Pub Date : 2024-09-17DOI: 10.1177/13524585241274580
Ellen O’Donnell, Allison Schuette, Michael Waltz, Gregory Aaen, Leslie Benson, Mark Gorman, Timothy Lotze, Soe Mar, Jayne Ness, Moses Rodriguez, Jan-Mendelt Tillema, Teri Schreiner, Yolanda Wheeler, T Charles Casper, Tanuja Chitnis
Background and Objectives:This survey study aimed to (1) identify patient/family research priorities in pediatric-onset multiple sclerosis (POMS), and (2) delineate optimized methods for research study/clinical trials design, engagement, and implementation.Methods:Participants were as follows: (1) parents of a child (<18 years) with POMS enrolled in a national registry, (2) adolescents (13–17 years) with POMS in the registry, and (3) adults (18–40 years) with POMS receiving care at a registry affiliated clinic. Of 293 eligible participants, 192 completed surveys.Results:Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition.Conclusions:Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.
{"title":"Patient and family views on research priorities and design of clinical trials and research studies in pediatric multiple sclerosis","authors":"Ellen O’Donnell, Allison Schuette, Michael Waltz, Gregory Aaen, Leslie Benson, Mark Gorman, Timothy Lotze, Soe Mar, Jayne Ness, Moses Rodriguez, Jan-Mendelt Tillema, Teri Schreiner, Yolanda Wheeler, T Charles Casper, Tanuja Chitnis","doi":"10.1177/13524585241274580","DOIUrl":"https://doi.org/10.1177/13524585241274580","url":null,"abstract":"Background and Objectives:This survey study aimed to (1) identify patient/family research priorities in pediatric-onset multiple sclerosis (POMS), and (2) delineate optimized methods for research study/clinical trials design, engagement, and implementation.Methods:Participants were as follows: (1) parents of a child (<18 years) with POMS enrolled in a national registry, (2) adolescents (13–17 years) with POMS in the registry, and (3) adults (18–40 years) with POMS receiving care at a registry affiliated clinic. Of 293 eligible participants, 192 completed surveys.Results:Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition.Conclusions:Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142256965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-14DOI: 10.1177/13524585241275013
Giuseppina Pilloni, Matthew Lustberg, Martin Malik, Charles Feinberg, Abhishek Datta, Marom Bikson, Josef Gutman, Lauren Krupp, Leigh Charvet
Background:Many individuals with progressive multiple sclerosis (PMS) are challenged by reduced manual dexterity and limited rehabilitation options. Transcranial direct current stimulation (tDCS) during motor training can improve rehabilitation outcomes. We developed a protocol for remotely supervising tDCS to deliver sessions of stimulation paired with training at home.Objective:This study evaluated the effectiveness of at-home tDCS paired with manual dexterity training for individuals with PMS.Methods:Sixty-five right-hand dominant participants with PMS and hand impairment were randomized to receive either active or sham M1-SO tDCS paired with manual dexterity training over 4 weeks. Clinical outcomes were measured by the changes in Nine-Hole Peg Test (9-HPT) and Dellon-Modified-Moberg-Pick-Up Test (DMMPUT).Results:The intervention had high rates of adherence and completion (98% of participants completed at least 18 of 20 sessions). The active tDCS group demonstrated significant improvement for the left hand compared with baseline in 9-HPT (−5.85 ± 6.19 vs −4.23 ± 4.34, p = 0.049) and DMMPUT (−10.62 ± 8.46 vs −8.97 ± 6.18, p = 0.049). The active tDCS group reported improvements in multiple sclerosis (MS)-related quality of life (mean increase: 5.93 ± 13.04 vs −0.05 ± −8.27; p = 0.04).Conclusion:At-home tDCS paired with manual dexterity training is effective for individuals with PMS, with M1-SO tDCS enhancing training outcomes and offering a promising intervention for improving and preserving hand dexterity.
{"title":"Hand functioning in progressive multiple sclerosis improves with tDCS added to daily exercises: A home-based randomized, double-blinded, sham-controlled clinical trial","authors":"Giuseppina Pilloni, Matthew Lustberg, Martin Malik, Charles Feinberg, Abhishek Datta, Marom Bikson, Josef Gutman, Lauren Krupp, Leigh Charvet","doi":"10.1177/13524585241275013","DOIUrl":"https://doi.org/10.1177/13524585241275013","url":null,"abstract":"Background:Many individuals with progressive multiple sclerosis (PMS) are challenged by reduced manual dexterity and limited rehabilitation options. Transcranial direct current stimulation (tDCS) during motor training can improve rehabilitation outcomes. We developed a protocol for remotely supervising tDCS to deliver sessions of stimulation paired with training at home.Objective:This study evaluated the effectiveness of at-home tDCS paired with manual dexterity training for individuals with PMS.Methods:Sixty-five right-hand dominant participants with PMS and hand impairment were randomized to receive either active or sham M1-SO tDCS paired with manual dexterity training over 4 weeks. Clinical outcomes were measured by the changes in Nine-Hole Peg Test (9-HPT) and Dellon-Modified-Moberg-Pick-Up Test (DMMPUT).Results:The intervention had high rates of adherence and completion (98% of participants completed at least 18 of 20 sessions). The active tDCS group demonstrated significant improvement for the left hand compared with baseline in 9-HPT (−5.85 ± 6.19 vs −4.23 ± 4.34, p = 0.049) and DMMPUT (−10.62 ± 8.46 vs −8.97 ± 6.18, p = 0.049). The active tDCS group reported improvements in multiple sclerosis (MS)-related quality of life (mean increase: 5.93 ± 13.04 vs −0.05 ± −8.27; p = 0.04).Conclusion:At-home tDCS paired with manual dexterity training is effective for individuals with PMS, with M1-SO tDCS enhancing training outcomes and offering a promising intervention for improving and preserving hand dexterity.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142256966","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
BACKGROUNDEvidence on the impact of dimethyl fumarate (DMF) during pregnancy in women with multiple sclerosis (MS) is limited.OBJECTIVESTo investigate disease activity and pregnancy outcomes in a retrospective cohort of women exposed to DMF in early pregnancy.METHODSWomen discontinuing DMF after pregnancy confirmation were identified from 29 Italian MS Centers. Disease activity 12 months before conception, during pregnancy, and 12 months postpartum were recorded, exploring reactivation predictors. Pregnancy and fetal outcomes were assessed.RESULTSThe study analyzed 137 pregnancies (12 pregnancy losses, 125 live births) from 137 women (mean age 32.9 ± 4.7 years), discontinuing DMF within a median (interquartile range (IQR)) interval of 4.9 (3.7-5.7) weeks from conception. In live birth pregnancies, annualized relapse rate (ARR) significantly decreased during pregnancy (ARR = 0.07, 95% confidence interval (CI): 0.03-0.14, p = 0.021) compared to pre-conception (ARR = 0.21 (95% CI: 0.14-0.30)) and increased postpartum ((ARR = 0.22 (95% CI: 0.15-0.32), p = 0.006). Median time to first relapse (TTFR) was 3.16 (IQR: 1:87-5.42) months. Higher pre-conception relapse number (hazard ratio (HR) = 2.33, 95% CI: 1.08-5.02) and Expanded Disability Status Scale (EDSS; HR = 1.81, 95% CI: 1.17-2.74) were associated with shorter TTFR, while treatment resumption with longer TTFR (HR = 0.29, 95% CI: 0.11-0.74). Fetal outcomes were unaffected by DMF exposure.CONCLUSIONDMF discontinuation does not increase relapse risk during pregnancy. Early therapy restart prevents postpartum relapses. Early DMF exposure shows no adverse fetal outcomes.
{"title":"Maternal and fetal outcomes in an Italian multicentric cohort of women with multiple sclerosis exposed to dimethyl fumarate during pregnancy.","authors":"Doriana Landi,Silvia Bartolomeo,Francesca Bovis,Maria Pia Amato,Simona Bonavita,Giovanna Borriello,Maria Buccafusca,Sebastiano Bucello,Paola Cavalla,Maria Cellerino,Diego Centonze,Eleonora Cocco,Antonella Conte,Antonio Cortese,Emanuele D'Amico,Massimiliano Di Filippo,Renato Docimo,Roberta Fantozzi,Elisabetta Ferraro,Massimo Filippi,Matteo Foschi,Antonio Gallo,Franco Granella,Antonio Ianniello,Roberta Lanzillo,Lorena Lorefice,Matteo Lucchini,Giacomo Lus,Giorgia Mataluni,Massimiliano Mirabella,Lucia Moiola,Francesca Napoli,Carolina Gabri Nicoletti,Francesco Patti,Paolo Ragonese,Sabrina Realmuto,Giuseppe Schirò,Elisabetta Signoriello,Leonardo Sinisi,Maria Laura Stromillo,Valentina Tomassini,Domizia Vecchio,Maria Pia Sormani,Girolama Alessandra Marfia","doi":"10.1177/13524585241274600","DOIUrl":"https://doi.org/10.1177/13524585241274600","url":null,"abstract":"BACKGROUNDEvidence on the impact of dimethyl fumarate (DMF) during pregnancy in women with multiple sclerosis (MS) is limited.OBJECTIVESTo investigate disease activity and pregnancy outcomes in a retrospective cohort of women exposed to DMF in early pregnancy.METHODSWomen discontinuing DMF after pregnancy confirmation were identified from 29 Italian MS Centers. Disease activity 12 months before conception, during pregnancy, and 12 months postpartum were recorded, exploring reactivation predictors. Pregnancy and fetal outcomes were assessed.RESULTSThe study analyzed 137 pregnancies (12 pregnancy losses, 125 live births) from 137 women (mean age 32.9 ± 4.7 years), discontinuing DMF within a median (interquartile range (IQR)) interval of 4.9 (3.7-5.7) weeks from conception. In live birth pregnancies, annualized relapse rate (ARR) significantly decreased during pregnancy (ARR = 0.07, 95% confidence interval (CI): 0.03-0.14, p = 0.021) compared to pre-conception (ARR = 0.21 (95% CI: 0.14-0.30)) and increased postpartum ((ARR = 0.22 (95% CI: 0.15-0.32), p = 0.006). Median time to first relapse (TTFR) was 3.16 (IQR: 1:87-5.42) months. Higher pre-conception relapse number (hazard ratio (HR) = 2.33, 95% CI: 1.08-5.02) and Expanded Disability Status Scale (EDSS; HR = 1.81, 95% CI: 1.17-2.74) were associated with shorter TTFR, while treatment resumption with longer TTFR (HR = 0.29, 95% CI: 0.11-0.74). Fetal outcomes were unaffected by DMF exposure.CONCLUSIONDMF discontinuation does not increase relapse risk during pregnancy. Early therapy restart prevents postpartum relapses. Early DMF exposure shows no adverse fetal outcomes.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142183114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-11DOI: 10.1177/13524585241275491
Sydney Lee,Ruth Ann Marrie,Giulia Fadda,Mark S Freedman,Liesly Lee,Alexandra Muccilli,Manav V Vyas,Andrea Konig,Dalia L Rotstein
BACKGROUNDIt is unknown whether people with aquaporin-4 antibody positive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD) experience a prodrome, although a few cases report AQP4 + serology up to 16 years before the first attack.OBJECTIVESTo evaluate whether individuals with AQP4-IgG + NMOSD have prodromal neurologic symptoms preceding the first attack.METHODSWe reviewed medical records of participants meeting the 2015 diagnostic criteria for AQP4-IgG + NMOSD from four demyelinating disease centres in the Canadian NMOSD cohort study CANOPTICS. We searched for neurologic symptoms occurring at least 30 days before the first attack.RESULTSOf 116 participants with NMOSD, 17 (14.7%) had prodromal neurologic symptoms. The median age was 48 years (range 25-83) at first attack; 16 (94.1%) were female. Participants presented with numbness/tingling (n = 9), neuropathic pain (n = 5), visual disturbance (n = 4), tonic spasms (n = 2), Lhermitte sign (n = 2), severe headache (n = 2), incoordination (n = 2), weakness (n = 1), psychosis (n = 1) or seizure (n = 1). Of eight who underwent magnetic resonance imaging (MRI) brain, orbits and/or spinal cord, five had T2 lesions. Within 1.5-245 months (median 14) from the onset of prodromal neurologic symptoms, participants experienced their first NMOSD attack.CONCLUSIONSOne in seven people with NMOSD experienced neurologic symptoms before their first attack. Further investigation of a possible NMOSD prodrome is warranted.
{"title":"Is there a prodrome to NMOSD? An investigation of neurologic symptoms preceding the first NMOSD attack.","authors":"Sydney Lee,Ruth Ann Marrie,Giulia Fadda,Mark S Freedman,Liesly Lee,Alexandra Muccilli,Manav V Vyas,Andrea Konig,Dalia L Rotstein","doi":"10.1177/13524585241275491","DOIUrl":"https://doi.org/10.1177/13524585241275491","url":null,"abstract":"BACKGROUNDIt is unknown whether people with aquaporin-4 antibody positive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD) experience a prodrome, although a few cases report AQP4 + serology up to 16 years before the first attack.OBJECTIVESTo evaluate whether individuals with AQP4-IgG + NMOSD have prodromal neurologic symptoms preceding the first attack.METHODSWe reviewed medical records of participants meeting the 2015 diagnostic criteria for AQP4-IgG + NMOSD from four demyelinating disease centres in the Canadian NMOSD cohort study CANOPTICS. We searched for neurologic symptoms occurring at least 30 days before the first attack.RESULTSOf 116 participants with NMOSD, 17 (14.7%) had prodromal neurologic symptoms. The median age was 48 years (range 25-83) at first attack; 16 (94.1%) were female. Participants presented with numbness/tingling (n = 9), neuropathic pain (n = 5), visual disturbance (n = 4), tonic spasms (n = 2), Lhermitte sign (n = 2), severe headache (n = 2), incoordination (n = 2), weakness (n = 1), psychosis (n = 1) or seizure (n = 1). Of eight who underwent magnetic resonance imaging (MRI) brain, orbits and/or spinal cord, five had T2 lesions. Within 1.5-245 months (median 14) from the onset of prodromal neurologic symptoms, participants experienced their first NMOSD attack.CONCLUSIONSOne in seven people with NMOSD experienced neurologic symptoms before their first attack. Further investigation of a possible NMOSD prodrome is warranted.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142183115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-11DOI: 10.1177/13524585241277400
Roee Holtzer,Frederick W Foley,Robert W Motl,Mark E Wagshul,Manuel E Hernandez,Michael L Lipton,Mary Ann Picone,Meltem Izzetoglu
OBJECTIVEWe examined whether brain hemodynamic responses, gait, and cognitive performances under single- and dual-task conditions predict falls during longitudinal follow-up in older adults with multiple sclerosis (OAMS) with relapsing-remitting and progressive subtypes.METHODSParticipants with relapsing-remitting (n = 53, mean age = 65.02 ± 4.17 years, %female = 75.5) and progressive (n = 28, mean age = 64.64 ± 4.31 years, %female = 50) multiple sclerosis (MS) subtypes completed a dual-task-walking paradigm and reported falls during longitudinal follow-up using a monthly structured telephone interview. We used functional near-infrared spectroscopy (fNIRS) to assess oxygenated hemoglobin (HbO) in the prefrontal cortex during active walking and while performing a cognitive test under single- and dual-task conditions.RESULTSAdjusted general estimating equations models indicated that higher HbO under dual-task walking was significantly associated with a reduction in the odds of reporting falls among participants with relapsing-remitting (odds ratio (OR) = 0.472, p = 0.004, 95% confidence interval (CI) = 0.284-0.785), but not progressive (OR = 1.056, p = 0.792, 95% CI = 0.703-1.588) MS. In contrast, faster stride velocity under dual-task walking was significantly associated with a reduction in the odds of reporting falls among progressive (OR = 0.658, p = 0.004, 95% CI = 0.495-0.874), but not relapsing-remitting (OR = 0.998, p = 0.995, 95% CI = 0.523-1.905) MS.CONCLUSIONFindings suggest that higher prefrontal cortex activation levels during dual-task walking, which may represent compensatory reallocation of brain resources, provide protection against falls for OAMS with relapsing-remitting subtype.
{"title":"Brain hemodynamic responses and fall prediction in older adults with multiple sclerosis.","authors":"Roee Holtzer,Frederick W Foley,Robert W Motl,Mark E Wagshul,Manuel E Hernandez,Michael L Lipton,Mary Ann Picone,Meltem Izzetoglu","doi":"10.1177/13524585241277400","DOIUrl":"https://doi.org/10.1177/13524585241277400","url":null,"abstract":"OBJECTIVEWe examined whether brain hemodynamic responses, gait, and cognitive performances under single- and dual-task conditions predict falls during longitudinal follow-up in older adults with multiple sclerosis (OAMS) with relapsing-remitting and progressive subtypes.METHODSParticipants with relapsing-remitting (n = 53, mean age = 65.02 ± 4.17 years, %female = 75.5) and progressive (n = 28, mean age = 64.64 ± 4.31 years, %female = 50) multiple sclerosis (MS) subtypes completed a dual-task-walking paradigm and reported falls during longitudinal follow-up using a monthly structured telephone interview. We used functional near-infrared spectroscopy (fNIRS) to assess oxygenated hemoglobin (HbO) in the prefrontal cortex during active walking and while performing a cognitive test under single- and dual-task conditions.RESULTSAdjusted general estimating equations models indicated that higher HbO under dual-task walking was significantly associated with a reduction in the odds of reporting falls among participants with relapsing-remitting (odds ratio (OR) = 0.472, p = 0.004, 95% confidence interval (CI) = 0.284-0.785), but not progressive (OR = 1.056, p = 0.792, 95% CI = 0.703-1.588) MS. In contrast, faster stride velocity under dual-task walking was significantly associated with a reduction in the odds of reporting falls among progressive (OR = 0.658, p = 0.004, 95% CI = 0.495-0.874), but not relapsing-remitting (OR = 0.998, p = 0.995, 95% CI = 0.523-1.905) MS.CONCLUSIONFindings suggest that higher prefrontal cortex activation levels during dual-task walking, which may represent compensatory reallocation of brain resources, provide protection against falls for OAMS with relapsing-remitting subtype.","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142182863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-08DOI: 10.1177/13524585241274620
Marcus W Koch, Carlos Camara-Lemarroy, Eva Strijbis, Jop Mostert, Victoria M Leavitt, Pavle Repovic, James D Bowen, Jacynthe Comtois, Bernard Uitdehaag, Gary Cutter
While relapsing-remitting multiple sclerosis (MS) has many therapeutic options, progressive forms of MS remain largely untreatable. Phase 2 clinical trials are our main tool to advance new treatments for progressive MS. Given the complexities of progressive MS, it will likely require many phase 2 trials to improve its treatment. To conduct informative and efficient phase 2 trials, it is important that such trials are designed in a way that they can identify a successful treatment as quickly and with as few participants as possible. In this topical review, we discuss cohort selection, outcome selection, cohort enrichment, and dosing selection as strategies to optimize the efficiency of phase 2 clinical trials in progressive MS.
{"title":"Selecting informative patients for phase 2 progressive trials in MS: Design considerations for phase 2 clinical trials in progressive MS.","authors":"Marcus W Koch, Carlos Camara-Lemarroy, Eva Strijbis, Jop Mostert, Victoria M Leavitt, Pavle Repovic, James D Bowen, Jacynthe Comtois, Bernard Uitdehaag, Gary Cutter","doi":"10.1177/13524585241274620","DOIUrl":"https://doi.org/10.1177/13524585241274620","url":null,"abstract":"<p><p>While relapsing-remitting multiple sclerosis (MS) has many therapeutic options, progressive forms of MS remain largely untreatable. Phase 2 clinical trials are our main tool to advance new treatments for progressive MS. Given the complexities of progressive MS, it will likely require many phase 2 trials to improve its treatment. To conduct informative and efficient phase 2 trials, it is important that such trials are designed in a way that they can identify a successful treatment as quickly and with as few participants as possible. In this topical review, we discuss cohort selection, outcome selection, cohort enrichment, and dosing selection as strategies to optimize the efficiency of phase 2 clinical trials in progressive MS.</p>","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-08DOI: 10.1177/13524585241273075
Todd A Hardy, Brian G Weinshenker
{"title":"Relapsing tumefactive demyelination: time to recognize a distinct demyelinating condition?","authors":"Todd A Hardy, Brian G Weinshenker","doi":"10.1177/13524585241273075","DOIUrl":"https://doi.org/10.1177/13524585241273075","url":null,"abstract":"","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-08DOI: 10.1177/13524585241273005
Paola Perini, Marta Gaggiola, Francesca Rinaldi, Paolo Gallo, Marco Puthenparampil
We report the case of a patient suffering from biopsy-proven relapsing tumefactive demyelinating lesions (TDLs) of the central nervous system who had five relapses in 16 years. No signs/symptoms suggestive of alternative pathologies emerged during the follow-up. A limited benefit was observed with intravenous (IV) high-dose steroids, while both plasma exchange and IV immunoglobulin G (IgG) administration were ineffective. A long-lasting (9 years) but transient clinical stabilization was obtained with cyclophosphamide. Our case supports the view that recurrent TDL is a relapsing brain inflammation not belonging to multiple sclerosis (MS) or myelin oligodendrocyte glycoprotein (MOG)-/AQP4-associated disorders. TDL concept and clinical features should be revised.
我们报告了一例经活检证实的复发性中枢神经系统肿瘤活动性脱髓鞘病变（TDLs）患者的病例，该患者在16年内复发了5次。在随访期间，没有出现其他病变的体征/症状。静脉注射大剂量类固醇的疗效有限，而血浆置换和静脉注射免疫球蛋白 G (IgG) 均无效。使用环磷酰胺后，患者的临床症状得到了长期（9 年）但短暂的稳定。我们的病例支持这样一种观点，即复发性 TDL 是一种复发性脑部炎症，不属于多发性硬化症（MS）或髓鞘少突胶质细胞糖蛋白（MOG）-/AQP4 相关疾病。TDL的概念和临床特征应予以修正。
{"title":"Relapsing tumefactive demyelination lesions: A unique, distinct inflammatory brain pathology.","authors":"Paola Perini, Marta Gaggiola, Francesca Rinaldi, Paolo Gallo, Marco Puthenparampil","doi":"10.1177/13524585241273005","DOIUrl":"https://doi.org/10.1177/13524585241273005","url":null,"abstract":"<p><p>We report the case of a patient suffering from biopsy-proven relapsing tumefactive demyelinating lesions (TDLs) of the central nervous system who had five relapses in 16 years. No signs/symptoms suggestive of alternative pathologies emerged during the follow-up. A limited benefit was observed with intravenous (IV) high-dose steroids, while both plasma exchange and IV immunoglobulin G (IgG) administration were ineffective. A long-lasting (9 years) but transient clinical stabilization was obtained with cyclophosphamide. Our case supports the view that recurrent TDL is a relapsing brain inflammation not belonging to multiple sclerosis (MS) or myelin oligodendrocyte glycoprotein (MOG)-/AQP4-associated disorders. TDL concept and clinical features should be revised.</p>","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154647","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-06DOI: 10.1177/13524585241272975
Ahmed Z Obeidat
{"title":"JC virus or extended interval dosing? More data are needed.","authors":"Ahmed Z Obeidat","doi":"10.1177/13524585241272975","DOIUrl":"https://doi.org/10.1177/13524585241272975","url":null,"abstract":"","PeriodicalId":18874,"journal":{"name":"Multiple Sclerosis Journal","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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