Literature DB >> 33442967

Primary Immune Regulatory Disorders and Targeted Therapies

Burcu Kolukısa1,2,3, Safa Barış1,2,3.   

Abstract

Primary immune regulatory disorders (PIRDs) are a group of diseases belonging to inborn errors of immunity. They usually exhibit lymphoproliferation, autoimmunities, and malignancies, with less susceptibility to recurrent infections. Unlike classical primary immune deficiencies, in autoimmune manifestations, such as cytopenias, enteropathy can be the first symptom of diseases, and they are typically resistant to treatment. Increasing awareness of PIRDs among specialists and a multidisciplinary team approach would provide early diagnosis and treatment that could prevent end-organ damage related to the diseases. In recent years, many PIRDs have been described, and understanding the immunological pathways linked to these disorders provides us an opportunity to use directed therapies for specific molecules, which usually offer better disease control than known classical immunosuppressants. In this review, in light of the most recent literature, we will discuss the common PIRDs and explain their clinical symptoms and recent treatment modalities.

Entities:  

Keywords:  Primary immune deficiencies; Immune dysregulation; Autoimmunity; Lymphoproliferation; Precision medicine; Targeted therapy

Mesh:

Substances:

Year:  2021        PMID: 33442967      PMCID: PMC7927447          DOI: 10.4274/tjh.galenos.2021.2020.0724

Source DB:  PubMed          Journal:  Turk J Haematol        ISSN: 1300-7777            Impact factor:   1.831


  91 in total

1.  Familial haemophagocytic reticulosis.

Authors:  J W FARQUHAR; A E CLAIREAUX
Journal:  Arch Dis Child       Date:  1952-12       Impact factor: 3.791

2.  Impaired natural killer cell functions in patients with signal transducer and activator of transcription 1 (STAT1) gain-of-function mutations.

Authors:  Giovanna Tabellini; Donatella Vairo; Omar Scomodon; Nicola Tamassia; Rosalba Monica Ferraro; Ornella Patrizi; Sara Gasperini; Annarosa Soresina; Giuliana Giardino; Claudio Pignata; Vassilios Lougaris; Alessandro Plebani; Laura Dotta; Marco A Cassatella; Silvia Parolini; Raffaele Badolato
Journal:  J Allergy Clin Immunol       Date:  2017-01-06       Impact factor: 10.793

Review 3.  CHAI and LATAIE: new genetic diseases of CTLA-4 checkpoint insufficiency.

Authors:  Bernice Lo; Jill M Fritz; Helen C Su; Gulbu Uzel; Michael B Jordan; Michael J Lenardo
Journal:  Blood       Date:  2016-07-14       Impact factor: 22.113

4.  Ruxolitinib for refractory/relapsed hemophagocytic lymphohistiocytosis.

Authors:  Jingshi Wang; Yini Wang; Lin Wu; Xinkai Wang; Zhili Jin; Zhuo Gao; Zhao Wang
Journal:  Haematologica       Date:  2019-09-12       Impact factor: 9.941

5.  Signal transducer and activator of transcription 1 (STAT1) gain-of-function mutations and disseminated coccidioidomycosis and histoplasmosis.

Authors:  Elizabeth P Sampaio; Amy P Hsu; Joseph Pechacek; Hannelore I Bax; Dalton L Dias; Michelle L Paulson; Prabha Chandrasekaran; Lindsey B Rosen; Daniel S Carvalho; Li Ding; Donald C Vinh; Sarah K Browne; Shrimati Datta; Joshua D Milner; Douglas B Kuhns; Debra A Long Priel; Mohammed A Sadat; Michael Shiloh; Brendan De Marco; Michael Alvares; Jason W Gillman; Vivek Ramarathnam; Maite de la Morena; Liliana Bezrodnik; Ileana Moreira; Gulbu Uzel; Daniel Johnson; Christine Spalding; Christa S Zerbe; Henry Wiley; David E Greenberg; Susan E Hoover; Sergio D Rosenzweig; John N Galgiani; Steven M Holland
Journal:  J Allergy Clin Immunol       Date:  2013-03-28       Impact factor: 10.793

Review 6.  Mechanism-Based Strategies for the Management of Autoimmunity and Immune Dysregulation in Primary Immunodeficiencies.

Authors:  Jolan E Walter; Jocelyn R Farmer; Zsofia Foldvari; Troy R Torgerson; Megan A Cooper
Journal:  J Allergy Clin Immunol Pract       Date:  2016 Nov - Dec

7.  Clinical, Immunologic, and Molecular Spectrum of Patients with LPS-Responsive Beige-Like Anchor Protein Deficiency: A Systematic Review.

Authors:  Sima Habibi; Majid Zaki-Dizaji; Hosein Rafiemanesh; Bernice Lo; Mahnaz Jamee; Laura Gámez-Díaz; Fereshte Salami; Ali N Kamali; Hamed Mohammadi; Hassan Abolhassani; Reza Yazdani; Asghar Aghamohammadi; Juan-Manuel Anaya; Gholamreza Azizi
Journal:  J Allergy Clin Immunol Pract       Date:  2019-04-14

Review 8.  PI3K pathway defects leading to immunodeficiency and immune dysregulation.

Authors:  Cristiane J Nunes-Santos; Gulbu Uzel; Sergio D Rosenzweig
Journal:  J Allergy Clin Immunol       Date:  2019-05       Impact factor: 10.793

9.  Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis.

Authors:  Franco Locatelli; Michael B Jordan; Carl Allen; Simone Cesaro; Carmelo Rizzari; Anupama Rao; Barbara Degar; Timothy P Garrington; Julian Sevilla; Maria-Caterina Putti; Franca Fagioli; Martina Ahlmann; Jose-Luis Dapena Diaz; Michael Henry; Fabrizio De Benedetti; Alexei Grom; Genevieve Lapeyre; Philippe Jacqmin; Maria Ballabio; Cristina de Min
Journal:  N Engl J Med       Date:  2020-05-07       Impact factor: 91.245

10.  Multiple Presentations of LRBA Deficiency: a Single-Center Experience.

Authors:  Sevgi Kostel Bal; Sule Haskologlu; Nina K Serwas; Candan Islamoglu; Caner Aytekin; Tanil Kendirli; Zarife Kuloglu; Gulsan Yavuz; Buket Dalgic; Zeynep Siklar; Aydan Kansu; Arzu Ensari; Kaan Boztug; Figen Dogu; Aydan Ikinciogullari
Journal:  J Clin Immunol       Date:  2017-09-27       Impact factor: 8.317

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