Page 339 - Haematologica Vol. 109 - July 2024

LETTER TO THE EDITOR
Figure 2. International Society of Thrombosis and Hemostasis Bleeding Assessment Tool Bleeding Score between participants with and without clinically significant bleeding events in each study group. Data are presented as medians and interquartile ranges. *P<0.05; **P<0.01. ISTH-BAT: International Society of Thrombosis and Hemostasis Bleeding Assessment Tools; vWD: von Willebrand disease; IPFD: inherited platelet function disorder. Controls had a bleeding phenotype but no identifiable bleeding disorder on comprehensive laboratory evaluation.
provided by Gresele et al., which reported <5% of pediatric IPFD bleeding events to be surgical, likely explained by the prospective nature of the study where IPFD diagnosis was already established and early intervention possible.12 ISTH-BAT scores were comparable for IPFD and vWD-1 par- ticipants. Gresele et al. found a significantly higher bleeding score in pediatric IPFD versus vWD-1 participants (8 vs. 4, respectively).12 However, Bidlingmaier et al. found no differ- ence in BS, with both pediatric IPFD and vWD-1 participants having a BS of 3.10 This is likely driven by the inclusion of severe platelet disorders in the Gresele study, whereas the Bidlingmaier study included a similar cohort of IPFD as ours, leading to comparable results.
For our IPFD participants, BS >4 predicted subsequent bleeding events, BS >3 predicted bleeding events requiring treatment, and BS >7 predicted severe bleeding events. Our study predicted bleeding events at a lower BS than Gresele et al. (BS >7), likely due to higher ABR within our study population.12 Gresele et al. found that 75% of adult IPFD patients with a BS >2 required treatment, but did not comment on the ability of ISTH-BAT to predict treat- ment requirements.13 Gresele et al. found that a BS >6 was predictive of severe bleeding events in adult IPFD partici- pants;12 their inclusion of severe bleeding disorders, such as Glanzmann thrombasthenia and Type 2 and 3 vWD, likely increased their ability to predict severe bleeding events at lower bleeding scores.
In summary, IPFD is common in children referred for bleed- ing and characterized by high ABR. IPFD presentation over- laps with children without an identified bleeding disorder and vWD-1. The ISTH-BAT alone was not discriminatory for IPFD in our cohort, highlighting the need for platelet aggre- gation testing in children referred for bleeding. Patients with
high ISTH-BAT scores at IPFD diagnosis are more likely to experience severe bleeding events and require treatment. While HMB was the most common cause of bleeding in all cohorts, IPFD participants received more surgical in- terventions compared to vWD-1 and controls, whose most common treatment was hormonal therapy; as such, HMB was undertreated in IPFD. Our results underscore the im- portance of high diagnostic suspicion for IPFD in children referred for a bleeding disorder evaluation. Further, we confirm the utility of the ISTH-BAT for IPFD and bleeding prognosis in pediatric patients.
Authors
Shelly Saini,1 Song Zhang,2 Sean Yates,3 Jessica Garcia,1,4 Ruchika Sharma,1,4 Joseph Formella,1 Ravindra Sarode3 and Ayesha Zia1,4
1Department of Pediatrics; 2O’Donnell School of Public Health; 3Department of Pathology and 4Division of Hematology/Oncology, The University of Texas Southwestern Medical Center, Dallas, TX, USA
Correspondence:
A. ZIA - Ayesha.zia@utsouthwestern.edu
https://doi.org/10.3324/haematol.2024.284996
Received: January 7, 2024. Accepted: March 19, 2024. Early view: March 28, 2024.
©2024 Ferrata Storti Foundation Published under a CC BY-NC license
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