To evaluate the long-term neurodevelopmental outcome in two groups of children with FNAIT. First, children with unanticipated disease *index cases* that were born without maternal IVIg administration during pregnancy. Second, children with…
ID
Source
Brief title
Condition
- Platelet disorders
- Foetal complications
- Developmental disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary outcome is cognitive development i.e., cognitive test score or
Intelligence Quotient (IQ).
Secondary outcome
Secondary outcome is neurodevelopmental impairment (NDI); a composite outcome
including cerebral palsy Gross Motor Functioning Classification System (GMFCS)
* II, cognitive and/or motor test score of less than 70 (-2 SD), hearing loss
requiring amplification or visual impairment (legally certifiable as blind or
partially sighted). Other secondary outcome parameters are HRQoL and behavioral
functioning and the academic performance. The proportion of children with an
abnormal course or incidence of infections, asthma, eczema and allergies will
be assessed.
Background summary
Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a disease caused by
allo-immunisation during pregnancy. If left untreated, FNAIT can lead to severe
fetal intracranial haemorrhage. This complication can be prevented by weekly
administration of intravenous immunoglobulin (IVIg) to the mother during
pregnancy. Although treatment with IVIg is nowadays considered as the golden
standard for the management of FNAIT, the exact mechanism of action of IVIg is
still under debate. Most importantly, the long-term effects and safety of IVIg
treatment in FNAIT have not yet been evaluated. The use of IVIg during
pregnancy for FNAIT is therefore still *off-label*. The medication leaflet of
IVIg continues to state that there is insufficient knowledge on the use of this
product during pregnancy, despite all the years of experience and
implementation in virtually all international guidelines.
Knowledge on long-term development of FNAIT survivors with or without IVIg
treatment is very limited but an important subject in the counselling of
parents of newly diagnosed cases. A greater understanding of the long-term
outcome and the effects of IVIg on child development is necessary.
Study objective
To evaluate the long-term neurodevelopmental outcome in two groups of children
with FNAIT. First, children with unanticipated disease *index cases* that were
born without maternal IVIg administration during pregnancy.
Second, children with anticipated disease; their mothers were treated with IVIg
during pregnancy.
Study design
Observational cohort study.
Study burden and risks
The burden of neurodevelopmental assessment tests is minimal (e.g. 120 minutes
at 2 to 6 years and 150 minutes at 7 to 17 years follow-up). Neurodevelopmental
assessment is generally experienced as enjoyable for children. Parents will
complete a HRQoL- and a behavioral questionnaire for their children. Academic
performance will be evaluated by scores that were obtained at school.
Participation does not result in any direct benefit.
Albinusdreef 2
Leiden 2333 ZA
NL
Albinusdreef 2
Leiden 2333 ZA
NL
Listed location countries
Age
Inclusion criteria
- Children diagnosed with FNAIT during pregnancy or postnatal, at moment of
inclusion 2 to 16 years of age.
- Children living in the Netherlands.
- Parents or guardian aged * 18 years old, with parental authority.
- Written informed consent form both parents with, form being approved by
Ethic Committee.
Exclusion criteria
- Children born with congenital and/or chromosomal abnormalities.
- Children that passed away before inclusion.
Design
Recruitment
metc-ldd@lumc.nl
metc-ldd@lumc.nl
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In other registers
| Register | ID |
|---|---|
| CCMO | NL70410.058.19 |