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Abatacept as a Long-Term Targeted Therapy for LRBA Deficiency

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dc.contributor.authorÖZEN, AHMET OĞUZHAN
dc.contributor.authorsKiykim, Ayca; Ogulur, Ismail; Dursun, Esra; Charbonnier, Louis Marie; Nain, Ercan; Cekic, Sukru; Dogruel, Dilek; Karaca, Neslihan Edeer; Cogurlu, Mujde Tuba; Bilir, Ozlem Arman; Cansever, Murat; Kapakli, Hasan; Baser, Dilek; Kasap, Nurhan; Kutlug, Seyhan; Altintas, Derya Ufuk; Al-Shaibi, Ahmad; Agrebi, Nourhen; Kara, Manolya; Guven, Ayla; Somer, Ayper; Aydogmus, Cigdem; Ayaz, Nuray Aktay; Metin, Ayse; Aydogan, Metin; Uncuoglu, Aysen; Patiroglu, Turkan; Yildiran, Alisan; Guner, Sukru Nail; Keles, Sevgi; Reisli, Ismail; Aksu, Guzide; Kutukculer, Necil; Kilic, Sara S.; Yilmaz, Mustafa; Karakoc-Aydiner, Elif; Lo, Bernice; Ozen, Ahmet; Chatila, Talal A.; Baris, Safa
dc.date.accessioned2022-03-14T10:04:17Z
dc.date.available2022-03-14T10:04:17Z
dc.date.issued2019-11
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. (C) 2019 American Academy of Allergy, Asthma & Immunology
dc.identifier.doi10.1016/j.jaip.2019.06.011
dc.identifier.eissn2213-2201
dc.identifier.issn2213-2198
dc.identifier.pubmed31238161
dc.identifier.urihttps://hdl.handle.net/11424/243999
dc.identifier.wosWOS:000495746100038
dc.language.isoeng
dc.publisherELSEVIER
dc.relation.ispartofJOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectLPS-responsive beige-like anchor
dc.subjectImmune dysregulation
dc.subjectAbatacept
dc.subjectT follicular helper cells
dc.subjectAutoimmunity
dc.subjectIMMUNE DYSREGULATION
dc.subjectCTLA-4 CHECKPOINT
dc.subjectMUTATIONS
dc.subjectPOLYENDOCRINOPATHY
dc.subjectENTEROPATHY
dc.subjectDISEASE
dc.titleAbatacept as a Long-Term Targeted Therapy for LRBA Deficiency
dc.typearticle
dspace.entity.typePublication
local.avesis.idd4bf9ce5-4820-40a5-8334-9e1d5ae07645
local.import.packageSS16
local.indexed.atWOS
local.indexed.atSCOPUS
local.indexed.atPUBMED
local.journal.numberofpages26
local.journal.quartileQ1
oaire.citation.endPage+
oaire.citation.issue8
oaire.citation.startPage2790
oaire.citation.titleJOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE
oaire.citation.volume7
relation.isAuthorOfPublication3e9c297b-e636-4836-8f61-dc9c8b7c29cf
relation.isAuthorOfPublication.latestForDiscovery3e9c297b-e636-4836-8f61-dc9c8b7c29cf

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