Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review

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dc.authoridALTAIE, Anmar/0000-0002-2183-6830
dc.authorwosidALTAIE, Anmar/ITV-8235-2023
dc.contributor.authorAl-Taie, Anmar
dc.contributor.authorKoseoglu, Aygul
dc.date.accessioned2024-05-19T14:46:46Z
dc.date.available2024-05-19T14:46:46Z
dc.date.issued2023
dc.departmentİstinye Üniversitesien_US
dc.description.abstractSpinal muscular atrophy (SMA) is a severe hereditary lower motor neuron disorder characterised by degeneration of alpha motor neurons in the spinal cord, resulting in progressive weakness and paralysis of proximal muscles. A systematic literature search was carried out by using PRISMA guidelines and searching through different databases that could provide findings of evidence on the health outcomes of the approved therapies for the management of paediatric SMA type 1 regarding efficacy with follow-up in terms of motor and respiratory functions and the tolerability and incidence of adverse drug reactions (ADRs) post-treatment from real-world publications. Half of the publications (50%) had a prospective observational design. Eight studies (66.7%) assessed nusinersen, and three studies (25%) assessed onasemnogene abeparvovec with a duration of follow-up ranging from 6 months to 3 years to evaluate the motor and respiratory functions using different assessment tools, hospitalisation rates, and the tolerability and incidence of ADRs post-treatment. The three currently approved treatments for SMA type 1 provided good support and health outcomes in terms of motor function, respiratory outcomes, reduction of hospitalisations, and improvement of survival. Nevertheless, uncertainties regarding continued improvement after long-term illness and the generalizability of results are still unknown.(c) 2023 Elsevier Ltd. All rights reserved.en_US
dc.identifier.doi10.1016/j.prrv.2023.06.004
dc.identifier.endpage71en_US
dc.identifier.issn1526-0542
dc.identifier.issn1526-0550
dc.identifier.pmid37563072en_US
dc.identifier.scopus2-s2.0-85167777992en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage65en_US
dc.identifier.urihttps://doi.org10.1016/j.prrv.2023.06.004
dc.identifier.urihttps://hdl.handle.net/20.500.12713/5589
dc.identifier.volume48en_US
dc.identifier.wosWOS:001142976900001en_US
dc.identifier.wosqualityN/Aen_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.language.isoenen_US
dc.publisherElsevier Sci Ltden_US
dc.relation.ispartofPaediatric Respiratory Reviewsen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.snmz20240519_kaen_US
dc.subjectSpinal Muscular Atrophy Type 1en_US
dc.subjectMotor Functionsen_US
dc.subjectNusinersenen_US
dc.subjectOnasemnogene Abeparvovecen_US
dc.subjectRespiratory Functionsen_US
dc.subjectRisdiplamen_US
dc.titleEvaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic reviewen_US
dc.typeReview Articleen_US

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