Mathematical Modeling to Predict the Duration of Thrombocytopenia in Neonates

NCT ID: NCT02802982

Last Updated: 2024-05-16

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 28 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2013-04-30
• Study Completion Date: 2024-12-31

Brief Summary

Parents of infants who have been thrombocytopenic for 3-4 days will be approached for consent to enter the study. For the purposes of the study, thrombocytopenia will be defined as a platelet count <60,000/uL or a platelet count <100,000/uL that prompted a platelet transfusion. Following enrollment, the platelet count will be followed in each infant. Participants will enter the study if on day 5 or later after the onset of thrombocytopenia (defined as above) infants either have a platelet count <60,000/uL or a platelet count <100,000/uL for which a platelet transfusion is ordered.

Detailed Description

Parents of infants who have been thrombocytopenic for 3-4 days will be approached for consent to enter the study. For the purposes of the study, thrombocytopenia will be defined as a platelet count <60,000/uL or a platelet count <100,000/uL that prompted a platelet transfusion. Following enrollment, the platelet count will be followed in each infant. Participants will enter the study if, on day 5 or later after the onset of thrombocytopenia (defined as above), infants either have a platelet count <60,000/uL or a platelet count <100,000/uL for which a platelet transfusion is ordered. If criteria are met, eligible infants will have a single blood sample drawn (approx. 800 mcL total) for a complete blood count with Immature Platelet Fraction (IPF) and for determination of a panel of factors important in the regulation of thrombopoiesis (including TPO, IL-6, IL-11, IL-3, PF-4, VEGF, HGF, PDGF, and Epo). Importantly, in patients who are being transfused for platelet counts <100,000/uL, this sample will need to be obtained immediately prior to the platelet transfusion. If the patient has a platelet count <60,000/uL and is not being transfused, the blood can be obtained at any time.

Following this initial sample, a platelet count with IPF will be obtained any time a CBC is ordered for clinical indications, using left-over blood stored in the clinical laboratory for <24 hrs (only 100 mcL are needed for this). In addition, left-over blood from clinically indicated studies will be collected from the clinical laboratory, processed and stored at -80C for future cytokine studies. Samples will continue to be collected and serial platelet counts with IPF followed until resolution of the thrombocytopenia, defined as a platelet count >60,000/uL for five days without platelet transfusions.

In addition, research nurses will collect and record the infants' demographic data (including gestational age, days of life, birth weight), diagnoses, clinical condition at the time of study entry (respiratory and/or hemodynamic support), time and volume of platelet transfusions, coagulation tests, liver enzymes, and tests of kidney function. Moderate and severe bleeding will also be recorded, using criteria defined a priori.

Conditions

Neonatal Thrombocytopenia

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

1. Are admitted to one of the participating NICUs or the CICU at BCH;

2. Have a post-conceptional age (gestational age + age in weeks) between 23 and 48 weeks; and

3. Have had thrombocytopenia, defined as a platelet count <60,000/uL or a platelet count <100,000/uL but receiving platelet transfusions, for ≥ 5 days.

Exclusion Criteria

1. Are on ECMO; or

2. Are not expected to survive by the attending neonatologist.

• Minimum Eligible Age: 5 Days
• Maximum Eligible Age: 3 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Beth Israel Deaconess Medical Center

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

University of Iowa

OTHER

Sponsor Role: collaborator

Boston Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Martha Sola-Visner

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Martha Sola-Visner, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

The University of Iowa

Iowa City, Iowa, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

5R01HL069990-11

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB-P00005413

Identifier Type: -

Identifier Source: org_study_id