Publication: Abatacept as a long-term targeted therapy for LRBA deficiency
dc.contributor.author | Kiykim, Ayça | |
dc.contributor.author | Ogulur, İsmail | |
dc.contributor.author | Dursun, Esra | |
dc.contributor.author | Dogruel, Dilek | |
dc.contributor.author | Karaca, Neslihan Edeer | |
dc.contributor.author | Cogurlu, Mujde Tuba | |
dc.contributor.author | Bilir, Ozlem Arman | |
dc.contributor.author | Cansever, Murat | |
dc.contributor.author | Kapakli, Hasan | |
dc.contributor.author | Baser, Dilek | |
dc.contributor.author | Kasap, Nurhan | |
dc.contributor.author | Kutlug, Seyhan | |
dc.contributor.author | Altintas, Derya Ufuk | |
dc.contributor.author | Al-Shaibi, Ahmad | |
dc.contributor.author | Agrebi, Nourhen | |
dc.contributor.author | Kara, Manolya | |
dc.contributor.author | Guven, Ayla | |
dc.contributor.author | Somer, Ayper | |
dc.contributor.author | Aydogmus, Cigdem | |
dc.contributor.author | Ayaz, Nuray Aktay | |
dc.contributor.author | Metin, Ayse | |
dc.contributor.author | Aydogan, Metin | |
dc.contributor.author | Uncuoglu, Aysen | |
dc.contributor.author | Patiroglu, Turkan | |
dc.contributor.author | Yildiran, Alisan | |
dc.contributor.author | Guner, Sukru Nail | |
dc.contributor.author | Keles, Sevgi | |
dc.contributor.author | Reisli, Ismail | |
dc.contributor.author | Aksu, Guzide | |
dc.contributor.author | Kutukculer, Necil | |
dc.contributor.author | Yilmaz, Mustafa | |
dc.contributor.author | Karakoc-Aydiner, Elif | |
dc.contributor.author | Lo, Bernice | |
dc.contributor.author | Ozan, Ahmet | |
dc.contributor.author | Chatila, Talal A. | |
dc.contributor.author | Barıs, Safa | |
dc.contributor.buuauthor | Çekiç, Şükrü | |
dc.contributor.buuauthor | Kılıç, Sara Şebnem | |
dc.contributor.department | Tıp Fakültesi | |
dc.contributor.department | Dahili Bilimler | |
dc.contributor.department | Çocuk Sağlığı ve Hastalıkları | |
dc.contributor.orcid | 0000-0002-9574-1842 | tr_TR |
dc.contributor.orcid | 0000-0001-8571-2581 | tr_TR |
dc.contributor.researcherid | L-1933-2017 | tr_TR |
dc.contributor.scopusid | 57094682600 | tr_TR |
dc.contributor.scopusid | 7102365439 | tr_TR |
dc.date.accessioned | 2022-11-24T11:37:56Z | |
dc.date.available | 2022-11-24T11:37:56Z | |
dc.date.issued | 2019-12 | |
dc.description.abstract | BACKGROUND: 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.sponsorship | United States Department of Health & Human Services National Institutes of Health (NIH) - USA (5R01AI085090) (R01AI065617) (R01AI085090) | en_US |
dc.identifier.citation | Kiykim, 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.endpage | 2800 | tr_TR |
dc.identifier.issn | 2213-2198 | |
dc.identifier.issn | 2213-2201 | |
dc.identifier.issue | 8 | tr_TR |
dc.identifier.pubmed | 31238161 | tr_TR |
dc.identifier.scopus | 2-s2.0-85068767793 | tr_TR |
dc.identifier.startpage | 2790 | tr_TR |
dc.identifier.uri | https://doi.org/10.1016/j.jaip.2019.06.011 | |
dc.identifier.uri | https://www.sciencedirect.com/science/article/abs/pii/S221321981930563X | |
dc.identifier.uri | http://hdl.handle.net/11452/29557 | |
dc.identifier.volume | 7 | tr_TR |
dc.identifier.wos | 000495746100038 | |
dc.indexed.pubmed | PubMed | en_US |
dc.indexed.scopus | Scopus | en_US |
dc.indexed.wos | SCIE | en_US |
dc.language.iso | en | en_US |
dc.publisher | Elsevier | en_US |
dc.relation.collaboration | Yurt dışı | tr_TR |
dc.relation.collaboration | Sanayi | tr_TR |
dc.relation.journal | Journal of Allergy and Clinical Immunology-Practice | en_US |
dc.relation.publicationcategory | Makale - Uluslararası Hakemli Dergi | tr_TR |
dc.relation.tubitak | 217S847 | tr_TR |
dc.rights | info:eu-repo/semantics/openAccess | en_US |
dc.subject | Immune dysregulatıon | en_US |
dc.subject | Ctla-4 checpoint | en_US |
dc.subject | Mutations | en_US |
dc.subject | Polyendocrinopathy | en_US |
dc.subject | Enteropathy | en_US |
dc.subject | Disease | en_US |
dc.subject | Lps-responsive beige-like anchor | en_US |
dc.subject | Immune dysregulation | en_US |
dc.subject | Abatacept | en_US |
dc.subject | T follicular helper cells | en_US |
dc.subject | Autoimmunity | en_US |
dc.subject.emtree | Abatacept | en_US |
dc.subject.emtree | Biological marker | en_US |
dc.subject.emtree | Carrier proteins and binding proteins | en_US |
dc.subject.emtree | Lipopolysaccharide responsive beige like anchor protein | en_US |
dc.subject.emtree | Unclassified drug | en_US |
dc.subject.emtree | Abatacept | en_US |
dc.subject.emtree | Immunosuppressive agent | en_US |
dc.subject.emtree | Lrba protein | en_US |
dc.subject.emtree | Human | en_US |
dc.subject.emtree | Signal transducing adaptor protein | en_US |
dc.subject.emtree | Adolescent | en_US |
dc.subject.emtree | Adult | en_US |
dc.subject.emtree | Article | en_US |
dc.subject.emtree | Autoimmunity | en_US |
dc.subject.emtree | Child | en_US |
dc.subject.emtree | Chronic diarrhea | en_US |
dc.subject.emtree | Clinical article | en_US |
dc.subject.emtree | Clinical feature | en_US |
dc.subject.emtree | Controlled study | en_US |
dc.subject.emtree | Cytopenia | en_US |
dc.subject.emtree | Cytotoxicity | en_US |
dc.subject.emtree | Disease activity | en_US |
dc.subject.emtree | Disease severity | en_US |
dc.subject.emtree | Drug efficacy | en_US |
dc.subject.emtree | Drug response | en_US |
dc.subject.emtree | Female | en_US |
dc.subject.emtree | Flow cytometry | en_US |
dc.subject.emtree | Follow up | en_US |
dc.subject.emtree | Immune dysregulation | en_US |
dc.subject.emtree | Immune response | en_US |
dc.subject.emtree | Immunopathology | en_US |
dc.subject.emtree | Infection sensitivity | en_US |
dc.subject.emtree | Long term care | en_US |
dc.subject.emtree | Lymphocyte proliferation | en_US |
dc.subject.emtree | Lymphocyte subpopulation | en_US |
dc.subject.emtree | Male | en_US |
dc.subject.emtree | Molecularly targeted therapy | en_US |
dc.subject.emtree | Phenotype | en_US |
dc.subject.emtree | Preschool child | en_US |
dc.subject.emtree | Protein deficiency | en_US |
dc.subject.emtree | Protein expression | en_US |
dc.subject.emtree | Recurrent infection | en_US |
dc.subject.emtree | School child | en_US |
dc.subject.emtree | T lymphocyte | en_US |
dc.subject.emtree | Tfh cell | en_US |
dc.subject.emtree | Therapy effect | en_US |
dc.subject.emtree | Young adult | en_US |
dc.subject.emtree | Controlled clinical trial | en_US |
dc.subject.emtree | Genetics | en_US |
dc.subject.emtree | Immune deficiency | en_US |
dc.subject.emtree | Molecularly targeted therapy | en_US |
dc.subject.emtree | Treatment outcome | en_US |
dc.subject.mesh | Abatacept | en_US |
dc.subject.mesh | Adaptor proteins, signal transducing | en_US |
dc.subject.mesh | Adolescent | en_US |
dc.subject.mesh | Adult | en_US |
dc.subject.mesh | Child | en_US |
dc.subject.mesh | Child preschool | en_US |
dc.subject.mesh | Female | en_US |
dc.subject.mesh | Humans | en_US |
dc.subject.mesh | Immunologic deficiency syndromes | en_US |
dc.subject.mesh | Immunosuppressive agents | en_US |
dc.subject.mesh | Male | en_US |
dc.subject.mesh | Molecular targeted therapy | en_US |
dc.subject.mesh | Treatment outcome | en_US |
dc.subject.mesh | Young adult | en_US |
dc.subject.scopus | Common Variable Immunodeficiency; Immunoglobulin Deficiency; Immunosuppression | en_US |
dc.subject.wos | Allergy | en_US |
dc.subject.wos | Immunology | en_US |
dc.title | Abatacept as a long-term targeted therapy for LRBA deficiency | en_US |
dc.type | Article | |
dc.wos.quartile | Q1 | en_US |
dspace.entity.type | Publication | |
local.contributor.department | Tıp Fakültesi/Dahili Bilimler/Çocuk Sağlığı ve Hastalıkları | tr_TR |
local.contributor.department | Tıp Fakültesi/Dahili Bilimler/Çocuk Sağlığı ve Hastalıkları | tr_TR |