Article
Immunology
Zhou Shu, Yue Zhang, Tongxin Han, Yan Li, Yurong Piao, Fei Sun, Jin Ma, Wenxiu Mo, Jiapeng Sun, Koon-Wing Chan, Wanling Yang, Yu-Lung Lau, Huawei Mao
Summary: Cryopyrin-associated periodic syndrome (CAPS) is a group of disorders characterized by recurrent bouts of systemic inflammation related to overactivation of the inflammasome. In this study, the clinical and genetic characteristics of 30 CAPS patients in China were investigated. The researchers identified novel mutations and analyzed the treatment outcomes of these patients. The findings provide guidance for future diagnosis and management of CAPS.
FRONTIERS IN IMMUNOLOGY
(2023)
Review
Rheumatology
Yuichi Yamasaki, Tomohiro Kubota, Syuji Takei, Hiroyuki Imanaka, Yukiko Nonaka, Yoshifumi Kawano
Summary: CAPS is a disorder characterized by unregulated interleukin-1 beta production due to mutations in the NLRP3 gene, with early-onset episodes of fever and rash. Effective therapies targeting interleukin-1 have emerged, but a case of CAPS patient developing inflammatory bowel disease during canakinumab therapy highlights the need to consider possible co-morbidities in patients with symptoms unresponsive to canakinumab.
CLINICAL RHEUMATOLOGY
(2021)
Article
Rheumatology
K. Hosono, C. Kato, T. Sasajima
Summary: The study aimed to evaluate the safety and effectiveness of canakinumab in CAPS patients. The interim results showed no new safety concerns and sustained remission in the majority of patients in a real-world setting.
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
(2022)
Article
Rheumatology
Eric Y. Yen, Devanshu R. Singh, Ram Raj Singh
Summary: This study aimed to assess the mortality of SSc in the general population by age over the past five decades. By using a national mortality database and census data, the proportions and age-standardized mortality rates (ASMRs) of SSc and non-SSc were calculated and compared by age groups. The results showed that the mortality for SSc has steadily decreased at younger ages, while it has steeply increased among the elderly population.
Article
Pediatrics
Anne M. Hause, David K. Shay, Nicola P. Klein, Winston E. Abara, James Baggs, Margaret M. Cortese, Bruce Fireman, Julianne Gee, Jason M. Glanz, Kristin Goddard, Kayla E. Hanson, Brandon Hugueley, Tat'Yana Kenigsberg, Elyse O. Kharbanda, Bruno Lewin, Ned Lewis, Paige Marquez, Tanya Myers, Allison Naleway, Jennifer C. Nelson, John R. Su, Deborah Thompson, Babatunde Olubajo, Matthew E. Oster, Eric S. Weintraub, Joshua T. B. Williams, Anna R. Yousaf, Ousseny Zerbo, Bicheng Zhang, Tom T. Shimabukuro
Summary: Limited postauthorization safety data for the Pfizer-BioNTech coronavirus disease 2019 vaccination among children ages 5 to 11 years are available. Most adverse events reported were mild and no safety signals were observed in active surveillance. The reporting rate of myocarditis after dose 2 in this age group was substantially lower than that observed among adolescents ages 12 to 15 years.
Article
Rheumatology
Ulrich A. Walker, Hugh H. Tilson, Philip N. Hawkins, Tom van der Poll, Stephanie Noviello, Jeremy Levy, Eleni Vritzali, Hal M. Hoffman, Jasmin B. Kuemmerle-Deschner
Summary: This study reported the long-term safety and effectiveness of canakinumab in patients with CAPS, showing sustained response over 6 years with a favorable safety profile. The drug demonstrated significant efficacy in reducing disease activity in CAPS patients.
Letter
Dermatology
Delin Ran, Baoqi Yang, Lulu Sun, Na Wang, Peng Qu, Jing Liu, Futang Pan, Guangjin Wang, Weizhi Wu, Zhaoxia Zhang, Zhongxiang Shi, Qing Yang, Hong Liu, Furen Zhang
Summary: Generalized pustular psoriasis (GPP) is a rare skin disease characterized by widespread sterile pustules and potential severe systemic symptoms. We present our experience of using spesolimab to treat GPP flare-ups in five Chinese patients.
CLINICAL AND EXPERIMENTAL DERMATOLOGY
(2023)
Review
Food Science & Technology
Zahra Golchinfar, Parastou Farshi, Maryam Mahmoudzadeh, Maryam Mohammadi, Mahnaz Tabibiazar, J. Scott Smith
Summary: This article reviews the factors affecting the formation of advanced glycation end product CML in the human body and food products, and discusses the biological fate of dietary CML.
FOOD REVIEWS INTERNATIONAL
(2023)
Article
Medicine, General & Internal
Nicole Toepfner, Wolfgang C. G. von Meissner, Christoph Strumann, Denisa Drinka, David Stuppe, Maximilian Jorczyk, Jeanne Moor, Johannes Puschel, Melanie Liss, Emilie von Poblotzki, Reinhard Berner, Matthias B. Moor, Cho-Ming Chao
Summary: This retrospective cohort study evaluated the safety of off-label BNT162b2 vaccine use in children younger than 5 years. The study found that the reported symptoms after BNT162b2 administration were comparable to those for non-SARS-CoV-2 vaccines in this age group.
Article
Rheumatology
Marco Gattorno, Laura Obici, Inmaculada Calvo Penades, Tilmann Kallinich, Susanne Benseler, Elise Dekker, Jeremy Levy, Fabrizio De Benedetti, Helen Lachmann
Summary: This study assessed the efficacy, safety, and tolerability of canakinumab in TRAPS patients during a 72-week long-term, open-label extension. The results showed that long-term canakinumab treatment effectively controlled disease activity and reduced flare incidence in TRAPS patients. No new safety concerns were identified.
ARTHRITIS & RHEUMATOLOGY
(2023)
Article
Pediatrics
Jessica L. Schue, Janneke H. van Dijk, Francis Hamangaba, Mutinta Hamahuwa, Nkumbula Moyo, Philip E. Thuma, William J. Moss, Catherine G. Sutcliffe
Summary: In Zambia, most children under 5 years old who initiated antiretroviral treatment responded well, but many remained underweight and stunted, and faced high mortality rates in the early stages of treatment.
Article
Pharmacology & Pharmacy
Pravin Hissaria, Kevinkumar Kansagra, Hardik Patel, Taufik Momin, Ashok Ghoghari, Harilal Patel, Bhavesh Sharma
Summary: In this proof-of-concept phase 2a study, we investigated the use of an oral NLRP3 inflammasome inhibitor in subjects with cryopyrin-associated periodic syndromes (CAPS). Three adult subjects with confirmed CAPS were treated with 50 mg of ZYIL1 twice daily for 7 days. The results showed mild treatment-emergent adverse events in 2 of the subjects, but these were unrelated to the study drug. Furthermore, inflammatory markers and disease activity significantly decreased 12 hours after the last dose.
CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT
(2023)
Article
Orthopedics
Walid Bouaicha, Mohamed Jlidi, Marouen Elarbi, Karim Mallek, Salma Jaziri, Achraf Abdennadher, Selim Daas
Summary: This study evaluated and compared three fixation modalities (CHS, DHS, and DHS with anti-rotating screw) for the treatment of femoral neck fracture in young adults. The results showed comparable healing and complication rates among the three groups, but the CHS group had significantly better HOOS scores.
INTERNATIONAL ORTHOPAEDICS
(2023)
Article
Multidisciplinary Sciences
Alistair W. Vickery, Sebastian Roth, Tracie Ernenwein, Jessica Kennedy, Patrizia Washer
Summary: This study analyzed a large cohort of Australian patients and found that oral medicinal cannabis is safe and well-tolerated for a sustained period. The results showed significant improvements in clinical outcomes over two years of treatment.
Article
Public, Environmental & Occupational Health
Lusine Ghazaryan, Kuangnan Xiong, Charlan Kroelinger, Kristin Rankin, Olivia Sappenfield, Marilyn Kacica
Summary: This study provides insights into the epidemiology of child maltreatment hospitalizations among children aged 17 and younger in New York State. It found that infants had the highest hospitalization rates, and physical abuse was the most prevalent type of maltreatment reported.
MATERNAL AND CHILD HEALTH JOURNAL
(2022)
Article
Biochemistry & Molecular Biology
Clive Hoggart, Chisato Shimizu, Rachel Galassini, Victoria J. Wright, Hannah Shailes, Evan Bellos, Jethro A. Herberg, Andrew J. Pollard, Daniel O'Connor, Shing Wan Choi, Eleanor G. Seaby, Stephanie Menikou, Martin Hibberd, Neneh Sallah, David Burgner, Paul Brogan, Harsita Patel, Jihoon Kim, Adriana H. Tremoulet, Eeva Salo, Diana van Stijn, Taco Kuijpers, Jane C. Burns, Michael Levin
Summary: The study identified a novel locus for CAA in KD patients of European descent and confirmed the association of susceptibility loci such as ITPKC and FCGR2A with KD. Meta-analysis with Japanese GWAS validated consistent effects of these loci across different populations.
EUROPEAN JOURNAL OF HUMAN GENETICS
(2021)
Article
Rheumatology
Paul A. Brogan, Barbara Arch, Helen Hickey, Jordi Anton, Este Iglesias, Eileen Baildam, Kamran Mahmood, Gavin Cleary, Elena Moraitis, Charalampia Papadopoulou, Michael W. Beresford, Phil Riley, Selcan Demir, Seza Ozen, Giovanna Culeddu, Dyfrig A. Hughes, Pavla Dolezalova, Lisa Hampson, John Whitehead, David Jayne, Nicola Ruperto, Despina Eleftheriou
Summary: The study showed that the effectiveness of CYC and MMF treatment for remission induction in childhood PAN is similar, but MMF may be associated with better health-related quality of life. Patients in the MMF group achieved the primary endpoint in a shorter time, with no relapses occurring in either group within 18 months.
ARTHRITIS & RHEUMATOLOGY
(2021)
Article
Rheumatology
Paul Brogan, Rae S. M. Yeung, Gavin Cleary, Satyapal Rangaraj, Ozgur Kasapcopur, Aimee O. Hersh, Suzanne Li, Dusan Paripovic, Kenneth Schikler, Andrew Zeft, Claudia Bracaglia, Despina Eleftheriou, Pooneh Pordeli, Simone Melega, Candice Jamois, Jacques Gaudreault, Margaret Michalska, Paul Brunetta, Jennifer C. Cooper, Patricia B. Lehane
Summary: In this study, 25 pediatric patients with new-onset or relapsing GPA or MPA completed the remission-induction phase with RTX treatment. Most patients experienced mild to moderate infusion-related adverse events, and the study results showed that RTX is well tolerated and effective in pediatric patients with GPA or MPA.
ARTHRITIS & RHEUMATOLOGY
(2022)
Article
Rheumatology
Marta Bustaffa, Isabelle Kone-Paut, Seza Ozen, Gayane Amaryan, Efimia Papadopoulou-Alataki, Romina Gallizzi, Maria Carrabba, Yonatan Butbul Aviel, Luca Cantarini, Maria Alessio, Jordi Anton, Laura Obici, Faysal Gok, Ezgi Deniz Batu, Estefania Moreno, Paul Brogan, Maria Trachana, Gabriele Simonini, Donato Rigante, Yosef Uziel, Antonella Insalaco, Maria Cristina Maggio, Nicolino Ruperto, Marco Gattorno, L. Rossi Semerano
Summary: The new EPCC and INSAID classification of MEFV variants can identify two distinct groups of FMF patients, which differ in clinical characteristics, therapeutic approach, and response to treatment.
SEMINARS IN ARTHRITIS AND RHEUMATISM
(2022)
Article
Rheumatology
Kader Cetin Gedik, Lovro Lamot, Micol Romano, Erkan Demirkaya, David Piskin, Sofia Torreggiani, Laura A. Adang, Thais Armangue, Kathe Barchus, Devon R. Cordova, Yanick J. Crow, Russell C. Dale, Karen L. Durrant, Despina Eleftheriou, Elisa M. Fazzi, Marco Gattorno, Francesco Gavazzi, Eric P. Hanson, Min Ae Lee-Kirsch, Gina A. Montealegre Sanchez, Benedicte Neven, Simona Orcesi, Seza Ozen, M. Cecilia Poli, Elliot Schumacher, Davide Tonduti, Katsiaryna Uss, Daniel Aletaha, Brian M. Feldman, Adeline Vanderver, Paul A. Brogan, Raphaela Goldbach-Mansky
Summary: This study aims to improve the diagnosis, treatment, and long-term monitoring of patients with autoinflammatory interferonopathies, CANDLE/PRAAS, SAVI, and AGS. The research team developed guidelines and considerations to enhance care quality and disease outcomes.
ARTHRITIS & RHEUMATOLOGY
(2022)
Article
Immunology
Ying Hong, Marina Casimir, Benjamin C. Houghton, Fang Zhang, Barbara Jensen, Ebun Omoyinmi, Robert Torrance, Charalampia Papadopoulou, Michelle Cummins, Marion Roderick, Adrian J. Thrasher, Paul A. Brogan, Despina Eleftheriou
Summary: Lentivirus-mediated ADA2 gene correction can ameliorate the immune cell damage and vasculitis in DADA2 patients, and restore the function of hematopoietic stem cells in patients with pure red cell aplasia.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Pediatrics
Ovgu Kul Cinar, Amber Putland, Karen Wynne, Despina Eleftheriou, Paul A. Brogan
Summary: Hereditary systemic autoinflammatory diseases (SAIDs) are rare and severe conditions characterized by mutations in key regulators of the immune system. Through translational research, we have gained a better understanding of these diseases and developed targeted treatments.
FRONTIERS IN PEDIATRICS
(2022)
Article
Rheumatology
Simone Melega, Paul Brogan, Gavin Cleary, Aimee O. Hersh, Ozgur Kasapcopur, Satyapal Rangaraj, Rae S. M. Yeung, Andrew Zeft, Jennifer Cooper, Pooneh Pordeli, Petra Kirchner, Patricia B. Lehane
Summary: This study assessed the impact of prolonged low immunoglobulin levels on the risk of serious infection in pediatric patients receiving rituximab treatment. The results showed that the majority of patients did not experience serious infections, and the infections that did occur were manageable.
RHEUMATOLOGY AND THERAPY
(2022)
Meeting Abstract
Rheumatology
F. De Benedetti, A. Grom, P. Brogan, C. Bracaglia, M. Pardeo, G. Marucci, D. Eleftheriou, C. Papadopoulou, P. Quartier, J. Anton, R. Frederiksen, V. Asnaghi, C. de Min
ANNALS OF THE RHEUMATIC DISEASES
(2022)
Article
Rheumatology
Paul Brogan, Ray Naden, Stacy P. Ardoin, Jennifer C. Cooper, Fabrizio De Benedetti, Jean-Francois Dicaire, Despina Eleftheriou, Brian Feldman, Jon Goldin, Seth E. Karol, Fiona Price-Kuehne, David Skuse, Constantine A. Stratakis, Nicholas Webb, John H. Stone
Summary: The study developed a Pediatric Glucocorticoid Toxicity Index (pGTI) as a weighted clinical assessment tool to measure changes in GC toxicity over time. It includes toxicity assessment and a Damage Checklist to comprehensively measure GC toxicity, providing an accurate and easy-to-use tool for clinical and research purposes.
SEMINARS IN ARTHRITIS AND RHEUMATISM
(2022)
Article
Medicine, General & Internal
Pui Y. Lee, Brad A. Davidson, Roshini S. Abraham, Blanche Alter, Juan I. Arostegui, Katherine Bell, Alexandre Belot, Jenna R. E. Bergerson, Timothy J. Bernard, Paul A. Brogan, Yackov Berkun, Natalie T. Deuitch, Dimana Dimitrova, Sophie A. Georgin-Lavialle, Marco Gattorno, Bodo Grimbacher, Hasan Hashem, Michael S. Hershfield, Rebecca N. Ichord, Kazushi Izawa, Jennifer A. Kanakry, Raju P. Khubchandani, Femke C. C. Klouwer, Evan A. Luton, Ada W. Man, Isabelle Meyts, Joris M. Van Montfrans, Seza Ozen, Janna Saarela, Gustavo C. Santo, Aman Sharma, Ariane Soldatos, Rachel Sparks, Troy R. Torgerson, Ignacio Leandro Uriarte, Taryn A. B. Youngstein, Qing Zhou, Ivona Aksentijevich, Daniel L. Kastner, Eugene P. Chambers, Amanda K. Ombrello
Summary: Deficiency of adenosine deaminase 2 (DADA2) is a recessively inherited disease characterized by systemic vasculitis, early-onset stroke, bone marrow failure, and/or immunodeficiency affecting both children and adults. This study provides consensus statements for the evaluation and management of DADA2, a potentially fatal disease that requires early diagnosis and treatment.
Article
Immunology
Samantha Cooray, Fiona Price-Kuehne, Ying Hong, Ebun Omoyinmi, Alice Burleigh, Kimberly C. Gilmour, Bilal Ahmad, Sangdun Choi, Mohammad W. Bahar, Paul Torpiano, Andrey Gagunashvili, Barbara Jensen, Evangelos Bellos, Vanessa Sancho-Shimizu, Jethro A. Herberg, Kshitij Mankad, Atul Kumar, Marios Kaliakatsos, Austen J. J. Worth, Despina Eleftheriou, Elizabeth Whittaker, Paul A. Brogan
Summary: We describe a novel, severe autoinflammatory syndrome characterized by neuroinflammation, systemic autoinflammation, splenomegaly, and anemia (NASA) caused by bi-allelic mutations in IRAK4. This study describes 5 affected patients from 2 unrelated families with compound heterozygous mutations in IRAK4 resulting in severe systemic autoinflammation, massive splenomegaly and severe transfusion dependent anemia and, in 3/5 cases, severe neuroinflammation and seizures. Immunological analysis demonstrated elevated levels of pro-inflammatory cytokines in affected patients.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Pediatrics
C. M. Foley, D. Mckenna, K. Gallagher, K. Mclellan, H. Alkhdher, S. Lacassagne, E. Moraitis, C. Papadopoulou, C. Pilkington, M. Al Obaidi, D. Eleftheriou, P. Brogan
Summary: Systemic juvenile idiopathic arthritis (sJIA) is a complex inflammatory disorder involving innate and adaptive immunity. Research shows that early use of IL-1 or IL-6 blockade as first-line treatment is effective. This study reviewed the medical records of sJIA patients over a 16-year period and examined clinical presentation, treatment interventions, and remission rates. The results indicate that patients who received early biologic treatment achieved higher rates of clinical remission.
FRONTIERS IN PEDIATRICS
(2023)
Article
Rheumatology
Fabrizio De Benedetti, Alexei A. Grom, Paul A. Brogan, Claudia Bracaglia, Manuela Pardeo, Giulia Marucci, Despina Eleftheriou, Charalampia Papadopoulou, Grant S. Schulert, Pierre Quartier, Jordi Anton, Christian Laveille, Rikke Frederiksen, Veronica Asnaghi, Maria Ballabio, Philippe Jacqmin, Cristina de Min
Summary: The objective of this study was to confirm the adequacy of an emapalumab dosing regimen in relation to interferon-γ (IFN-γ) activity and assess its efficacy and safety in treating MAS secondary to sJIA or AOSD. The results showed that emapalumab was efficacious in inducing remission of MAS and viral infections were observed.
ANNALS OF THE RHEUMATIC DISEASES
(2023)
Meeting Abstract
Rheumatology
Adriana Almeida de Jesus, Guibin Chen, Dan Yang, Tomas Brdicka, Natasha Ruth, David Bennin, Dita Cebecauerova, Hana Malcova, Helen Freeman, Neil Martin, Karel Svojgr, Murray Passo, Farzana Bhuyan, Sara Alehashemi, Andre Rastegar, Kat Uss, Lela Kardova, Iris Duric, Ebun Omoyinmi, Petra Peldova, Chyi-Chia Richard Lee, David Kleiner, Colleen Hadigan, Stephen Hewitt, Stefania Pittaluga, Carmelo Carmona-Rivera, Katherine R. Calvo, Nirali Shah, Miroslava Balascakova, Danielle Fink, Radana Kotalova, Zuzana Parackova, Lucie Peterkova, Daniela Kuzilkova, Vit Campr, Lucie Sramkova, Stephen Brooks, Eric Meffre, Rebecca Harper, Hyesun Kuehn, Mariana Kaplan, Paul Brogan, Sergio Rosenzweig, Zuoming Deng, Anna Huttenlocher, Susan Moir, Douglas Kuhns, Manfred Boehm, Karolina Skvarova Kramarzova, Raphaela Goldbach-Mansky
ARTHRITIS & RHEUMATOLOGY
(2022)