Therapeutic modalities and clinical outcomes in a large cohort with LRBA deficiency and CTLA4 insufficiency

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dc.contributor.authorTaghizade, Nigar
dc.contributor.authorBabayeva, Royala
dc.contributor.authorKara, Altan
dc.contributor.authorKarakus, Ibrahim Serhat
dc.contributor.authorCatak, Mehmet Cihangir
dc.contributor.authorBulutoglu, Alper
dc.contributor.authorHaskologlu, Zehra Sule
dc.date.accessioned2024-02-23T14:12:30Z
dc.date.available2024-02-23T14:12:30Z
dc.date.issued2023
dc.departmentNEÜen_US
dc.description.abstractBackground: LPS-responsive beige-like anchor (LRBA) deficiency (LRBA-/-) and cytotoxic T-lymphocyte-associated antigen-4 (CTLA4) insufficiency (CTLA41/-) are mechanistically overlapped diseases presenting with recurrent infections and autoimmunity. The effectiveness of different treatment regimens remains unknown. Objective: Our aim was to determine the comparative efficacy and long-term outcome of therapy with immunosuppressants, CTLA4-immunoglobulin (abatacept), and hematopoietic stem cell transplantation (HSCT) in a single-country multicenter cohort of 98 patients with a 5-year median follow-up.Methods: The 98 patients (63 LRBA-/- and 35 CTLA41/-) were followed and evaluated at baseline and every 6 months for clinical manifestations and response to the respective therapies.Results: The LRBA-/- patients exhibited a more severe disease course than did the CTLA41/- patients, requiring more immunosuppressants, abatacept, and HSCT to control their symptoms. Among the 58 patients who received abatacept as either a primary or rescue therapy, sustained complete control was achieved in 46 (79.3%) without severe side effects. In contrast, most patients who received immunosuppressants as primary therapy (n = 61) showed either partial or no disease control (72.1%), necessitating additional immunosuppressants, abatacept, or transplantation. Patients with partial or no response to abatacept (n = 12) had longer disease activity before abatacept therapy, with higher organ involvement and poorer disease outcomes than those with a complete response. HSCT was performed in 14 LRBA-/- patients; 9 patients (64.2%)showed complete remission , 3 (21.3%) continued to receive immunosuppressants after transplantation. HSCT , abatacept therapy gave rise to similar probabilities of survival. Conclusions: Abatacept is superior to immunosuppressants in controlling disease manifestations over the long term, especially when started early, and it may provide a safe and effective therapeutic alternative to transplantation. (J Allergy Clin Immunol 2023;152:1634-45.)en_US
dc.identifier.doi10.1016/j.jaci.2023.08.004
dc.identifier.endpage1645en_US
dc.identifier.issn0091-6749
dc.identifier.issn1097-6825
dc.identifier.issue6en_US
dc.identifier.pmid37595759en_US
dc.identifier.scopus2-s2.0-85172936642en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage1634en_US
dc.identifier.urihttps://doi.org/10.1016/j.jaci.2023.08.004
dc.identifier.urihttps://hdl.handle.net/20.500.12452/12087
dc.identifier.volume152en_US
dc.identifier.wosWOS:001133672100001en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherMosby-Elsevieren_US
dc.relation.ispartofJournal Of Allergy And Clinical Immunologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectInborn Errors Of Immunityen_US
dc.subjectLps-Responsive Beige-Like Anchoren_US
dc.subjectCytotoxic T-Lymphocyte-Associated Antigen-4en_US
dc.subjectImmune Dysre-Gulationen_US
dc.subjectNatural Historyen_US
dc.subjectAbatacepten_US
dc.subjectHematopoietic Stem Cell Transplantationen_US
dc.titleTherapeutic modalities and clinical outcomes in a large cohort with LRBA deficiency and CTLA4 insufficiencyen_US
dc.typeArticleen_US

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