Treatment of Erdheim-Chester disease (ECD), a rare non-Langerhans histiocytosis, relies on interferon-α, chemotherapeutic agents such as purine analogs, cytokine-blocking agents and BRAF inhibitors. Since interleukin (IL)-6 levels are elevated in serum and lesions of ECD patients, we evaluated the therapeutic efficacy and safety of IL-6 blockade with tocilizumab. We conducted an open-label, single-arm, phase II, prospective study of tocilizumab in three patients with multisystem ECD and poor tolerance/contraindications to IFN-α. Modifications of symptoms attributed to ECD represented the criteria for evaluation of clinical response. Changes at positron emission tomography scan, computed tomography scan, and magnetic resonance imaging at month 6 represented the main criteria for the evaluation of radiological response. Sustained complete clinical response and partial radiological improvement were observed in two patients, paralleled by modulation of systemic pro-inflammatory mediators. In spite of disease stabilization or improvement at extra-neurological sites, a third patient experienced a radiologic and clinical progression of central nervous system involvement, mirrored by a dramatic increase of circulating IL-6 and related cytokines. These findings indicate that IL-6 inhibition can be effective in ECD, but caution is advisable in patients with neurologic involvement. IL-6 emerges as a central mediator in ECD pathogenesis.
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http://dx.doi.org/10.1080/2162402X.2017.1318237 | DOI Listing |
Breathe (Sheff)
January 2025
Division of Respiratory, Department of Medicine, Sarawak General Hospital, Ministry of Health Malaysia, Kuching, Sarawak, Malaysia.
https://bit.ly/4eJ0PHT.
View Article and Find Full Text PDFFront Cell Infect Microbiol
January 2025
Department of Genetics and Bioinformatics, Dasman Diabetes Institute, Kuwait City, Kuwait.
Background: Multisystem inflammatory syndrome in children (MIS-C) is a severe complication arising from SARS-CoV-2 infection, with indications that rare inborn errors of immunity may play a role in its pathogenesis. Recent studies suggest that genetic predispositions, particularly monogenic forms, could significantly influence the immune responses to SARS-CoV-2 in MIS-C.
Methods: We analysed 24 children under 12 years old, all of whom met the criteria provided by the World Health Organization, 2020 for MIS-C diagnosis, from the Paediatric COVID-19 Registry in Kuwait (PCR-Q8).
Rev Paul Pediatr
January 2025
Afyonkarahisar Health Sciences University, Faculty of Medicine, Department of Child Health and Diseases, Afyonkarahisar, Turkey.
Objective: Brucellosis is a multisystem infectious disease and may cause an increase in acute phase reactants. This study aimed to examine the platelet mass index (PMI), the neutrophil-lymphocyte ratio (NLR), and systemic immune-inflammation index (SII) in children with brucellosis and to determine their roles in focal involvement.
Methods: This retrospective observational study included 69 patients with brucellosis and a control group of 69 healthy children.
Spine Deform
January 2025
Great North Children's Hospital, Newcastle Upon Tyne, UK.
Purpose: To determine the prevalence of scoliosis in patients who have undergone surgical repair of CDH and attempt to assess the aetiology of scoliosis in affected cases.
Methods: A prospectively collected database of patients with CDH treated in a single centre between 1997 and 2023 was reviewed. Cases with adequate records who continued to reside locally having survived beyond age 2 and > 2 years following CDH repair were included.
Child Obes
January 2025
Department of Pediactrics, The Children's Obesity Clinic, Accredited European Centre for Obesity Management, Copenhagen University Hospital Holbæk, Holbæk, Denmark.
Steatotic liver disease (SLD) represents a multisystem disease and is a common complication of childhood obesity. We studied fat content at the abdominal level (liver, subcutaneous, and visceral) and the response to childhood obesity management. In this retrospective longitudinal study, 8-18-year-olds with a body mass index (BMI) z-score above 1.
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