PB1084 - Long-term prophylaxis outcome in patients with prothrombin deficiency: A single center experience

Background: Prophylaxis with a dose schedule of weekly infusions starting at 25–40 U/kg prothrombin complex concentrate (PCC) aiming to maintain trough levels at 7–10 IU/dL may be appropriate in patients with significant hemorrhagic problems/ symptoms.

Aims: Our aim is to evaluate the long-term efficacy of prophylaxis in two pediatric patients with severe prothrombin deficiency.

Methods: A cross section study was conducted at Shiraz University of Medical Sciences, Iran in 2023 on two patients with prothrombin deficiency.
Consent form was taken, and the study comply with the Declaration of Helsinki.

Results: Case 1: 7-year-old girl presented with umbilical cord bleeding followed by GI bleeding, was diagnosed with prothrombin deficiency (FII: 1.8%). He underwent prophylaxis with a dose of 25-30 units/kg PCC once weekly at the age of 45 days. The patient developed convulsion and brain CT was consistent with ICH at age of 8 months and so the frequency was switched to twice weekly with trough level of 7.1% without having any breakthrough bleeding.
Case 2: 7-year-old boy presented with umbilical cord bleeds, was diagnosed with prothrombin deficiency (2%), underwent prophylaxis with dose of 25-30 units/kg PCC once weekly but he developed ICH after a flight at age of 7.5 months. (Figure 1) The prophylaxis regimen was switched to twice weekly with trough level of 6-14% without any reported significant bleeds.

Conclusion(s): Our data shows long-term administration of PCC twice weekly as a secondary prophylaxis is efficacious and safe.
Prophylaxis with dose of 20–30 IU/ kg-1 PCC once weekly has been used previously and seem to be effective, but our results showed higher doses or shorter frequency may be required in the event of life-threatening bleeding like ICH or major surgery. Our results revealed twice weekly is completely more appropriate than once weekly aiming to maintain minimum trough levels at 7 IU/dL.