Clinical trial NCT06676904: Neonatal Platelet Transfusion Threshold Trial
Official title: Neonatal Platelet Transfusion Threshold Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Thrombocytopenia
Neonatal
Platelet Transfusion
Infant, Newborn, Diseases
Infant, Extremely Low Birth Weight
Infant, Small for Gestational Age
Thrombosis
Keywords: platelet transfusion
neonatal
thrombosis
Lead sponsor: NICHD Neonatal Research Network
Sponsor class: NETWORK
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2024-11-06
Enrollment: 2433
Sex: ALL
Minimum age (years): 1
Maximum age (years): 48
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomized trial with 1:1 allocation to parallel arms within strata of Study Center and gestational age (23-24 weeks and 25-26 weeks PMA). Multiples will be randomized independently. Approximately 30% of enrolled participants are expected to meet the platelet count threshold for randomization. The target sample size for randomization is 730, or 365 per arm.
Primary purpose: PREVENTION

Brief summary: The objective of the NeoPlaTT trial is to test whether, among extremely preterm infants born at 23 0/7 to 26 6/7 weeks' gestation, a lower platelet transfusion threshold, compared to a higher threshold, improves survival without major or severe bleeding up to 40 0/7 weeks' postmenstrual age (PMA).

Detailed description: Thrombocytopenia, defined as a platelet count \<150 x 10\^9/L, is a common neonatal problem that affects 22% to 35% of infants admitted to the neonatal intensive care unit. Platelets are important for primary hemostasis to prevent blood extravasation after vascular injury. Based on the role of platelets in hemostasis, prophylactic platelet transfusions are routinely administered to preterm infants with thrombocytopenia to prevent bleeding. The incidence of thrombocytopenia and administration of platelet transfusion are both inversely related to the gestational age at birth. Currently, there is uncertainty regarding the optimal platelet transfusion threshold, particularly among the most immature infants in the first week of life, which represents the period of highest bleeding risk.

The NeoPlaTT trial was designed to address this pressing uncertainty in the highest risk population (\<27 weeks GA). It will test whether a threshold of 20x10\^9/L could be safely used after the first week of life, when the risk of serious bleeding is significantly lower, and reduce the need for platelet transfusion altogether. The results of this study have a potential to change clinical practice and improve outcomes in this vulnerable population, while also decreasing costs and resource utilization.

This is a randomized trial with 1:1 allocation to parallel arms. Infants, inborn or outborn, who are admitted to participating NICUs, and who meet the inclusion and exclusion criteria, will be invited to enroll into the trial for platelet count monitoring. Only consented and enrolled infants meeting the additional platelet count trigger of \< 50 x 10\^9/L (postnatal days 1-7) or \<35 x 10\^9/L (8 or more postnatal days) will be randomized. Postnatal day 1 starts at birth. Randomization will be allowed to occur up to 36 6/7 weeks' PMA; subjects will be monitored through 40 0/7 weeks PMA. Approximately 30% of consented and enrolled infants are expected to meet the platelet count threshold for randomization.

Eligibility criteria: Inclusion Criteria:

* Gestational age of 23 0/7 to 26 6/7 weeks
* Postnatal age of \< 48 hours

Exclusion Criteria:

* Comfort care or withdrawal of care planned
* Neonatal alloimmune thrombocytopenia or suspected/confirmed congenital platelet or bleeding disorder
* Receipt of platelet transfusion
* No receipt of Vitamin K
* Parents/guardian decline consent

Primary outcomes:
measure: Survival without major or severe bleeding
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
Secondary outcomes:
measure: Death
timeFrame: Randomization to 120 days' postnatal age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 112 days postnatal age)
measure: Major or severe bleeding
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
measure: Number of platelet transfusions
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
measure: At least one platelet transfusion
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
measure: Bronchopulmonary dysplasia among survivors to 36 weeks postmenstrual age
timeFrame: At 36 weeks postmenstrual age
measure: Retinopathy of Prematurity (ROP)
timeFrame: Randomization to 120 days' postnatal age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 112 days postnatal age)
measure: Periventricular leukomalacia
timeFrame: Randomization to 120 days' postnatal age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 112 days postnatal age)
Other outcomes:
measure: Late-onset sepsis
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
measure: Necrotizing enterocolitis
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
measure: Thrombosis requiring therapy
timeFrame: Randomization to 40 0/7 weeks postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 98 days postnatal age)
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: Sharp Mary Birch Hospital for Women & Newborns
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Northwestern Lurie Children's Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Case Western Reserve University, Rainbow Babies and Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
city: San Antonio
country: United States
facility: Pediatrix Medical Group
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: Emory University
name: Ravi M Patel, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Venkatesh V, Curley A, Khan R, Clarke P, Watts T, Josephson C, Muthukumar P, New H, Seeney F, Morris S, Stanworth S. A novel approach to standardised recording of bleeding in a high risk neonatal population. Arch Dis Child Fetal Neonatal Ed. 2013 May;98(3):F260-3. doi: 10.1136/archdischild-2012-302443. Epub 2012 Nov 9.