Neurodevelopmental Outcome After Fetal Neonatal AlloImmune Thrombocytopenia

NCT04529382 | Unknown

• 1 other identifier: P19.069
• Study Type: observational
• Target: 78
• Locations: 1 country
• Sites: 1

Brief 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. 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. To evaluate the long-term neurodevelopmental outcome in two groups of children with FNAIT will be asked to participate in our study in an outpatient clinic setting.

Conditions

Fetal and Neonatal Alloimmune Thrombocytopenia; Long Term Adverse Effects; Neurodevelopmental Abnormality; Intravenous Immunoglobulin Adverse Reaction

Outcome Measures

Primary Outcomes (1)

• Cognitive test score

  IQ test score calculated from a standardized cognitive test.

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

Secondary Outcomes (13)

• Neurodevelopmental injury (NDI)

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

• Health Related Quality of Life

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

• Cerebral Palsy

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

• Bilateral blindness

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

• Bilateral deafness

  From birth until study enrollment. Average age of the participants is expected to be 8 years old.

Other Outcomes (24)

• Maternal characteristics

  Measured at delivery of the child that is assessed. Calculated in years.

• Obstetric history

  Measured at delivery of the child that is assessed.

• Gravidity/Parity

  Measured at delivery of the child that is assessed.

Study Arms (2)

Cohort 1: Unanticipated FNAIT cases

All children born between 2002 and 2017 and diagnosed with FNAIT are eligible for this study. Children in cohort 1 will be FNAIT cases that were not antenatally treated with by maternal IVIg administration (or other forms of fetal therapy).

Diagnostic Test: Cognitive testing (Bayley III, WPPSI III and WISC V)

Cohort 2: Anticipated FNAIT cases

All children born between 2002 and 2017 and diagnosed with FNAIT are eligible for this study. Children in cohort 2 will be FNAIT cases which were anticipated antenatally by maternal IVIg administration according to our local protocol.

Diagnostic Test: Cognitive testing (Bayley III, WPPSI III and WISC V)

Interventions

Cognitive testing (Bayley III, WPPSI III and WISC V) DIAGNOSTIC_TEST

Parents and children will be invited to come for an outpatient clinic visit where cognitive testing and neurologic examination will be performed. In addition to this parents will be asked to fill in questionnaires, provide latest school results and medical files will be requested from treating physicians.

Also known as: Pediatric Quality of Life Inventory, Child Behavior Checklist, Questionnaire based on 'Richtlijn diagnostiek naar onderliggende aandoeningen bij kinderen met recidiverende luchtweginfecties', Questionnaire composited in collaboration with allergist (Dr. H. de Groot)., Neurologic Examination

Cohort 1: Unanticipated FNAIT cases; Cohort 2: Anticipated FNAIT cases

Eligibility Criteria

• Age: 18 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

All children born between 2002 and 2017 and diagnosed with FNAIT are eligible for this study. They will be invited for an assessment at our outpatient clinic. FNAIT cases that were not antenatally treated with IVIg will be eligible for the study (cohort 1), as well as FNAIT cases which were anticipated antenatally by materal IVIg administration (cohort 2).

You may qualify if:

• 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.

You may not qualify if:

• Children born with congenital and/or chromosomal abnormalities.

Sponsors & Collaborators

• Leiden University Medical Center: lead
• Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research: collaborator

Study Sites (1)

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

Location

Related Publications (29)

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MeSH Terms

Conditions

Thrombocytopenia, Neonatal Alloimmune; Long Term Adverse Effects

Interventions

Neurologic Examination

Condition Hierarchy (Ancestors)

Thrombocytopenia; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Diagnostic Techniques, Neurological; Diagnostic Techniques and Procedures; Diagnosis; Physical Examination

Study Officials

• Enrico Lopriore, Prof. MD PhD

  Department of Neonatology, Leiden University Medical Center

  PRINCIPAL INVESTIGATOR

• Masja de Haas, Prof. MD PhD

  Stichting Sanquin Bloedvoorziening

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. E. Lopriore

Study Record Dates

• First Submitted: July 15, 2020
• First Posted: August 27, 2020
• Study Start: December 17, 2019
• Primary Completion: December 17, 2020
• Study Completion: December 17, 2021
• Last Updated: August 27, 2020

Record last verified: 2020-08

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)