Neonatal Platelet Transfusion Threshold Trial (NeoPlaTT)

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).

Study Overview

• Status: Recruiting
• Conditions: Infant, Newborn, Diseases; Thrombosis; Thrombocytopenia; Infant, Extremely Low Birth Weight; Infant, Small for Gestational Age; Neonatal; Platelet Transfusion
• Intervention / Treatment: Procedure: Higher Platelet Transfusion Threshold; Procedure: Lower Platelet Transfusion Threshold

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.

• Study Type: Interventional
• Enrollment (Estimated): 2433
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Abhik Das, PhD; Phone Number: 301-230-4640; Email: adas@rti.org
• Study Contact Backup: Name: Ravi M Patel, MD; Phone Number: 404-727-5905; Email: rmpatel@emory.edu

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Recruiting
      • University of Alabama at Birmingham
      • Contact: Waldemar A Carlo, MD
  • California
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Stanford University
      • Principal Investigator: Valerie Chock, MD
      • Contact: Valerie Chock, MD
    • San Diego, California, United States, 92123
      • Recruiting
      • Sharp Mary Birch Hospital for Women & Newborns
      • Contact: Anup Katheria, MD
      • Principal Investigator: Anup Katheria, MD
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado
      • Contact: Sunah Hwang, MD
      • Principal Investigator: Sunah Hwang, MD
  • Georgia
    • Atlanta, Georgia, United States, 30303
      • Recruiting
      • Emory University
      • Sub-Investigator: Brenda Poindexter, MD
      • Contact: Ravi Patel, MD
      • Principal Investigator: Ravi Patel, MD
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Northwestern Lurie Children's Hospital of Chicago
      • Contact: Aaron Hamvas, MD
      • Principal Investigator: Aaron Hamvas, MD
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Recruiting
      • University of Iowa
      • Principal Investigator: Edward F. Bell, MD
      • Contact: Tarah Colaizy, MD, MPH; Phone Number: 319-356-3508
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Recruiting
      • Beth Israel Deaconess Medical Center
      • Contact: Helen Healy, MD
      • Principal Investigator: Helen Healy, MD
  • New Mexico
    • Albuquerque, New Mexico, United States, 87131
      • Recruiting
      • University of New Mexico
      • Principal Investigator: Janell Fuller, MD
      • Contact: Janell Fuller, MD
  • New York
    • Rochester, New York, United States, 14642
      • Recruiting
      • University of Rochester
      • Contact: Carl T D'Angio, MD
      • Principal Investigator: Carl T D'Angio, MD
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Duke University
      • Contact: Michael Cotten, MD
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • Cincinnati Children's Medical Center
      • Contact: Stephanie Merhar, MD MS
      • Sub-Investigator: Vivek Narendran, MD, MBA
      • Principal Investigator: Stephanie Merhar, MD MS
    • Cleveland, Ohio, United States, 44106
      • Recruiting
      • Case Western Reserve University, Rainbow Babies and Children's Hospital
      • Contact: Anna Maria Hibbs, MD
      • Principal Investigator: Anna Maria Hibbs, MD
    • Columbus, Ohio, United States, 43205
      • Recruiting
      • Nationwide Children's Hospital
      • Contact: Pablo Sanchez, MD
      • Principal Investigator: Pablo Sanchez, MD
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • University of Pennsylvania
      • Contact: Sara DeMauro, MD
      • Principal Investigator: Sara DeMauro, MD
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University
      • Contact: Hendrick Weitkamp, MD
      • Principal Investigator: Hendrick Weitkamp, MD
  • Texas
    • Dallas, Texas, United States, 75235
      • Recruiting
      • University of Texas Southwestern Medical Center at Dallas
      • Contact: Myra Myckoff, MD
      • Principal Investigator: Myra Wyckoff, MD
    • Houston, Texas, United States, 77030
      • Recruiting
      • University of Texas Health Science Center at Houston
      • Contact: Jon Tyson
      • Principal Investigator: Jon Tyson, MD, MPH
    • San Antonio, Texas, United States, 78229
      • Recruiting
      • Pediatrix Medical Group
      • Principal Investigator: Kaashif Ahmad, MD
      • Contact: Kaashif Ahmad, MD
  • Utah
    • Salt Lake City, Utah, United States, 84108
      • Recruiting
      • University of Utah
      • Contact: Robin Ohls, MD
      • Principal Investigator: Robin Ohls, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: Yes

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Higher Platelet Transfusion Threshold; Infants randomized to this arm will be monitored for a platelet transfusion threshold of 50 x 10^9/L during postnatal days 1-7, and then for a platelet transfusion threshold of 35 x 10^9/L at 8 or more postnatal days of life. Infants will remain on this protocol-driven threshold through 40 0/7 weeks postmenstrual age. The platelet dose will be 10 ml/kg administered over 60-120 minutes.	Procedure: Higher Platelet Transfusion Threshold; Infants randomized to this arm will be monitored for a platelet transfusion threshold of 50 x 10^9/L during postnatal days 1-7, and then for a platelet transfusion threshold of 35 x 10^9/L at 8 or more postnatal days of life. Infants will remain on this protocol-driven threshold through 40 0/7 weeks postmenstrual age. The platelet dose will be 10 ml/kg administered over 60-120 minutes.; Other Names: Liberal Transfusion Threshold
Other: Lower Platelet Transfusion Threshold; Infants randomized to this arm will be monitored for a platelet transfusion threshold of 25 x 10^9/L during postnatal days 1-7, and then for a platelet transfusion threshold of 20 x 10^9/L at 8 or more postnatal days of life. Infants will remain on this protocol-driven threshold through 40 0/7 weeks postmenstrual age. The platelet dose will be 10 ml/kg administered over 60-120 minutes.	Procedure: Lower Platelet Transfusion Threshold; Infants randomized to this arm will be monitored for a platelet transfusion threshold of 25 x 10^9/L during postnatal days 1-7, and then for a platelet transfusion threshold of 20 x 10^9/L at 8 or more postnatal days of life. Infants will remain on this protocol-driven threshold through 40 0/7 weeks postmenstrual age. The platelet dose will be 10 ml/kg administered over 60-120 minutes.; Other Names: Restrictive Transfusion Threshold

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival without major or severe bleeding	Bleeding will be assessed using the neonatal Bleeding Assessment Tool (NeoBAT), a validated bleeding assessment tool (Venkatesh V, 2013)	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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Major or severe bleeding	Bleeding will be assessed using the neonatal Bleeding Assessment Tool (NeoBAT), a validated bleeding assessment tool (Venkatesh V, 2013)	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)
Number of platelet transfusions		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)
At least one platelet transfusion		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)
Bronchopulmonary dysplasia among survivors to 36 weeks postmenstrual age		At 36 weeks postmenstrual age
Death		Randomization to 52 0/7 weeks' postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first, per Neonatal Research Network Generic Research Database Registry protocol.
Retinopathy of Prematurity (ROP)	Stage 3 ROP, or stage 1 or 2 in Zone 1, or plus disease	Randomization to 52 0/7 weeks' postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first, per Neonatal Research Network Generic Research Database Registry protocol.
Periventricular leukomalacia		Randomization to 52 0/7 weeks' postmenstrual age, death, discharge, or transfer outside of the Study Center, whichever occurs first, per Neonatal Research Network Generic Research Database Registry protocol.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Late-onset sepsis		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)
Necrotizing enterocolitis	Modified Bells Stage IIA or greater	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)
Thrombosis requiring therapy	Thrombosis requiring therapy such as heparin, enoxaparin, or aspirin	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)

Collaborators and Investigators

• Sponsor: NICHD Neonatal Research Network
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
• Investigators: Principal Investigator: Ravi M Patel, MD, Emory University

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): June 13, 2025
• Primary Completion (Estimated): January 31, 2031
• Study Completion (Estimated): April 30, 2031

Study Registration Dates

• First Submitted: October 3, 2024
• First Submitted That Met QC Criteria: November 4, 2024
• First Posted (Actual): November 6, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2026
• Last Update Submitted That Met QC Criteria: March 24, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: thrombosis; neonatal; platelet transfusion
• Additional Relevant MeSH Terms: Cytopenia; Vascular Diseases; Cardiovascular Diseases; Hematologic Diseases; Embolism and Thrombosis; Blood Platelet Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Thrombosis; Thrombocytopenia; Infant, Newborn, Diseases
• Other Study ID Numbers: NICHD-NRN-0065; 1U24HL173304-01 (U.S. NIH Grant/Contract); UG3HL173303 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: NIH has had a long-standing policy to share and make available to the public the results and accomplishments of the activities that it funds. The NRN plans to share de-identified data after final publication in an NIH supported data repository such as the NICHD Data and Specimen Hub (https://dash.nichd.nih.gov).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No