CNS/Neurology

Spelman T, Herring WL, Acosta C, Hyde R, Jokubaitis VG, Pucci E, Lugaresi A, Laureys G, Havrdova EK, Horakova D, Eichau S, Ozakbas S, Alroughani R, Kalincik T, Duquette P, Girard M, Petersen T, Patti F, Csepany T, Granella F, Grand'Maison F, Ferraro D, Karabudak R, Jose Sa M, Trojano M, Van Pesch V, Van Mijmeersch B, Cartechini E, McCombe P, Gerlach O, Spitaleri D, Rozsa C, Hodgkinson S, Bergamaschi R, Gouider R, Soysal A, Castillo-Trivino T, Prevost J, Garber J, de Gans K, Ampapa R, Simo M, Sanchez-Menoyo JL, Iuliano G, Sas A, van der Walt A, John N, Gray O, Hughes S, De Luca G, Izquierdo G, Onofrj M, Buzzard K, Skibina O, Terzi M, Slee M, Solaro C, Oreja-Guevara C, Ramo-Tello C, Fragoso Y, Shaygannejad V, Moore F, Rajda C, Aguera Morales E, Butzkueven H. Comparative effectiveness and cost-effectiveness of natalizumab and fingolimod in rapidly evolving severe relapsing-remitting multiple sclerosis in the United Kingdom. J Med Econ. 2023 Dec;27(1):109-25. doi: 10.1080/13696998.2023.2293379


AIM: To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS).

Sawant R, Paret K, Petrillo J, Koenig A, Wolowacz S, Ronquest N, Rickards H. Health state utility estimates for value assessments of novel treatments in Huntington's disease: a systematic literature review. Health Qual Life Outcomes. 2024 Apr 16;22(1):33. doi: 10.1186/s12955-024-02242-1


Gerbasi ME, Goss D, Petrillo J, Nejati M, Lewis S. Patient experiences in essential tremor: mapping functional impacts to existing measures using qualitative research. Poster presented at the 2023 International Congress of Parkinson's Disease and Movement Disorders; August 27, 2023. Copenhagen, Denmark. [abstract] Mov Disord. 2023; 38(Suppl 1).


OBJECTIVE: To understand patient experience in essential tremor (ET), describe meaningful concepts related to treatment benefit, and compare concepts to existing clinical outcome assessments (COAs). Background: ET is among the most common movement disorders, but patient experience in ET is understudied and has not been mapped to COAs.

Shapouri S, Candrilli S, Miles L, Simpson A, Guittari CJ. Real-world treatment with risdiplam in adults with SMA: a multicenter study. Poster presented at the 2023 Annual SMA Research & Clinical Care Meeting; June 28, 2023. Orlando, FL.


BACKGROUND: Although there is a body of literature on the use of risdiplam (EVRYSDI®) in adult patients from clinical trials, including SUNFISH (NCT02908685; 2–25 years) and JEWELFISH (NCT03032172; 1–60 years), available real-world evidence on the use of risdiplam is limited in adult patients.

Halsby K, Gildea L, Colosia A, Madhava H, Erber W, Angulo F, Pilz A, Schley K, Moisi J, Sellner J. The clinical spectrum of TBE infections in adult and paediatric populations in Europe: a systematic review. Poster presented at the International Symposium on Tick-Borne Pathogens and Disease (ITPD) 2023; October 24, 2023. Vienna, Austria.


Tahami Monfared AA, Phan NTN, Pearson I, Mauskopf J, Cho M, Zhang Q, Hampel H. A systematic review of clinical practice guidelines for Alzheimer's disease and strategies for future advancements. Neurol Ther. 2023 Aug;12(4):1257-84. doi: 10.1007/s40120-023-00504-6


INTRODUCTION: Alzheimer’s disease (AD) is a disease continuum from pathophysiological, biomarker, and clinical perspectives. With the advent of advanced technologies, diagnosing and managing patients is evolving.

Lansdall CJ, McDougall F, Butler LM, Delmar P, Pross N, Qin S, McLeod L, Zhou X, Kerchner GA, Doody RS. Establishing clinically meaningful change on outcome assessments frequently used in trials of mild cognitive impairment due to Alzheimer's disease. J Prev Alzheimers Dis. 2023;10(1):9-18. doi: 10.14283/jpad.2022.102


BACKGROUND: Consensus is lacking on what constitutes a meaningful score change for individual patients on clinical outcome assessments (COAs) that are commonly used in clinical trials of Alzheimer's disease. Such thresholds are one important approach to help contextualize trial results and demonstrate meaningful treatment benefit.

Razavi M, Herring WL, Maserejian N, Pemberton-Ross P, Nejati M. Economic burden of transitions to later stages of Alzheimer's disease in Europe: elevating the sense of urgency to treat. Poster presented at the AAIC 2023 Conference; July 16, 2023. Amsterdam, Netherlands.


Romano CD, Slota C, Bratlee-Whitaker E, Edwards T, Herring WL, McLeod L, Callahan L, Bullok K, Raimundo K, Taylor J, Taylor G, Kremer I, Monks D, Lappin D, Frangiosa T, Gnanasakthy K, Goss D, Comer M, Paulsen R, Hartry A, DiBenedetti D. Measuring "What Matters Most™" to individuals living with Alzheimer's disease and care partners: a conceptual model of Alzheimer's disease. Poster presented at the AAIC 2023 Conference; July 16, 2023. Amsterdam, Netherlands.


Clinch S, McDougall F, Barrett A, Olayinka-Amao B, Craveiro L, Baker M, Lindemann M. A patient-focused qualitative study to support content validity of digital performance assessments in MS. Poster presented at the 2021 ECTRIMS Annual Congress; October 13, 2021. [abstract] Mult Scler J Exp Transl Clin. 2021 Oct 21; 27(2 Suppl):329. doi: 10.1177/13524585211044667


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