Abstract Number: PB0901
Meeting: ISTH 2021 Congress
Background: For pediatric patients with Bernard-Soulier Syndrome (BSS) and Glanzmann Thrombasthenia (GT), frequent and severe bleeding events (SBE) can lead to chronic blood loss and secondary iron deficiency anemia (IDA). SBE are primarily treated on-demand with platelet transfusions or recombinant activated factor VII (rFVIIa) infusions. The impact of instituting prophylactic administration of platelets and/or rFVIIa on bleeding and bleeding-associated complications is unclear.
Aims: To evaluate bleeding outcomes and management in pediatric patients with BSS or GT.
Methods: A retrospective chart-review of patients with BSS/GT followed at our pediatric hemophilia treatment center between 2007-2019 was conducted.
Results: We identified 14 patients with a diagnosis of BSS (n = 2) or GT (n = 12). Annualized bleeding rates ranged from 0.1 – 18.4 events/year, but 93% patients had at least one SBE. The most common bleeding symptoms were epistaxis and oral bleeding. Patients were treated with on-demand rFVIIa infusions (7%), platelets (7%), or a combination of platelets and rFVIIa (57%) for bleeds. Approximately 86% of patients had IDA requiring iron replacement (11 oral and 3 intravenous) or packed red blood cell transfusions (pRBC, 7/14). Due to recurrent SBE resulting in refractory IDA, 3 patients (21%) received rFVIIa prophylaxis at 90 micrograms/kilogram 2-3 times/week for 15-31 months. Patients started on rFVIIa prophylaxis had a median hemoglobin of 9.77 g/dL (8.0-10.7 g/dL) compared to 11.65 g/dL (8.4-13.8 g/dL) for on-demand treated patients. In these 3 patients, median hemoglobin and ferritin increased by 1.28 g/dL (0.7-2.5 g/dL) and 14.63 mcg/dL (0.23-42.9 mcg/dL), respectively. One patient on thrice-weekly rFVIIa prophylaxis also required platelet transfusions every 2 weeks to further prevent epistaxis.
Conclusions: IDA is an important indicator of bleeding severity in pediatric patients with inherited bleeding disorders. Routine monitoring for IDA may help identify patients with BSS/GT that could benefit from prophylaxis regimens to reduce bleed burden.
To cite this abstract in AMA style:Lee A, Batsuli G. Impact of Iron Deficiency Anemia on Bleeding Management in Pediatric Patients with Bernard-Soulier Syndrome and Glanzmann Thrombasthenia: A Single-institution Analysis [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/impact-of-iron-deficiency-anemia-on-bleeding-management-in-pediatric-patients-with-bernard-soulier-syndrome-and-glanzmann-thrombasthenia-a-single-institution-analysis/. Accessed August 16, 2022.
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