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An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation.
Front Immunol
March 2024
Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.
Introduction: High sustained anti-rhGAA antibody titers (HSAT; ≥12,800) are directly linked to reduced efficacy of enzyme replacement therapy (ERT) and subsequent clinical deterioration in infantile-onset Pompe disease (IOPD). We have previously demonstrated the safety and effectiveness of a bortezomib-based immune-tolerance induction (ITI) regimen (bortezomib, rituximab, methotrexate, and IVIG) in eliminating HSAT.
Methods: Here, we describe two IOPD cases (patients 6 and 8) who developed HSAT at 8 and 10 weeks on ERT despite transient low-dose methotrexate ITI administration in the ERT-naïve setting and were treated with a bortezomib-based ITI regimen, and we compare their courses to a series of six historical patients (patients 1-5, and 7) with a similar presentation who exemplify our evolving approach to treatment.
Differential effects of growth restriction and immaturity on predicted psychomotor development at 4 years of age in preterm infants.
AJOG Glob Rep
February 2024
Department of Paediatric Neurology, University of Giessen, Germany (Dr Neuhäuser).
Background: Fetal growth restriction and immaturity are associated with poor neurocognitive development and child psychopathology affecting educational success at school and beyond. However, the differential effects of either obstetrical risk factor on predicted psychomotor development have not yet been deciphered.
Objective: This study aimed to separately study the impact of growth restriction and that of immaturity on predicted psychomotor development at the preschool age of 4.
A Structured Training for Trans Anal Irrigation in Pediatric Patients Improves Outcomes and Reduce Failures: Results of an Interventional Multicenter Prospective Study.
J Pediatr Surg
September 2024
Pediatric Surgery Unit, ASST Papa Giovanni XXIII, Bergamo, Italy.
Background: Continence issues due to organic causes including previous colorectal surgery or neurological issues might benefit from Transanal irrigation (TAI) that proved to be highly effective but with a number of limitations including a relatively high discontinuation rates. Our study was aimed at evaluating the efficacy of an advanced protocol tailored to each patient to prevent dropout and increase satisfaction, independence, and quality of life.
Materials And Methods: This was a prospective, interventional, multicenter, nonrandomized study involving children aged 4-18 years with bowel dysfunction unresponsive to conventional treatments who required TAI.
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