Challenges and opportunities in designing clinical trials for neuromyelitis optica.
Fiche publication
Date publication
avril 2015
Journal
Neurology
Auteurs
Membres identifiés du Cancéropôle Est :
Pr DE SEZE Jérôme
Tous les auteurs :
Weinshenker BG, Barron G, Behne JM, Bennett JL, Chin PS, Cree BA, de Seze J, Flor A, Fujihara K, Greenberg B, Higashi S, Holt W, Khan O, Knappertz V, Levy M, Melia AT, Palace J, Smith TJ, Sormani MP, Van Herle K, VanMeter S, Villoslada P, Walton MK, Wasiewski W, Wingerchuk DM, Yeaman MR,
Lien Pubmed
Résumé
Current management of neuromyelitis optica (NMO) is noncurative and only partially effective. Immunosuppressive or immunomodulatory agents are the mainstays of maintenance treatment. Safer, better-tolerated, and proven effective treatments are needed. The perceived rarity of NMO has impeded clinical trials for this disease. However, a diagnostic biomarker and recognition of a wider spectrum of NMO presentations has expanded the patient population from which study candidates might be recruited. Emerging insights into the pathogenesis of NMO have provided rationale for exploring new therapeutic targets. Academic, pharmaceutical, and regulatory communities are increasingly interested in meeting the unmet needs of patients with NMO. Clinical trials powered to yield unambiguous outcomes and designed to facilitate rapid evaluation of an expanding pipeline of experimental agents are needed. NMO-related disability occurs incrementally as a result of attacks; thus, limiting attack frequency and severity are critical treatment goals. Yet, the severity of NMO and perception that currently available agents are effective pose challenges to study design. We propose strategies for NMO clinical trials to evaluate agents targeting recovery from acute attacks and prevention of relapses, the 2 primary goals of NMO treatment. Aligning the interests of all stakeholders is an essential step to this end.
Mots clés
Clinical Trials as Topic, standards, Humans, Neuromyelitis Optica, drug therapy, Research Design, standards
Référence
Neurology. 2015 Apr 28;84(17):1805-15