Publication:
Abatacept as a long-term targeted therapy for LRBA deficiency

dc.contributor.authorKiykim, Ayça
dc.contributor.authorOgulur, İsmail
dc.contributor.authorDursun, Esra
dc.contributor.authorDogruel, Dilek
dc.contributor.authorKaraca, Neslihan Edeer
dc.contributor.authorCogurlu, Mujde Tuba
dc.contributor.authorBilir, Ozlem Arman
dc.contributor.authorCansever, Murat
dc.contributor.authorKapakli, Hasan
dc.contributor.authorBaser, Dilek
dc.contributor.authorKasap, Nurhan
dc.contributor.authorKutlug, Seyhan
dc.contributor.authorAltintas, Derya Ufuk
dc.contributor.authorAl-Shaibi, Ahmad
dc.contributor.authorAgrebi, Nourhen
dc.contributor.authorKara, Manolya
dc.contributor.authorGuven, Ayla
dc.contributor.authorSomer, Ayper
dc.contributor.authorAydogmus, Cigdem
dc.contributor.authorAyaz, Nuray Aktay
dc.contributor.authorMetin, Ayse
dc.contributor.authorAydogan, Metin
dc.contributor.authorUncuoglu, Aysen
dc.contributor.authorPatiroglu, Turkan
dc.contributor.authorYildiran, Alisan
dc.contributor.authorGuner, Sukru Nail
dc.contributor.authorKeles, Sevgi
dc.contributor.authorReisli, Ismail
dc.contributor.authorAksu, Guzide
dc.contributor.authorKutukculer, Necil
dc.contributor.authorYilmaz, Mustafa
dc.contributor.authorKarakoc-Aydiner, Elif
dc.contributor.authorLo, Bernice
dc.contributor.authorOzan, Ahmet
dc.contributor.authorChatila, Talal A.
dc.contributor.authorBarıs, Safa
dc.contributor.buuauthorÇekiç, Şükrü
dc.contributor.buuauthorKılıç, Sara Şebnem
dc.contributor.departmentTıp Fakültesi
dc.contributor.departmentDahili Bilimler
dc.contributor.departmentÇocuk Sağlığı ve Hastalıkları
dc.contributor.orcid0000-0002-9574-1842tr_TR
dc.contributor.orcid0000-0001-8571-2581tr_TR
dc.contributor.researcheridL-1933-2017tr_TR
dc.contributor.scopusid57094682600tr_TR
dc.contributor.scopusid7102365439tr_TR
dc.date.accessioned2022-11-24T11:37:56Z
dc.date.available2022-11-24T11:37:56Z
dc.date.issued2019-12
dc.description.abstractBACKGROUND: LPS-responsive beige-like anchor (LRBA) deficiency presents with susceptibility to infections, autoimmunity, and lymphoproliferation. The long-term efficacy of cytotoxic T-lymphocyte-associated antigen 4-immunoglobulin (abatacept) as targeted therapy for its immune dysregulatory features remains to be established. OBJECTIVE: To determine the clinical and immunologic features of LRBA deficiency and long-term efficacy of abatacept treatment in controlling the different disease manifestations. METHODS: Twenty-two LRBA-deficient patients were recruited from different immunology centers and followed prospectively. Eighteen patients on abatacept were evaluated every 3 months for long-term clinical and immunologic responses. LRBA expression, lymphocyte subpopulations, and circulating T follicular helper cells were determined by flow cytometry. RESULTS: The mean age of the patients was 13.4 +/- 7.9 years, and the follow-up period was 3.4 +/- 2.3 years. Recurrent infections (n = 19 [86.4%]), immune dysregulation (n = 18 [81.8%]), and lymphoproliferation (n = 16 [72.7%]) were common clinical features. The long-term benefits of abatacept in 16 patients were demonstrated by complete control of lymphoproliferation and chronic diarrhea followed by immune dysregulation, most notably autoimmune cytopenias. Weekly or every other week administration of abatacept gave better disease control compared with every 4 weeks. There were no serious side effects related to the abatacept therapy. Circulating T follicular helper cell frequencies were found to be a reliable biomarker of disease activity, which decreased on abatacept therapy in most subjects. However, high circulating T follicular helper cell frequencies persisted in 2 patients who had a more severe disease phenotype that was relatively resistant to abatacept therapy. CONCLUSIONS: Long-term abatacept therapy is effective in most patients with LRBA deficiency.en_US
dc.description.sponsorshipUnited States Department of Health & Human Services National Institutes of Health (NIH) - USA (5R01AI085090) (R01AI065617) (R01AI085090)en_US
dc.identifier.citationKiykim, A. vd. (2019). ''Abatacept as a long-term targeted therapy for LRBA deficiency ''. Journal of Allergy and Clinical Immunology-in Practice, 7(8), 2790-2800.en_US
dc.identifier.endpage2800tr_TR
dc.identifier.issn2213-2198
dc.identifier.issn2213-2201
dc.identifier.issue8tr_TR
dc.identifier.pubmed31238161tr_TR
dc.identifier.scopus2-s2.0-85068767793tr_TR
dc.identifier.startpage2790tr_TR
dc.identifier.urihttps://doi.org/10.1016/j.jaip.2019.06.011
dc.identifier.urihttps://www.sciencedirect.com/science/article/abs/pii/S221321981930563X
dc.identifier.urihttp://hdl.handle.net/11452/29557
dc.identifier.volume7tr_TR
dc.identifier.wos000495746100038
dc.indexed.pubmedPubMeden_US
dc.indexed.scopusScopusen_US
dc.indexed.wosSCIEen_US
dc.language.isoenen_US
dc.publisherElsevieren_US
dc.relation.collaborationYurt dışıtr_TR
dc.relation.collaborationSanayitr_TR
dc.relation.journalJournal of Allergy and Clinical Immunology-Practiceen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergitr_TR
dc.relation.tubitak217S847tr_TR
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectImmune dysregulatıonen_US
dc.subjectCtla-4 checpointen_US
dc.subjectMutationsen_US
dc.subjectPolyendocrinopathyen_US
dc.subjectEnteropathyen_US
dc.subjectDiseaseen_US
dc.subjectLps-responsive beige-like anchoren_US
dc.subjectImmune dysregulationen_US
dc.subjectAbatacepten_US
dc.subjectT follicular helper cellsen_US
dc.subjectAutoimmunityen_US
dc.subject.emtreeAbatacepten_US
dc.subject.emtreeBiological markeren_US
dc.subject.emtreeCarrier proteins and binding proteinsen_US
dc.subject.emtreeLipopolysaccharide responsive beige like anchor proteinen_US
dc.subject.emtreeUnclassified drugen_US
dc.subject.emtreeAbatacepten_US
dc.subject.emtreeImmunosuppressive agenten_US
dc.subject.emtreeLrba proteinen_US
dc.subject.emtreeHumanen_US
dc.subject.emtreeSignal transducing adaptor proteinen_US
dc.subject.emtreeAdolescenten_US
dc.subject.emtreeAdulten_US
dc.subject.emtreeArticleen_US
dc.subject.emtreeAutoimmunityen_US
dc.subject.emtreeChilden_US
dc.subject.emtreeChronic diarrheaen_US
dc.subject.emtreeClinical articleen_US
dc.subject.emtreeClinical featureen_US
dc.subject.emtreeControlled studyen_US
dc.subject.emtreeCytopeniaen_US
dc.subject.emtreeCytotoxicityen_US
dc.subject.emtreeDisease activityen_US
dc.subject.emtreeDisease severityen_US
dc.subject.emtreeDrug efficacyen_US
dc.subject.emtreeDrug responseen_US
dc.subject.emtreeFemaleen_US
dc.subject.emtreeFlow cytometryen_US
dc.subject.emtreeFollow upen_US
dc.subject.emtreeImmune dysregulationen_US
dc.subject.emtreeImmune responseen_US
dc.subject.emtreeImmunopathologyen_US
dc.subject.emtreeInfection sensitivityen_US
dc.subject.emtreeLong term careen_US
dc.subject.emtreeLymphocyte proliferationen_US
dc.subject.emtreeLymphocyte subpopulationen_US
dc.subject.emtreeMaleen_US
dc.subject.emtreeMolecularly targeted therapyen_US
dc.subject.emtreePhenotypeen_US
dc.subject.emtreePreschool childen_US
dc.subject.emtreeProtein deficiencyen_US
dc.subject.emtreeProtein expressionen_US
dc.subject.emtreeRecurrent infectionen_US
dc.subject.emtreeSchool childen_US
dc.subject.emtreeT lymphocyteen_US
dc.subject.emtreeTfh cellen_US
dc.subject.emtreeTherapy effecten_US
dc.subject.emtreeYoung adulten_US
dc.subject.emtreeControlled clinical trialen_US
dc.subject.emtreeGeneticsen_US
dc.subject.emtreeImmune deficiencyen_US
dc.subject.emtreeMolecularly targeted therapyen_US
dc.subject.emtreeTreatment outcomeen_US
dc.subject.meshAbatacepten_US
dc.subject.meshAdaptor proteins, signal transducingen_US
dc.subject.meshAdolescenten_US
dc.subject.meshAdulten_US
dc.subject.meshChilden_US
dc.subject.meshChild preschoolen_US
dc.subject.meshFemaleen_US
dc.subject.meshHumansen_US
dc.subject.meshImmunologic deficiency syndromesen_US
dc.subject.meshImmunosuppressive agentsen_US
dc.subject.meshMaleen_US
dc.subject.meshMolecular targeted therapyen_US
dc.subject.meshTreatment outcomeen_US
dc.subject.meshYoung adulten_US
dc.subject.scopusCommon Variable Immunodeficiency; Immunoglobulin Deficiency; Immunosuppressionen_US
dc.subject.wosAllergyen_US
dc.subject.wosImmunologyen_US
dc.titleAbatacept as a long-term targeted therapy for LRBA deficiencyen_US
dc.typeArticle
dc.wos.quartileQ1en_US
dspace.entity.typePublication
local.contributor.departmentTıp Fakültesi/Dahili Bilimler/Çocuk Sağlığı ve Hastalıklarıtr_TR
local.contributor.departmentTıp Fakültesi/Dahili Bilimler/Çocuk Sağlığı ve Hastalıklarıtr_TR

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