Impact of Thrombocytopenia and Platelet Transfusions on Neonatal Bleeding and Inflammation

NCT03848923 | Recruiting

• 2 other identifiers: IRB-P000294822P01HL046925-21A1
• Study Type: observational
• Target: 160
• Locations: 1 country
• Sites: 2

Brief Summary

This is a prospective observational study that was designed with the following two Specific Aims:

1. 1.To determine whether the Immature Platelet Fraction percentage (IPF%) and the Immature Platelet Count (IPC) are better predictors of bleeding than the platelet count alone in neonates of different gestational and post-conceptional ages and with different etiologies of thrombocytopenia; and
2. 2.To characterize the effects of neonatal thrombocytopenia and platelet transfusions (PLT Tx) on bleeding and on markers of systemic inflammation, thrombosis, and neutrophil extracellular traps (NET) formation in neonates with different underlying conditions.

Conditions

Neonatal Thrombocytopenia

Keywords

Thrombocytopenia; Neonates; Platelets; NET Formation

Outcome Measures

Primary Outcomes (1)

• Assessment of the bleeding score using the Neo-BAT (Neonatal Bleeding Assessment Tool),

  The Neo-BAT categorizes bleeding as none (0), minor (1), moderate (2), severe (3), and major (4). A bleeding score will be obtained by the bedside nurse within 2 hours of every PLT count and IPF% checked. NeoBAT scores will include any bleeding since the last PLT count or over the prior 24 hours, whichever is shortest. This will serve to correlate bleeding scores with PLT counts, and to quantify changes following PLT Tx.

  Approximately 3 years

Secondary Outcomes (1)

• Measurement of changes in cytokine levels and markers of intravascular coagulation and NET formation following PLT Tx

  Approximately 3 years

Eligibility Criteria

• Age: 0 Days - 6 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

This study will enroll neonates admitted to the NICUs at Boston Children's Hospital (BCH) and Beth Israel Deaconess Medical Center (BIDMC) and the Cardiac Intensive Care Unit (CICU) at BCH who fulfill the following criteria

You may qualify if:

• Have a post-menstrual age between 23 and 44 weeks;
• Have a PLT count \<100 x 109/L; and
• Have a parent/guardian willing to provide written informed consent.

You may not qualify if:

• Are not expected to survive for \>5 days by the Attending Neonatologist;
• Are thought to have a congenital thrombocytopenia or platelet dysfunction, based on family history or clinical presentation (e.g. congenital malformations, platelet morphology); or
• Are on extracorporeal membrane oxygenation (ECMO).
• Importantly, patients will be consented when they have a platelet count \<100 x 109/L, but they will enter study only when the platelet count falls to \<50 x 109/L.

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Beth Israel Deaconess Medical Center: collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (2)

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

2 study-specific blood samples (0.5-1.0 cc each) will be taken to the study laboratory for a CBC and plasma separation and storage for future measurements of dsDNA and MPO-DNA ELISA, markers of NET formation, TAT complexes (markers of intravascular coagulation), and for a panel of serum cytokines/vascular injury markers (IFNɣ, IL-6, IL-8, IL-10, IL-17, IL-18, TNFα/β, IP-10, MCP-1, ICAM, VCAM, and VEGF) by Luminex; In addition, left-over plasma samples from clinical tests will be collected daily from the clinical laboratory, aliquoted, and frozen for future cytokine measurements, as we did to generate the preliminary data for this study.

MeSH Terms

Conditions

Thrombocytopenia, Neonatal Alloimmune; Thrombocytopenia

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Martha Sola-Visner, MD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Martha Sola-Visner, MD

CONTACT

617-919-4845; martha.sola-visner@childrens.harvard.edu

Vanessa J Young, RN, BA

CONTACT

617-355-8330; vanessa.young@childrens.harvard.edu

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Pediatrics

Study Record Dates

• First Submitted: January 3, 2019
• First Posted: February 21, 2019
• Study Start: August 19, 2019
• Primary Completion: March 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: May 16, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)