Fredrik Sandesjö, Peter Alping, Katharina Fink, Ronny Wickström, Fredrik Piehl, Thomas Frisell, Kyla A McKay
{"title":"瑞典多发性硬化症登记对儿科发病多发性硬化症的验证。","authors":"Fredrik Sandesjö, Peter Alping, Katharina Fink, Ronny Wickström, Fredrik Piehl, Thomas Frisell, Kyla A McKay","doi":"10.1177/20552173251314118","DOIUrl":null,"url":null,"abstract":"<p><p>Few controlled trials of disease-modifying therapies (DMTs) have been conducted on the pediatric-onset multiple sclerosis (PoMS) population, leading to extensive off-label use of therapies approved only for adults. This highlights the need for real-world evidence to guide clinical practice. Clinical registries can offer high-quality data, but limitations such as missing and erroneous information must be considered. This validation study compared Swedish Multiple Sclerosis registry data from 122 PoMS patients to medical records. Generally (≥89%), data were confirmed. However, missing data exceeded 30% for rituximab infusions, magnetic resonance imaging, and relapses. Overall, the registry provides valid, real-world data on DMT use in PoMS.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"11 1","pages":"20552173251314118"},"PeriodicalIF":2.5000,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11808745/pdf/","citationCount":"0","resultStr":"{\"title\":\"Validation of the Swedish Multiple Sclerosis registry for pediatric-onset multiple sclerosis.\",\"authors\":\"Fredrik Sandesjö, Peter Alping, Katharina Fink, Ronny Wickström, Fredrik Piehl, Thomas Frisell, Kyla A McKay\",\"doi\":\"10.1177/20552173251314118\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Few controlled trials of disease-modifying therapies (DMTs) have been conducted on the pediatric-onset multiple sclerosis (PoMS) population, leading to extensive off-label use of therapies approved only for adults. This highlights the need for real-world evidence to guide clinical practice. Clinical registries can offer high-quality data, but limitations such as missing and erroneous information must be considered. This validation study compared Swedish Multiple Sclerosis registry data from 122 PoMS patients to medical records. Generally (≥89%), data were confirmed. However, missing data exceeded 30% for rituximab infusions, magnetic resonance imaging, and relapses. Overall, the registry provides valid, real-world data on DMT use in PoMS.</p>\",\"PeriodicalId\":18961,\"journal\":{\"name\":\"Multiple Sclerosis Journal - Experimental, Translational and Clinical\",\"volume\":\"11 1\",\"pages\":\"20552173251314118\"},\"PeriodicalIF\":2.5000,\"publicationDate\":\"2025-02-09\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11808745/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Multiple Sclerosis Journal - Experimental, Translational and Clinical\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/20552173251314118\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/20552173251314118","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Validation of the Swedish Multiple Sclerosis registry for pediatric-onset multiple sclerosis.
Few controlled trials of disease-modifying therapies (DMTs) have been conducted on the pediatric-onset multiple sclerosis (PoMS) population, leading to extensive off-label use of therapies approved only for adults. This highlights the need for real-world evidence to guide clinical practice. Clinical registries can offer high-quality data, but limitations such as missing and erroneous information must be considered. This validation study compared Swedish Multiple Sclerosis registry data from 122 PoMS patients to medical records. Generally (≥89%), data were confirmed. However, missing data exceeded 30% for rituximab infusions, magnetic resonance imaging, and relapses. Overall, the registry provides valid, real-world data on DMT use in PoMS.