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Children Newly Diagnosed with Fetal and Neonatal Alloimmune Thrombocytopenia: Neurodevelopmental Outcome at School
Thijs W de Vos1, Maud van Zagten2, Masja de Haas3
1Division of Neonatology, Department of Pediatrics, Leiden University Medical Center, Willem-Alexander Children's Hospital, The Netherlands; Center of Clinical Transfusion Research, Sanquin Research, Amsterdam; Department of Experimental Immunohematology, Sanquin Research, Amsterdam.
Insights
Children with fetal and neonatal alloimmune thrombocytopenia (FNAIT) face increased risks of long-term neurodevelopmental problems. These risks persist even in cases without intracranial hemorrhage (ICH), highlighting the need for ongoing monitoring.
Area of Science:
- Pediatrics
- Neurology
- Immunology
Background:
- Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a condition that can lead to significant health issues in newborns.
- Neurodevelopmental outcomes in children with FNAIT have not been extensively studied, particularly at school age.
- Understanding long-term neurodevelopmental effects is crucial for appropriate patient management and support.
Purpose of the Study:
- To evaluate the neurodevelopmental outcome at school age in children diagnosed with FNAIT.
- To identify risk factors associated with neurodevelopmental impairment (NDI) in this population.
- To assess the prevalence of severe and mild-to-moderate NDI.
Main Methods:
- An observational cohort study included children diagnosed with FNAIT between 2002 and 2014.
- Children underwent cognitive and neurological testing, behavioral questionnaires, and school performance assessments.
- Neurodevelopmental impairment (NDI) was defined using IQ scores, cerebral palsy classification, and visual/hearing impairment, with severe NDI as the primary outcome.
Main Results:
- Out of 44 children studied, 7% experienced severe NDI and 25% had mild-to-moderate NDI.
- Intracranial hemorrhage (ICH) was present in 14% of children with available neuroimaging.
- An adverse outcome (perinatal death or NDI) was observed in 39% of cases.
Conclusions:
- Children diagnosed with FNAIT are at an elevated risk for long-term neurodevelopmental problems.
- This increased risk is present even in cases where intracranial hemorrhage (ICH) is not detected.
- Early identification and intervention strategies are essential for improving outcomes in children with FNAIT.
Objective:
To evaluate the neurodevelopmental outcome at school age in children newly diagnosed with fetal and neonatal alloimmune thrombocytopenia (FNAIT).
Study Design:
This observational cohort study included children diagnosed with FNAIT between 2002 and 2014. Children were invited for cognitive and neurological testing. Behavioral questionnaires and school performance results were obtained. A composite outcome of neurodevelopmental impairment (NDI) was used, defined, and subdivided into mild-to-moderate and severe NDI. Primary outcome was severe NDI, defined as IQ <70, cerebral palsy with Gross Motor Functioning Classification System level ≥ III, or severe visual/hearing impairment. Mild-to-moderate NDI was defined as IQ 70-85, minor neurological dysfunction or cerebral palsy with Gross Motor Functioning Classification System level ≤ II, or mild visual/hearing impairment.
Results:
In total, 44 children were included at a median age of 12 years (range: 6-17 years). Neuroimaging at diagnosis was available in 82% (36/44) of children. High-grade intracranial hemorrhage (ICH) was detected in 14% (5/36). Severe NDI was detected in 7% (3/44); two children had high-grade ICH, and one had low-grade ICH and perinatal asphyxia. Mild-to-moderate NDI was detected in 25% (11/44); one child had high-grade ICH, and eight children were without ICH, yet for two children, neuroimaging was not performed. Adverse outcome (perinatal death or NDI) was 39% (19/49). Four children (9%) attended special needs education, three of whom had severe NDI and one had mild-to-moderate NDI. Total behavioral problems within the clinical range were reported in 12%, which is comparable with 10% in the general Dutch population.
Conclusion:
Children who are newly diagnosed with FNAIT are at increased risk for long-term neurodevelopmental problems, even those without ICH.
Trial Registration:
The study was registered at ClinicalTrials.gov (Identifier: NCT04529382).
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