Clinical trial NCT02802982: Mathematical Modeling to Predict the Duration of Thrombocytopenia in Neonates
Official title: Mathematical Modeling to Predict the Duration of Thrombocytopenia in Neonates
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Neonatal Thrombocytopenia
Keywords: Thrombocytopenia
Neonates
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
Collaborators: name: Beth Israel Deaconess Medical Center
name: National Institutes of Health (NIH)
name: National Heart, Lung, and Blood Institute (NHLBI)
name: University of Iowa
First posted: 2016-06-16
Enrollment: 28
Sex: ALL
Minimum age (years): 5
Maximum age (years): 3
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants who have been thrombocytopenic for 5 days will be approached for the study.

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.

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.

Primary outcomes:
measure: Prediction of the duration of thrombocytopenia in neonates.
timeFrame: 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: The University of Iowa
state: Iowa
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
Study officials:
affiliation: Boston Children's Hospital
name: Martha Sola-Visner, MD
role: PRINCIPAL_INVESTIGATOR
References: