Files

Download

Download Full Text (944 KB)

Document Type

Poster

Description

CysticFibrosis(CF)isanautosomalrecessivedisorderthatshortensone’slifeduetoitseffectonthecysticfibrosistransmembraneregulator(CFTR)gene.CFTRmodulatormedicationscancorrecttheCFTRgeneandconsistofapotentiator(ivacaftor),andcorrector(lumacaftor,tezacaftorandelexacaftor).ThisresearchandliteraturereviewsoughttoevaluateiflungfunctionimprovedinanindividualwithgeneticallyconfirmedCFafterbeingstartedonCFTRmodulatormedications.Tocompletethereview,sixdatabasesweresearched:CINAHL,PubMed,ClinicalKey,CochraneLibrary,EmbaseandDynamedPlus.BothkeywordandmeshtermswereusedtodefineasetoftheliteraturediscussingCFandCFTRmodulatoruse.Atotalof24studieswereincludedandmettheinclusioncriteriaof:peerreviewed,publishedwithinthepastsixyears,greaterthan20studyparticipants,andwerenotlimitedcasereports.Phase2studieswereincludedforelexacaftor-tezacaftor-ivacaftorandtezacaftor-ivacaftor,duetolimitedresearchinthisarea.Researchindicatedthativacaftorimprovedpercentpredictedforcedexpiratoryvolumeinonesecond(ppFEV1)inindividualswithG551D,non-G551Dgatingmutations,Gly-Asp-CFTRandARG117HISmutations.Elexacaftor-tezacaftor-ivacaftorimprovedppFEV1inheterozygousandhomozygousPhe508delgreaterthanivacaftororlumacaftor-ivacaftor.Ivacaftorandlumacaftor-ivacaftormayhaveagedependentchangesonppFEV1.IndividualswithppFEV1<40%appeartohaveapositiveresponsetotreatment,howevertheymayhaveincreasedsideeffectsandresponsemaybedelayed.Additionalresearchisneededonagedependent,longitudinalresponse,andefficacyoftreatment.

Department

Physician Assistant Studies

Date of Work

2021

Publication Date

2021

Keywords

Cystic Fibrosis, CFTR Modulators, Respiratory Function, autosomal recessive disorder, multi-system disorder, genetic mutation CFTR gene, obstructive lung disease, Ivacaftor monotherapy

Disciplines

Medicine and Health Sciences

Effect of CFTR Modulators on Respiratory Function in Adults with Cystic Fibrosis

Share

COinS