Introduction: A few data on the prevalence of renal involvement in cystic fibrosis and its spectrum in childhood is available. In the present study, we conducted a prospective study on children who had cystic fibrosis and evaluated their renal involvement. In fact, the aim of the study was to provide data on the clinical consequences of proper identification of kidney disease in a group of children with cystic fibrosis.
Methods: This prospective study was conducted on 55 consecutive patients with previous diagnosis of cystic fibrosis during a threeyear period and at least 3 months to over 5 years or more follow-up. The inclusion criteria was the diagnosis of cystic fibrosis which was made by clinical presentation of cystic fibrosis and laboratory results. Initially, patients' medical records were reviewed and relevant data were collected. A 24-hour urine collection (or a random urine sampling in very young infants) was used to assess crystalluria and renal function was evaluated by blood sampling.
Results: Totally, 55 patients with cystic fibrosis were admitted in two hospitals with the mean age of 8.22 ± 5.66 years. GFR totally reduced in 34.5%. The overall prevalence of hypercalciuria was estimated to be 60%, while hyperoxaluria, hypocitraturia, and hyperuricosuria in 41.8%, 24.5%, and 47.3%; respectively.
Conclusion: Crystalluria is a common consequence of cystic fibrosis in childhood. The prevailing crystalluric finding includes hypercalciuria followed by hyperuricosuria, and hyperoxaluria. During disease GFR may be decreased due to several reasons such as nephrotoxic drugs usage.
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Lancet
January 2025
Centre for Inflammatory Disease, Department of Immunology and Inflammation, Imperial College London, London, UK; Imperial College Renal and Transplant Centre, Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK. Electronic address:
Background: In the UK, booster COVID-19 vaccinations have been recommended biannually to people considered immune vulnerable. We investigated, at a population level, whether the absence of detectable anti-SARS-CoV-2 spike protein IgG antibody (anti-S Ab) following three or more vaccinations in immunosuppressed individuals was associated with greater risks of infection and severity of infection.
Methods: In this prospective cohort study using UK national disease registers, we recruited participants with solid organ transplants (SOTs), rare autoimmune rheumatic diseases (RAIRDs), and lymphoid malignancies.
J Biol Chem
January 2025
Department of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, CA 94158, USA; Department of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA. Electronic address:
Carboxyl-terminus of Hsp70-Interacting Protein (CHIP) is an E3 ubiquitin ligase that marks misfolded substrates for degradation. Hyper-activation of CHIP has been implicated in multiple diseases, including cystic fibrosis and cancer, suggesting that it may be a potential drug target. However, there are few tools available for exploring this possibility.
View Article and Find Full Text PDFInt J Pharm
January 2025
CIDETEC, Basque Research and Technology Alliance (BRTA), Parque Científico y Tecnológico de Gipuzkoa, Donostia-San Sebastián, Spain; Kusudama Therapeutics SA, Parque Científico y Tecnológico de Gipuzkoa, Donostia-San Sebastián, Spain; Biogipuzkoa Health Research Institute, Group of Innovation, 20014 San Sebastian, Spain.
Cystic fibrosis (CF) is characterized by abnormal mucus hydration due to a defective CF Transmembrane Regulator (CFTR) protein, leading to the production of difficult-to-clear mucus. This causes airflow obstruction, recurrent infections, and respiratory complications. Chronic lung infections are the leading cause of death for CF patients and inhaled tobramycin is the first-in-line antibiotic treatment against these infections, mainly caused by Pseudomonas aeruginosa in adult patients.
View Article and Find Full Text PDFPharmaceutics
January 2025
School of Pharmacy, Queen's University Belfast, Belfast BT9 7BL, UK.
This paper presents a comprehensive review of the current literature, clinical trials, and products approved for the delivery of antibiotics to the lungs. While there are many literature reports describing potential delivery systems, few of these have translated into marketed products. Key challenges remaining are the high doses required and, for powder formulations, the ability of the inhaler and powder combination to deliver the dose to the correct portion of the respiratory tract for maximum effect.
View Article and Find Full Text PDFInt J Mol Sci
January 2025
Department of Pediatrics, National Jewish Health, Denver, CO 80206, USA.
The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel that is dysfunctional in individuals with cystic fibrosis (CF). The permeability of CFTR can be experimentally manipulated though different mechanisms, including activation via inducing the phosphorylation of residues in the regulatory domain as well as altering the gating/open probability of the channel. Phosphorylation/activation of the channel is achieved by exposure to compounds that increase intracellular cAMP, with forskolin and IBMX commonly used for this purpose.
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