PRedicting OutcomeS in Preterm nEonates With thromboCyTopenia

Summary

Preterm neonates often receive platelet transfusions when their platelet count is low to prevent bleeding. However, it is currently unclear which infants benefit from such transfusions. A recent randomized controlled trial (PlaNeT-2/MATISSE trial) showed that the higher platelet count threshold for transfusion was associated with a higher risk of major bleeding or death. Current transfusion protocols are based only on platelet count thresholds. However, neonates with similar platelet counts may have different bleeding risks due to varying clinical conditions. There is an important unmet medical need to identify which neonates with low platelet counts (i.e., severe thrombocytopenia) will benefit from a transfusion. Ideally, clinicians would be able to repeatedly predict a neonate's risk of major bleeding or death with and without giving a platelet transfusion, taking into account the neonate's clinical condition at that particular time. Obtaining personalized risk estimates under specific treatment strategies, with updated predictions at each new treatment decision moment, is called 'sequential prediction under interventions'. The investigators set up an international multicenter observational cohort study to develop a model to predict major bleeding or death with and without platelet transfusion at any time point during the first week after the onset of severe thrombocytopenia. This model is designed to support platelet transfusion decisions in the NICU and may help clinicians balance the benefits and harms of platelet transfusion based on updated characteristics of the neonate at the time of prediction.

Conditions

• Neonatal Thrombocytopenia
• Intraventricular Hemorrhage
• Platelet Transfusion

Interventions

DRUG

Platelets

Observational data: all platelet transfusions recorded in routine care medical file data.

Sponsors & Collaborators

• Sanquin Research & Blood Bank Divisions

  collaborator OTHER

• Amsterdam University Medical Center

  collaborator OTHER

• Leiden University Medical Center

  lead OTHER

Principal Investigators

• Hilde van der Staaij, MD · Leiden University Medical Center and Sanquin Blood Supply Foundation

• Johanna G van der Bom, MD/PhD/Prof · Leiden University Medical Center

• Camila Caram-Deelder, MSc/PhD · Leiden University Medical Center

• Enrico Lopriore, MD/PhD/Prof · Leiden University Medical Center

• Karin Fijnvandraat, MD/PhD/Prof · Amsterdam University Medical Center

• Suzanne F Fustolo-Gunnink, MD/PhD · Sanquin Blood Supply Foundation

• Wes Onland, MD/PhD · Amsterdam University Medical Center

• Nan van Geloven, MSc/PhD · Leiden University Medical Center

• Ilaria Prosepe, MSc · Leiden University Medical Center

Eligibility

Min Age

22 Weeks

Max Age

34 Weeks

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2022-09-26

Primary Completion

2024-04-30

Completion

2024-04-30

Countries

• Germany
• Netherlands
• Sweden

Study Locations