Leiden University Scholarly Publications

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Vos, T.W. de (2023)

Fetal and neonatal alloimmune thrombocytopenia: the proof of the pudding is in the eating

Doctoral Thesis
Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a rare but potentially life threatening disease that can lead to intracranial haemorrhage (ICH) in babies during fetal development and the neonatal period. ICH is associated with perinatal mortality and can lead to long-term neurodevelopmental impairment. If pregnancies at risk for FNAIT are identified upon antenatal screening, timely intervention could prevent the occurrence of fetal ICH. Implementation of population-based screening to prevent FNAIT is hampered by the lack of knowledge on the natural history, whom to treat and costs of case finding. We performed a large nationwide screening study and provide new evidence on the incidence of FNAIT. In addition, we confirm the value of risk factors for immunisation and severe disease. We describe current postnatal treatment strategies and the long-term outcome of cases that were affected by FNAIT. Based on these studies, we conducted a cost-effectiveness analysis comparing...Show moreFetal and neonatal alloimmune thrombocytopenia (FNAIT) is a rare but potentially life threatening disease that can lead to intracranial haemorrhage (ICH) in babies during fetal development and the neonatal period. ICH is associated with perinatal mortality and can lead to long-term neurodevelopmental impairment. If pregnancies at risk for FNAIT are identified upon antenatal screening, timely intervention could prevent the occurrence of fetal ICH. Implementation of population-based screening to prevent FNAIT is hampered by the lack of knowledge on the natural history, whom to treat and costs of case finding. We performed a large nationwide screening study and provide new evidence on the incidence of FNAIT. In addition, we confirm the value of risk factors for immunisation and severe disease. We describe current postnatal treatment strategies and the long-term outcome of cases that were affected by FNAIT. Based on these studies, we conducted a cost-effectiveness analysis comparing the situation with antenatal screening to the current situation without screening. In the general discussion, we evaluate the knowledge gained in this thesis and in the available literature guided by the principles from Wilson and Junger. We conclude that knowledge is available to all principles and nationwide screening for FNAIT during pregnancy seems warranted.
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Human platelet antigen
Alloimmunisation during pregnancy
Fetal medicine
Antenatal screening
Intravenous immune globulin
Platelet transfusion
Neurodevelopmental outcome
All authors
Vos, T.W. de
Supervisor
Haas, M. de; Lopriore, E.
Co-supervisor
Klink, J.M.M. van
Committee
Lankester, A.C.; Tiller, H.; Bein, G.; Bekker, M.N.
Qualification
Doctor (dr.)
Awarding Institution
Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
Date
2023-04-13
ISBN (print)
9789464196719

Funding

Sponsorship
The research described in this thesis was supported by Process and Product Development Diagnostic Services Sanquin (SQI/00034), Fonds Gezond Geboren (E50-03-LUMC) and Landsteiner Foundation for Blood Transfusion Research (1440). Financial support for printing this thesis was kindly provided by, Naitbabies.org, Sanquin Research, Universitaire Bibliotheken Leiden, Immucor and Chiesi Pharmaceuticals B.V.