Infection Complications in Pediatric Patients With Inborn Errors of Immunity After Umbilical Cord Blood

Zhongling Wei1,2, Ping Wang1, Wenjin Jiang1

  • 1Department of Hematology/Oncology, National Children's Medical Center, Children's Hospital of Fudan University, Shanghai, China.

Insights

Infections are common in children with inborn errors of immunity (IEIs) after umbilical cord blood transplantation (UCBT). Early-stage infections and bloodstream infections (BSIs) significantly worsen patient outcomes, highlighting the need for targeted surveillance.

Area of Science:

  • Pediatric Hematology/Oncology
  • Immunology
  • Infectious Diseases

Background:

  • Infection is a frequent complication after umbilical cord blood transplantation (UCBT).
  • Data on infection profiles in pediatric patients with inborn errors of immunity (IEIs) in China are limited.
  • This study characterizes infection patterns and their prognostic impact in IEIs children undergoing UCBT.

Purpose of the Study:

  • To investigate infection prevalence and risk factors in pediatric IEIs patients post-UCBT.
  • To determine the prognostic implications of different infection types and stages on patient survival.
  • To inform risk-stratified surveillance and preemptive strategies for high-risk subgroups.

Main Methods:

  • Retrospective analysis of 165 pediatric IEIs patients undergoing UCBT (January 2015 - June 2020).
  • Categorization of post-transplantation period into early (≤30 days) and middle (31-100 days) stages.
  • Statistical analysis to identify predictors of infection and their association with outcomes.

Main Results:

  • 69.7% of patients experienced infections within 100 days post-UCBT.
  • Engraftment failure, low pre-transplant IgM, and specific graft-versus-host disease (GVHD) prophylaxis regimens were linked to increased infection risk.
  • Early-stage infections and bloodstream infections (BSIs) were associated with significantly poorer survival.
  • Bacterial infections, particularly carbapenem-resistant strains, and BSIs correlated with adverse prognosis.

Conclusions:

  • Infections are highly prevalent in pediatric IEIs patients following UCBT.
  • Early-stage infections and BSIs are critical indicators of poor prognosis.
  • Findings support the implementation of risk-stratified surveillance and preemptive interventions for vulnerable patient groups.
Abstract

Related Concept Videos

Inborn Errors of Metabolism01:20

Inborn Errors of Metabolism

Phenylketonuria (PKU) is a protein metabolism disorder characterized by high blood levels of the amino acid phenylalanine. This results from a mutation in the gene responsible for phenylalanine hydroxylase, an enzyme that converts phenylalanine into tyrosine. When this enzyme is deficient, phenylalanine builds up in the blood, leading to symptoms such as vomiting, rashes, seizures, growth deficiency, and severe mental retardation. An early diagnosis and a diet restricting phenylalanine intake...
915
Patient-centered Care01:13

Patient-centered Care

Patient-centered care involves delivering care beyond inpatient hospitalization. Reflective practice can enhance a patient-centered approach. Reflective practice is a process of reasoning that considers all aspects of the present situation, including practicalities, learning from personal practice, and consideration of patient needs. Patients appreciate care decisions made while considering their input. Involving the patient in their care provides the patient with a sense of contribution rather...
3.1K
Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
279
Pharmacokinetics in Pediatric Patients: Drug Distribution01:17

Pharmacokinetics in Pediatric Patients: Drug Distribution

Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
334
Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
251
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption01:23

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
316