Background: Factor X deficiency is a rare bleeding disorder that requires long-term management. The female infant discussed within this case study, Patient A, was diagnosed with severe factor X deficiency two days after birth. Therapy with prothrombin complex concentrate (PCC) was initiated until factor X concentrate was approved by the insurance provider. After initially receiving twice weekly factor X concentrate infusions at her local pediatric Hemophilia Treatment Center (HTC) for prophylaxis of bleeding episodes, infusion therapy was transitioned to the home environment at one-month old. 

Aims: The purpose of this case study is to highlight the successful long-term, effective use of factor X concentrate in a female infant patient in the home environment.

Methods: A retrospective review of medical records was performed by a multidisciplinary team. The review included customized clinical assessments specific to bleeding disorders that were performed by the specialty infusion pharmacy, as well as progress notes, nursing visit assessments, and hospital and physician office visit notes from June 9, 2017 through January 23, 2020.

Results: During the 30 months that Patient A received factor X concentrate infusions in the home environment, she experienced zero bleeding episodes and zero adverse reactions. The family became independent with twice weekly infusions within two months of initiating services through the specialty infusion pharmacy. Several catheter events, such as occlusions and breakages, did occur; however, no events were related to catheter infections. Patient A’s underlying factor X deficiency did cause her to suffer a brain injury either during or before birth, but physical limitations were resolved with physical and occupational therapy.

Conclusions: Factor X concentrate is an effective targeted therapy for factor X deficiency that is well-tolerated. With proper use and frequent monitoring through customized clinical assessments, factor X deficiency can be successfully managed in the home environment at any age.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/successful-prophylactic-use-of-coagulation-factor-x-in-an-infant-patient-in-the-home-environment/