Clinical trial NCT04598750: The Neonatal Hemorrhagic Risk Assessment in Thrombocytopenia
Official title: The Neonatal Hemorrhagic Risk Assessment in Thrombocytopenia Study-2
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Neonatal Thrombocytopenia
Bleeding
Lead sponsor: Karolinska Institutet
Sponsor class: OTHER
Collaborators: name: Karolinska University Hospital
name: National Heart, Lung, and Blood Institute (NHLBI)
name: Boston Children's Hospital
name: Harvard Medical School (HMS and HSDM)
name: Region Stockholm
name: The Swedish Society of Medicine
First posted: 2020-10-22
Enrollment: 250
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: - Infants will be eligible for study if they have a gestational age \<32 weeks and a birth weight ≥500 grams; and have a platelet count \<100 x 109/L.

Brief summary: This is a prospective observational study designed to evaluate Immature Platelet Fraction or Immature Platelet Count and Platelet Function Analyzer-100/200 Closure Time-ADP (in vitro bleeding time) as markers of bleeding risk in thrombocytopenic preterm neonates admitted to the Neonatal Intensive Care Unit.

Detailed description: Thrombocytopenia is a known risk factor for clinically significant bleeding in neonates. However, there is a poor correlation between degree of thrombocytopenia and bleeding risk. A better marker of bleeding risk suitable for use in neonates could help physicians more accurately determine the risk/benefit ratio of platelet transfusions, guiding platelet transfusion decisions, and potentially protecting vulnerable infants from exposure to unnecessary transfusion-related risks. The investigators recently found that the Platelet Function Analyzer (PFA) Closure Time-Collagen/ADP (CT-ADP) was a better marker of bleeding than the platelet count in preterm neonates. However, the CT-ADP requires 0.8 mL blood limiting its potential widespread use. The Immature Platelet Fraction (IPF) is a new laboratory marker measuring the % newly released and more active platelets, measured from the same sample as the platelet count. This is a prospective observational study designed to evaluate IPF as marker of bleeding risk in thrombocytopenic neonates admitted to the Neonatal Intensive Care Unit, compared to platelet counts alone. And also, to validate the previously found association between PFA-100/200 CT-ADP and bleeding in a bigger cohort, to compare the IPF with the PFA-100/200 CT-ADP as bleeding predictors and to assess whether the PFA-100/200 CT-ADP combined with the IPF is able to predict bleeding in thrombocytopenic preterm neonates.

Eligibility criteria: Inclusion Criteria:

* Have a gestational age \<32 weeks and a birth weight ≥500 grams;
* Have a platelet count \<100 x 109/L; and
* Have a parent/guardian willing to provide written informed consent.

Exclusion Criteria:

* Are not expected to survive for \>24 hours by the Attending Neonatologist;
* Are thought to have a familial thrombocytopenia or platelet dysfunction, based on family history or clinical presentation (associated congenital malformations, platelet morphology).

Primary outcomes:
measure: NeoBAT score
timeFrame: 24 hours
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Murray
country: United States
facility: Intermountain Medical Center
state: Utah
city: Provo
country: United States
facility: Utah Valley Hospital
state: Utah
city: Amsterdam
country: Netherlands
facility: Amsterdam University Medical Centre
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
city: Huddinge
country: Sweden
facility: Karolinska University Hospital Huddinge campus
city: Stockholm
country: Sweden
facility: Karolinska University Hospital Solna campus, Astrid Lindgren Children's Hospital
Study officials:
affiliation: Karolinska Institutet
name: Emöke Deschmann, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Utah Medical Center
name: Robert Christensen, MD
role: STUDY_CHAIR
References: