Yoga as an Add-on Therapy in Parkinson's Disease: A Single Group Open-label Trial.","authors":"Pooja Mailankody, Nitish Kamble, Amitabh Bhattacharya, G S Shubha Bhat, Thamodharan Arumugam, K Thennarasu, Rashmi Arasappa, Shivarama Varambally, Ravi Yadav, Pramod Kumar Pal","doi":"10.1017/cjn.2024.43","DOIUrl":"10.1017/cjn.2024.43","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to evaluate the effect of yoga on motor and non-motor symptoms and cortical excitability in patients with Parkinson's disease (PD).</p><p><strong>Methods: </strong>We prospectively evaluated 17 patients with PD at baseline, after one month of conventional care, and after one month of supervised yoga sessions. The motor and non-motor symptoms were evaluated using the Unified Parkinson's disease Rating Scale (motor part III), Hoehn and Yahr stage, Montreal Cognitive Assessment, Hamilton depression rating scale, Hamilton anxiety rating scale, non-motor symptoms questionnaire and World Health Organization quality of life questionnaire. Transcranial magnetic stimulation was used to record resting motor threshold, central motor conduction time, ipsilateral silent period (iSP), contralateral silent period (cSP), short interval intracortical inhibition (SICI), and intracortical facilitation.</p><p><strong>Results: </strong>The mean age of the patients was 55.5 ± 10.8 years, with a mean duration of illness of 4.0 ± 2.5 years. The postural stability of the patients significantly improved following yoga (0.59 ± 0.5 to 0.18 ± 0.4, <i>p</i> = 0.039). There was a significant reduction in the cSP from baseline (138.07 ± 27.5 ms) to 4 weeks of yoga therapy (116.94 ± 18.2 ms, <i>p</i> = 0.004). In addition, a significant reduction in SICI was observed after four weeks of yoga therapy (0.22 ± 0.10) to (0.46 ± 0.23), <i>p</i> = 0.004).</p><p><strong>Conclusion: </strong>Yoga intervention can significantly improve postural stability in patients with PD. A significant reduction of cSP and SICI suggests a reduction in GABAergic neurotransmission following yoga therapy that may underlie the improvement observed in postural stability.</p><p><strong>Clinicaltrialsgov identifier: </strong>CTRI/2019/02/017564.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"102-109"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140208368","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}
Pub Date : 2025-01-01Epub Date: 2024-03-27DOI: 10.1017/cjn.2024.49
Isabelle Côté, Victoria Hodgkinson, Marianne Nury, Louis Bastenier-Boutin, Xavier Rodrigue
Background: Spinal muscular atrophy (SMA) is a progressive genetic disorder characterized by muscle weakness ultimately leading to pulmonary impairments that can be fatal. The recent approval of nusinersen, a disease-modifying therapy, substantially changed the prognosis for patients, particularly in children. However, real-world evidence about its long-term effectiveness in adults remains limited. This study aimed to document longitudinal data on motor function, pulmonary function and patient-reported outcome measures of Canadian adults with SMA type 2 and 3 treated with nusinersen.
Methods: Outcomes from 17 patients were collected at the Institut de réadaptation en déficience physique de Québec during routine clinical visits over 36 months post nusinersen treatment, using the Hammersmith Functional Motor Scale Expanded for SMA (HFMSE), Revised Upper Limb Module (RULM), 6-Minute Walk Test (6MWT), Children's Hospital of Philadelphia Adult Test of Neuromuscular Disorders (CHOP-ATEND), SMA functional rating scale (SMAFRS), pulmonary function testing and subjective changes reported by patients.
Results: After 36 months, 9 patients showed motor function improvement. Changes beyond the minimal clinically important difference were seen for four patients on the HFMSE, four patients on the RULM and five patients on the 6MWT. Pulmonary function remained stable for most subjects. Subjective positive changes were reported in 88% of patients and five patients showed improvement in the SMAFRS.
Conclusion: This real-world study demonstrates the positive effects of nusinersen in adults with SMA types 2 and 3. Although stabilizing the patient's condition is considered therapeutic success, this study shows an improvement in motor function and subjective gains in several patients.
{"title":"A Real-World Study of Nusinersen Effects in Adults with Spinal Muscular Atrophy Type 2 and 3.","authors":"Isabelle Côté, Victoria Hodgkinson, Marianne Nury, Louis Bastenier-Boutin, Xavier Rodrigue","doi":"10.1017/cjn.2024.49","DOIUrl":"10.1017/cjn.2024.49","url":null,"abstract":"<p><strong>Background: </strong>Spinal muscular atrophy (SMA) is a progressive genetic disorder characterized by muscle weakness ultimately leading to pulmonary impairments that can be fatal. The recent approval of nusinersen, a disease-modifying therapy, substantially changed the prognosis for patients, particularly in children. However, real-world evidence about its long-term effectiveness in adults remains limited. This study aimed to document longitudinal data on motor function, pulmonary function and patient-reported outcome measures of Canadian adults with SMA type 2 and 3 treated with nusinersen.</p><p><strong>Methods: </strong>Outcomes from 17 patients were collected at the Institut de réadaptation en déficience physique de Québec during routine clinical visits over 36 months post nusinersen treatment, using the Hammersmith Functional Motor Scale Expanded for SMA (HFMSE), Revised Upper Limb Module (RULM), 6-Minute Walk Test (6MWT), Children's Hospital of Philadelphia Adult Test of Neuromuscular Disorders (CHOP-ATEND), SMA functional rating scale (SMAFRS), pulmonary function testing and subjective changes reported by patients.</p><p><strong>Results: </strong>After 36 months, 9 patients showed motor function improvement. Changes beyond the minimal clinically important difference were seen for four patients on the HFMSE, four patients on the RULM and five patients on the 6MWT. Pulmonary function remained stable for most subjects. Subjective positive changes were reported in 88% of patients and five patients showed improvement in the SMAFRS.</p><p><strong>Conclusion: </strong>This real-world study demonstrates the positive effects of nusinersen in adults with SMA types 2 and 3. Although stabilizing the patient's condition is considered therapeutic success, this study shows an improvement in motor function and subjective gains in several patients.</p>","PeriodicalId":56134,"journal":{"name":"Canadian Journal of Neurological Sciences","volume":" ","pages":"119-128"},"PeriodicalIF":2.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140295383","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}