Literature DB >> 36050429

Three Adult Cases of STAT1 Gain-of-Function with Chronic Mucocutaneous Candidiasis Treated with JAK Inhibitors.

Emilie W Borgström1,2, Marie Edvinsson3, Lucía P Pérez4, Anna C Norlin5,6, Sara L Enoksson6, Susanne Hansen5, Anders Fasth7, Vanda Friman8, Olle Kämpe9, Robert Månsson4, Hernando Y Estupiñán4,10, Qing Wang4, Tan Ziyang11, Tadepally Lakshmikanth11, Carl Inge E Smith5,12, Petter Brodin11,13, Peter Bergman14,5.   

Abstract

PURPOSE: The aim of this study was to characterize clinical effects and biomarkers in three patients with chronic mucocutaneous candidiasis (CMC) caused by gain-of-function (GOF) mutations in the STAT1 gene during treatment with Janus kinase (JAK) inhibitors.
METHODS: Mass cytometry (CyTOF) was used to characterize mononuclear leukocyte populations and Olink assay to quantify 265 plasma proteins. Flow-cytometric Assay for Specific Cell-mediated Immune-response in Activated whole blood (FASCIA) was used to quantify the reactivity against Candida albicans.
RESULTS: Overall, JAK inhibitors improved clinical symptoms of CMC, but caused side effects in two patients. Absolute numbers of neutrophils, T cells, B cells, and NK cells were sustained during baricitinib treatment. Detailed analysis of cellular subsets, using CyTOF, revealed increased expression of CD45, CD52, and CD99 in NK cells, reflecting a more functional phenotype. Conversely, monocytes and eosinophils downregulated CD16, consistent with reduced inflammation. Moreover, T and B cells showed increased expression of activation markers during treatment. In one patient with a remarkable clinical effect of baricitinib treatment, the immune response to C. albicans increased after 7 weeks of treatment. Alterations in plasma biomarkers involved downregulation of cellular markers CXCL10, annexin A1, granzyme B, granzyme H, and oncostatin M, whereas FGF21 was the only upregulated marker after 7 weeks. After 3 months, IFN-ɣ and CXCL10 were downregulated.
CONCLUSIONS: The clinical effect of JAK inhibitor treatment of CMC is promising. Several biological variables were altered during baricitinib treatment demonstrating that lymphocytes, NK cells, monocytes, and eosinophils were affected. In parallel, cellular reactivity against C. albicans was enhanced.
© 2022. The Author(s).

Entities:  

Keywords:  JAK inhibitor; Olink; STAT1; chronic mucocutaneous candidiasis; mass cytometry

Year:  2022        PMID: 36050429     DOI: 10.1007/s10875-022-01351-0

Source DB:  PubMed          Journal:  J Clin Immunol        ISSN: 0271-9142            Impact factor:   8.542


  59 in total

Review 1.  Stats: transcriptional control and biological impact.

Authors:  David E Levy; J E Darnell
Journal:  Nat Rev Mol Cell Biol       Date:  2002-09       Impact factor: 94.444

2.  Interleukins 1beta and 6 but not transforming growth factor-beta are essential for the differentiation of interleukin 17-producing human T helper cells.

Authors:  Eva V Acosta-Rodriguez; Giorgio Napolitani; Antonio Lanzavecchia; Federica Sallusto
Journal:  Nat Immunol       Date:  2007-08-05       Impact factor: 25.606

3.  STAT1 Gain-of-Function and Dominant Negative STAT3 Mutations Impair IL-17 and IL-22 Immunity Associated with CMC.

Authors:  Julia Hiller; Beate Hagl; Renate Effner; Anne Puel; Martin Schaller; Birgit Mascher; Stefanie Eyerich; Kilian Eyerich; Annette F Jansson; Johannes Ring; Jean-Laurent Casanova; Ellen D Renner; Claudia Traidl-Hoffmann
Journal:  J Invest Dermatol       Date:  2017-10-18       Impact factor: 8.551

4.  Gain-of-function STAT1 mutations are associated with intracranial aneurysms.

Authors:  Mete Dadak; Roland Jacobs; Jelena Skuljec; Adan Chari Jirmo; Özlem Yildiz; Frank Donnerstag; Niklas Thomas Baerlecken; Reinhold Ernst Schmidt; Heinrich Lanfermann; Thomas Skripuletz; Philipp Schwenkenbecher; Christoph Kleinschnitz; Hayrettin Tumani; Martin Stangel; Refik Pul
Journal:  Clin Immunol       Date:  2017-02-02       Impact factor: 3.969

Review 5.  Inborn errors of human JAKs and STATs.

Authors:  Jean-Laurent Casanova; Steven M Holland; Luigi D Notarangelo
Journal:  Immunity       Date:  2012-04-20       Impact factor: 31.745

Review 6.  STAT1 and STAT3 mutations: important lessons for clinical immunologists.

Authors:  Peter Olbrich; Alexandra F Freeman
Journal:  Expert Rev Clin Immunol       Date:  2018-10-25       Impact factor: 4.473

Review 7.  Human STAT1 Gain-of-Function Heterozygous Mutations: Chronic Mucocutaneous Candidiasis and Type I Interferonopathy.

Authors:  Satoshi Okada; Takaki Asano; Kunihiko Moriya; Stephanie Boisson-Dupuis; Masao Kobayashi; Jean-Laurent Casanova; Anne Puel
Journal:  J Clin Immunol       Date:  2020-08-27       Impact factor: 8.317

8.  IL-21 initiates an alternative pathway to induce proinflammatory T(H)17 cells.

Authors:  Thomas Korn; Estelle Bettelli; Wenda Gao; Amit Awasthi; Anneli Jäger; Terry B Strom; Mohamed Oukka; Vijay K Kuchroo
Journal:  Nature       Date:  2007-06-20       Impact factor: 49.962

9.  Gain-of-function human STAT1 mutations impair IL-17 immunity and underlie chronic mucocutaneous candidiasis.

Authors:  Luyan Liu; Satoshi Okada; Xiao-Fei Kong; Alexandra Y Kreins; Sophie Cypowyj; Avinash Abhyankar; Julie Toubiana; Yuval Itan; Magali Audry; Patrick Nitschke; Cécile Masson; Beata Toth; Jérome Flatot; Mélanie Migaud; Maya Chrabieh; Tatiana Kochetkov; Alexandre Bolze; Alessandro Borghesi; Antoine Toulon; Julia Hiller; Stefanie Eyerich; Kilian Eyerich; Vera Gulácsy; Ludmyla Chernyshova; Viktor Chernyshov; Anastasia Bondarenko; Rosa María Cortés Grimaldo; Lizbeth Blancas-Galicia; Ileana Maria Madrigal Beas; Joachim Roesler; Klaus Magdorf; Dan Engelhard; Caroline Thumerelle; Pierre-Régis Burgel; Miriam Hoernes; Barbara Drexel; Reinhard Seger; Theresia Kusuma; Annette F Jansson; Julie Sawalle-Belohradsky; Bernd Belohradsky; Emmanuelle Jouanguy; Jacinta Bustamante; Mélanie Bué; Nathan Karin; Gizi Wildbaum; Christine Bodemer; Olivier Lortholary; Alain Fischer; Stéphane Blanche; Saleh Al-Muhsen; Janine Reichenbach; Masao Kobayashi; Francisco Espinosa Rosales; Carlos Torres Lozano; Sara Sebnem Kilic; Matias Oleastro; Amos Etzioni; Claudia Traidl-Hoffmann; Ellen D Renner; Laurent Abel; Capucine Picard; László Maródi; Stéphanie Boisson-Dupuis; Anne Puel; Jean-Laurent Casanova
Journal:  J Exp Med       Date:  2011-07-04       Impact factor: 14.307

10.  Transcriptional Profiling of STAT1 Gain-of-Function Reveals Common and Mutation-Specific Fingerprints.

Authors:  Simone Giovannozzi; Jonas Demeulemeester; Rik Schrijvers; Rik Gijsbers
Journal:  Front Immunol       Date:  2021-02-17       Impact factor: 7.561

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.