Background: Children with inherited metabolic disorders are a potential high-risk group for vaccine-preventable diseases, yet information regarding immunization rates and vaccine safety within this population is limited.
Methods: Using Northern California Kaiser Permanente's electronic medical record, we identified children with inborn errors of metabolism from 1990 to 2007. We assessed immunization rates among infants with inborn errors of metabolism born at Northern California Kaiser Permanente matched to healthy infants (1 to 20), comparing both vaccines received by 2 years of age and age at vaccination. We assessed postvaccination adverse events among children up to 18 years old with inborn errors of metabolism, separately comparing emergency-department visits and hospitalizations during postvaccine days 0 to 30 (primary) and days 0 to 14 (secondary).
Results: Comparing infants with inborn errors of metabolism (n = 77) versus matched control subjects (n = 1540), similar proportions were up to date for vaccines at 2 years of age, and there was no evidence of delay in receipt of recommended vaccines during the first year. Vaccination of children with inborn errors of metabolism (n = 271) was not associated with any significant increase in emergency-department visits or hospitalizations during the 30 days after vaccination. Secondary analyses suggested that there may be increased rates of hospitalizations 2 weeks after vaccination for the sickest 1- to 4-year-old children.
Conclusions: Children with inborn errors of metabolism at Northern California Kaiser Permanente received vaccines on the same immunization schedule as healthy infants. Immunization was not associated with increased risk for serious adverse events during the month after vaccination, providing overall reassurance that routine vaccination of children with inborn errors of metabolism does not result in adverse effects.
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http://dx.doi.org/10.1542/peds.2010-3706 | DOI Listing |
J Immunol Methods
January 2025
Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN, United States of America. Electronic address:
Complement functional assays are essential first-tier tests for a gamut of disorders spanning from inborn errors of the immune system which lead to recurrent severe infections, to angioedema attacks, presentation of autoimmune disease, thrombotic microangiopathies and rare kidney disorders. These assays evaluate the activity of the three complement pathways and specific complement components, which helps in differential diagnosis and monitoring disease progression. The rising use of complement inhibitors for treating complement-mediated thrombotic microangiopathies has heightened the demand for personalized treatment plans and laboratory assessment of complement blockage.
View Article and Find Full Text PDFJ Infect Chemother
January 2025
Division of Infectious Diseases, Department of Pediatrics, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan; Division of Immunology, Department of Pediatrics, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan.
Cytomegalovirus (CMV) infection remains one of the most common and challenging post-transplant infections. Children with inborn errors of immunity (IEI) and T-cell dysfunction are at high risk for CMV infection, which can be complicated by refractory and/or resistant cases. This case describes a Nepalese girl with MHC class II deficiency, who presented at 3 months of age with CMV and Pneumocystis jirovecii pneumonia.
View Article and Find Full Text PDFNutrients
January 2025
Unidad de Gestión Clínica de Endocrinología y Nutrición, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC/Universidad de Sevilla, Avda. Manuel Siurot s/n, 41013 Seville, Spain.
Background: This study addresses hypoglycemia in adults with inherited metabolic disorders (IMDs), highlighting the importance of intermittently scanned continuous glucose monitoring (isCGM). Despite the elevated risk of hypoglycemia in an important group of these diseases, the use of isCGM remains uncommon and there is limited evidence supporting its effectiveness.
Methods: A longitudinal quasi-experimental study was performed in 18 adults with IMDs, evaluating the use of isCGM for 2 months.
Life (Basel)
December 2024
Department of Translational Medical Sciences, Pediatric Section, Federico II University of Naples, 80131 Naples, Italy.
MyD88 deficiency is a rare inborn error of immunity (IEI) characterized by susceptibility to pyogenic infections without overt signs of inflammation. Half of the reported patients belong to Roma descent, an itinerant ethnic group living mostly in Europe, with an increased risk of childhood mortality due to limited access to healthcare services. We describe three unrelated patients from the Campania region in Italy with MyD88 deficiency, all belonging to Roma descent and displaying severe or recurrent infections in early infancy.
View Article and Find Full Text PDFMedicina (Kaunas)
January 2025
Division of Allergy/Immunology, Department of Pediatrics, Jackson Memorial Holtz Children's Hospital, University of Miami Miller School of Medicine, Miami, FL 33136, USA.
Inborn errors of immunity (IEIs), also known as primary immunodeficiencies, are a group of genetic disorders affecting the development and function of the immune system. While IEIs traditionally present with recurrent infections, an increasing number of cases manifest with early-onset severe atopy, including atopic dermatitis, food allergies, asthma, and allergic rhinitis-features that are often overlooked. This can lead to delayed diagnosis and treatment, which is crucial for IEI patients due to the risk of severe infections.
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