Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases
Autoinflammatory diseases (AIDs) are heterogeneous disorders characterized by dysregulation in the inflammasome, a large intracellular multiprotein platform, leading to overproduction of interleukin-1(IL-1)β that plays a predominant pathogenic role in such diseases. Appropriate treatment is...
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doaj-91785438270f4a0ea2801bf0c3c2eb782020-11-25T00:49:17ZengMDPI AGInternational Journal of Molecular Sciences1422-00672019-04-01208189810.3390/ijms20081898ijms20081898Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory DiseasesAlessandra Bettiol0Giuseppe Lopalco1Giacomo Emmi2Luca Cantarini3Maria Letizia Urban4Antonio Vitale5Nunzio Denora6Antonio Lopalco7Annalisa Cutrignelli8Angela Lopedota9Vincenzo Venerito10Marco Fornaro11Alfredo Vannacci12Donato Rigante13Rolando Cimaz14Florenzo Iannone15Department of Neurosciences, Psychology, Pharmacology and Child Health (NEUROFARBA), University of Florence, 50139 Florence, ItalyDepartment of Emergency and Organ Transplantation, Rheumatology Unit, University of Bari, 70121 Bari, ItalyDepartment of Experimental and Clinical Medicine, University of Florence, 50134 Florence, ItalyDepartment of Medical Sciences, Surgery and Neurosciences, University of Siena, 53100 Siena, ItalyDepartment of Experimental and Clinical Medicine, University of Florence, 50134 Florence, ItalyDepartment of Medical Sciences, Surgery and Neurosciences, University of Siena, 53100 Siena, ItalyDepartment of Pharmacy-Drug Sciences, University of Bari “A. Moro”, 70125 Bari, ItalyDepartment of Pharmacy-Drug Sciences, University of Bari “A. Moro”, 70125 Bari, ItalyDepartment of Pharmacy-Drug Sciences, University of Bari “A. Moro”, 70125 Bari, ItalyDepartment of Pharmacy-Drug Sciences, University of Bari “A. Moro”, 70125 Bari, ItalyDepartment of Emergency and Organ Transplantation, Rheumatology Unit, University of Bari, 70121 Bari, ItalyDepartment of Emergency and Organ Transplantation, Rheumatology Unit, University of Bari, 70121 Bari, ItalyDepartment of Neurosciences, Psychology, Pharmacology and Child Health (NEUROFARBA), University of Florence, 50139 Florence, ItalyInstitute of Pediatrics, Fondazione Policlinico Universitario “A. Gemelli” IRCCS, 00168 Rome, ItalyDepartment of Neurosciences, Psychology, Drug Research and Child Health, Rheumatology Unit, Meyer Children’s Hospital, University of Florence, 50139 Florence, ItalyDepartment of Emergency and Organ Transplantation, Rheumatology Unit, University of Bari, 70121 Bari, ItalyAutoinflammatory diseases (AIDs) are heterogeneous disorders characterized by dysregulation in the inflammasome, a large intracellular multiprotein platform, leading to overproduction of interleukin-1(IL-1)β that plays a predominant pathogenic role in such diseases. Appropriate treatment is crucial, also considering that AIDs may persist into adulthood with negative consequences on patients’ quality of life. IL-1β blockade results in a sustained reduction of disease severity in most AIDs. A growing experience with the human IL-1 receptor antagonist, Anakinra (ANA), and the monoclonal anti IL-1β antibody, Canakinumab (CANA), has also been engendered, highlighting their efficacy upon protean clinical manifestations of AIDs. Safety and tolerability have been confirmed by several clinical trials and observational studies on both large and small cohorts of AID patients. The same treatment has been proposed in refractory Kawasaki disease, an acute inflammatory vasculitis occurring in children before 5 years, which has been postulated to be autoinflammatory for its phenotypical and immunological similarity with systemic juvenile idiopathic arthritis. Nevertheless, minor concerns about IL-1 antagonists have been raised regarding their employment in children, and the development of novel pharmacological formulations is aimed at minimizing side effects that may affect adherence to treatment. The present review summarizes current findings on the efficacy, safety, and tolerability of ANA and CANA for treatment of AIDs and Kawasaki vasculitis with a specific focus on the pediatric setting.https://www.mdpi.com/1422-0067/20/8/1898Interleukin-1anakinracanakinumabinnovative biotechnologiesautoinflammatory diseaseKawasaki diseasesystemic juvenile idiopathic arthritispersonalized medicinechildpediatrics |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Alessandra Bettiol Giuseppe Lopalco Giacomo Emmi Luca Cantarini Maria Letizia Urban Antonio Vitale Nunzio Denora Antonio Lopalco Annalisa Cutrignelli Angela Lopedota Vincenzo Venerito Marco Fornaro Alfredo Vannacci Donato Rigante Rolando Cimaz Florenzo Iannone |
spellingShingle |
Alessandra Bettiol Giuseppe Lopalco Giacomo Emmi Luca Cantarini Maria Letizia Urban Antonio Vitale Nunzio Denora Antonio Lopalco Annalisa Cutrignelli Angela Lopedota Vincenzo Venerito Marco Fornaro Alfredo Vannacci Donato Rigante Rolando Cimaz Florenzo Iannone Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases International Journal of Molecular Sciences Interleukin-1 anakinra canakinumab innovative biotechnologies autoinflammatory disease Kawasaki disease systemic juvenile idiopathic arthritis personalized medicine child pediatrics |
author_facet |
Alessandra Bettiol Giuseppe Lopalco Giacomo Emmi Luca Cantarini Maria Letizia Urban Antonio Vitale Nunzio Denora Antonio Lopalco Annalisa Cutrignelli Angela Lopedota Vincenzo Venerito Marco Fornaro Alfredo Vannacci Donato Rigante Rolando Cimaz Florenzo Iannone |
author_sort |
Alessandra Bettiol |
title |
Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases |
title_short |
Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases |
title_full |
Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases |
title_fullStr |
Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases |
title_full_unstemmed |
Unveiling the Efficacy, Safety, and Tolerability of Anti-Interleukin-1 Treatment in Monogenic and Multifactorial Autoinflammatory Diseases |
title_sort |
unveiling the efficacy, safety, and tolerability of anti-interleukin-1 treatment in monogenic and multifactorial autoinflammatory diseases |
publisher |
MDPI AG |
series |
International Journal of Molecular Sciences |
issn |
1422-0067 |
publishDate |
2019-04-01 |
description |
Autoinflammatory diseases (AIDs) are heterogeneous disorders characterized by dysregulation in the inflammasome, a large intracellular multiprotein platform, leading to overproduction of interleukin-1(IL-1)β that plays a predominant pathogenic role in such diseases. Appropriate treatment is crucial, also considering that AIDs may persist into adulthood with negative consequences on patients’ quality of life. IL-1β blockade results in a sustained reduction of disease severity in most AIDs. A growing experience with the human IL-1 receptor antagonist, Anakinra (ANA), and the monoclonal anti IL-1β antibody, Canakinumab (CANA), has also been engendered, highlighting their efficacy upon protean clinical manifestations of AIDs. Safety and tolerability have been confirmed by several clinical trials and observational studies on both large and small cohorts of AID patients. The same treatment has been proposed in refractory Kawasaki disease, an acute inflammatory vasculitis occurring in children before 5 years, which has been postulated to be autoinflammatory for its phenotypical and immunological similarity with systemic juvenile idiopathic arthritis. Nevertheless, minor concerns about IL-1 antagonists have been raised regarding their employment in children, and the development of novel pharmacological formulations is aimed at minimizing side effects that may affect adherence to treatment. The present review summarizes current findings on the efficacy, safety, and tolerability of ANA and CANA for treatment of AIDs and Kawasaki vasculitis with a specific focus on the pediatric setting. |
topic |
Interleukin-1 anakinra canakinumab innovative biotechnologies autoinflammatory disease Kawasaki disease systemic juvenile idiopathic arthritis personalized medicine child pediatrics |
url |
https://www.mdpi.com/1422-0067/20/8/1898 |
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