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.authorid0000-0002-7406-4600
dc.contributor.authorAl-Taie, Anmar
dc.contributor.authorKöseoğlu, Aygül
dc.date.accessioned2024-01-30T10:49:38Z
dc.date.available2024-01-30T10:49:38Z
dc.date.issued2023
dc.departmentİstanbul Medipol Üniversitesi, Eczacılık Fakültesi, Eczacılık Meslek Bilimleri Bölümü, Klinik Eczacılık Ana Bilim Dalı
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.
dc.identifier.citationAl-Taie, A. ve Köseoğlu, A. (2023). 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. Paediatric Respiratory Reviews, 48, 65-71. https://dx.doi.org/10.1016/j.prrv.2023.06.004
dc.identifier.doi10.1016/j.prrv.2023.06.004
dc.identifier.endpage71
dc.identifier.issn1526-0542
dc.identifier.issn1526-0550
dc.identifier.pmid37563072
dc.identifier.startpage65
dc.identifier.urihttps://dx.doi.org/10.1016/j.prrv.2023.06.004
dc.identifier.urihttps://hdl.handle.net/20.500.12511/12218
dc.identifier.volume48
dc.identifier.wos001142976900001en_US
dc.identifier.wosqualityQ1
dc.indekslendigikaynakWeb of Science
dc.indekslendigikaynakPubMed
dc.institutionauthorKöseoğlu, Aygül
dc.language.isoen
dc.publisherElsevier Science Ltd
dc.relation.ispartofPaediatric Respiratory Reviewsen_US
dc.relation.publicationcategoryDiğer
dc.rightsinfo:eu-repo/semantics/embargoedAccess
dc.subjectSpinal Muscular Atrophy Type 1
dc.subjectMotor Functions
dc.subjectNusinersen
dc.subjectOnasemnogene Abeparvovec
dc.subjectRespiratory Functions
dc.subjectRisdiplam
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 review
dc.typeReview Article

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