Matches in SemOpenAlex for { <https://semopenalex.org/work/W4200241961> ?p ?o ?g. }
- W4200241961 endingPage "59" @default.
- W4200241961 startingPage "45" @default.
- W4200241961 abstract "Pediatric-onset multiple sclerosis (POMS) is a rare neuroinflammatory and neurodegenerative disease that has a significant impact on long-term physical and cognitive patient outcomes. A small percentage of multiple sclerosis (MS) diagnoses occur before the age of 18 years. Before treatment initiation, a careful differential diagnosis and exclusion of other similar acquired demyelinating syndromes such as anti-aquaporin-4-associated neuromyelitis optica spectrum disorder (AQP4-NMOSD) and myelin oligodendrocyte glycoprotein antibody spectrum disorder (MOGSD) is warranted. The recent 2017 changes to the McDonald criteria can successfully predict up to 71% of MS diagnoses and have good specificity of 95% and sensitivity of 71%. Additional measures such as the presence of T1-weighted hypointense lesions and/or contrast-enhancing lesions significantly increase the accuracy of diagnosis. In adults, early use of disease-modifying therapies (DMTs) is instrumental to a better long-term prognosis, including lower rates of relapse and disability worsening, and numerous FDA-approved therapies for adult-onset MS are available. However, unlike their adult counterparts, the development, testing, and regulatory approval of POMS treatments have been significantly slower and hindered by logistic and/or ethical considerations. Currently, only two MS DMTs (fingolimod and teriflunomide) have been tested in large phase III trials and approved by regulatory agencies for use in POMS. First-line therapies not approved by the FDA for use in children (interferon-β and glatiramer acetate) are also commonly used and result in a significant reduction in inflammatory activity when compared with non-treated POMS patients. An increasing number of POMS patients are now treated with moderate efficacy therapies such as dimethyl fumarate and high-efficacy therapies such as natalizumab, anti-CD20 monoclonal antibodies, anti-CD52 monoclonal antibodies, and/or autologous hematopoietic stem cell transplantation. These high-efficacy DMTs generally provide additional reduction in inflammatory activity when compared with the first-line medications (up to 62% of relapse-rate reduction). Therefore, a number of phase II and III trials are currently investigating their efficacy and safety in POMS patients. In this review, we discuss potential changes in the regulatory approval process for POMS patients that are recommended for DMTs already approved for the adult MS population, including smaller sample size for pharmacokinetic/pharmacodynamic studies, MRI-centered primary outcomes, and/or inclusion of teenagers in the adult trials." @default.
- W4200241961 created "2021-12-31" @default.
- W4200241961 creator A5007419149 @default.
- W4200241961 creator A5049031620 @default.
- W4200241961 creator A5068757910 @default.
- W4200241961 creator A5075596287 @default.
- W4200241961 creator A5078270995 @default.
- W4200241961 date "2021-12-23" @default.
- W4200241961 modified "2023-10-17" @default.
- W4200241961 title "Multiple Sclerosis in Children: Differential Diagnosis, Prognosis, and Disease-Modifying Treatment" @default.
- W4200241961 cites W1606806706 @default.
- W4200241961 cites W1910618472 @default.
- W4200241961 cites W1940540259 @default.
- W4200241961 cites W1968634262 @default.
- W4200241961 cites W1975434298 @default.
- W4200241961 cites W1983382084 @default.
- W4200241961 cites W1991230604 @default.
- W4200241961 cites W2004913798 @default.
- W4200241961 cites W2008101102 @default.
- W4200241961 cites W2010766933 @default.
- W4200241961 cites W2041135783 @default.
- W4200241961 cites W2055514366 @default.
- W4200241961 cites W2070362842 @default.
- W4200241961 cites W2082733383 @default.
- W4200241961 cites W2087353379 @default.
- W4200241961 cites W2095666175 @default.
- W4200241961 cites W2122004881 @default.
- W4200241961 cites W2128597204 @default.
- W4200241961 cites W2129229339 @default.
- W4200241961 cites W2149626428 @default.
- W4200241961 cites W2150189960 @default.
- W4200241961 cites W2152959293 @default.
- W4200241961 cites W2163795029 @default.
- W4200241961 cites W2171946334 @default.
- W4200241961 cites W2255179201 @default.
- W4200241961 cites W2266233634 @default.
- W4200241961 cites W2282058593 @default.
- W4200241961 cites W2313649737 @default.
- W4200241961 cites W2337612449 @default.
- W4200241961 cites W2399044152 @default.
- W4200241961 cites W2417250624 @default.
- W4200241961 cites W2463007509 @default.
- W4200241961 cites W2470605326 @default.
- W4200241961 cites W2530586594 @default.
- W4200241961 cites W2585644883 @default.
- W4200241961 cites W2598831576 @default.
- W4200241961 cites W2600313485 @default.
- W4200241961 cites W2603619943 @default.
- W4200241961 cites W2624913077 @default.
- W4200241961 cites W2727812173 @default.
- W4200241961 cites W2761348774 @default.
- W4200241961 cites W2763089819 @default.
- W4200241961 cites W2780085207 @default.
- W4200241961 cites W2784314693 @default.
- W4200241961 cites W2786295168 @default.
- W4200241961 cites W2790072792 @default.
- W4200241961 cites W2890515892 @default.
- W4200241961 cites W2893009209 @default.
- W4200241961 cites W2896092607 @default.
- W4200241961 cites W2922135738 @default.
- W4200241961 cites W2926802240 @default.
- W4200241961 cites W2943065586 @default.
- W4200241961 cites W2943914475 @default.
- W4200241961 cites W2945360219 @default.
- W4200241961 cites W2945464797 @default.
- W4200241961 cites W2948384015 @default.
- W4200241961 cites W2949607593 @default.
- W4200241961 cites W2951963316 @default.
- W4200241961 cites W2953750028 @default.
- W4200241961 cites W2971252766 @default.
- W4200241961 cites W2974098954 @default.
- W4200241961 cites W2975976017 @default.
- W4200241961 cites W2991627431 @default.
- W4200241961 cites W2996124872 @default.
- W4200241961 cites W2999174237 @default.
- W4200241961 cites W3003719100 @default.
- W4200241961 cites W3010457503 @default.
- W4200241961 cites W3014545366 @default.
- W4200241961 cites W3033837102 @default.
- W4200241961 cites W3035067402 @default.
- W4200241961 cites W3036104027 @default.
- W4200241961 cites W3040620803 @default.
- W4200241961 cites W3041549013 @default.
- W4200241961 cites W3044854809 @default.
- W4200241961 cites W3051923090 @default.
- W4200241961 cites W3093960533 @default.
- W4200241961 cites W3095068517 @default.
- W4200241961 cites W3095227904 @default.
- W4200241961 cites W3096498029 @default.
- W4200241961 cites W3118270602 @default.
- W4200241961 cites W3124681486 @default.
- W4200241961 cites W3130127311 @default.
- W4200241961 cites W3131513247 @default.
- W4200241961 cites W3133266325 @default.
- W4200241961 cites W3158406722 @default.
- W4200241961 cites W3203041565 @default.
- W4200241961 cites W3211389104 @default.
- W4200241961 cites W4240233136 @default.