Diagnosis of platelet dysfunction in children: clinical predictors and test methods

Bhavya S Doshi; Eric N Thompson; Walter Faig; Abigail Wax; Amrom E Obstfeld; Michele P Lambert

doi:10.1016/j.bvth.2025.100135

Diagnosis of platelet dysfunction in children: clinical predictors and test methods

Blood Vessel Thromb Hemost. 2025 Dec 15;3(1):100135. doi: 10.1016/j.bvth.2025.100135. eCollection 2026 Feb.

Authors

Bhavya S Doshi^{1

2

3}, Eric N Thompson⁴, Walter Faig⁵, Abigail Wax¹, Amrom E Obstfeld^{6

7}, Michele P Lambert^{1

2}

Affiliations

¹ Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.
² Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
³ Aflac Cancer and Blood Disorders Center, Division of Hematology/Oncology, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA.
⁴ Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA.
⁵ Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, PA.
⁶ Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA.
⁷ Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.

Abstract

Evaluation for platelet function disorders (PFD) in children is complicated by their limited exposure to hemostatic challenges, large volumes needed for light transmission aggregometry (LTA) testing, and limited data on the performance characteristics of whole-blood methods such as whole-blood impedance lumiaggregometry (WBILA). The objective of this study was to determine the clinical variables associated with the diagnosis of a PFD. A single-center, retrospective, cohort study of children evaluated for PFD was conducted. Medical charts were abstracted for demographics, medications, testing indications, bleeding sites and severity, and laboratory results. Univariate odds ratios (OR) and multivariable modeling were conducted for association of clinical variables with PFD diagnosis in children tested by LTA or WBILA. Of 667 patients, 20.5% were diagnosed with a PFD. The PFD cohort was more likely male (OR, 1.54; P = .025) and younger (8.8 vs 10.1 years; P = .026). Neither mucocutaneous bleeding (most common presenting indication) nor bleeding severity scores correlated with increased odds of PFD diagnosis. Both LTA and WBILA showed sensitivity of >91% and specificity of >84% for PFD diagnosis. A multivariable model identified younger age, male sex, thrombocytopenia, genetic disorders, and gastrointestinal bleeding with PFD diagnosis in the WBILA cohort. In conclusion, younger, male patients have a higher incidence of PFD. These data support that WBILA can effectively rule out PFD in a pediatric population.

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