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Patient-centered approach to managing factor XIII deficiency
Varun Iyengar1, Caitlin Montcrieff2, Salley Pels2
1Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA viyengar@bidmc.harvard.edu.
BMJ Case Reports
|September 17, 2021
Summary
This study details individualized recombinant Factor XIII (FXIII) dosing for rare bleeding disorders. Pharmacokinetic data helped optimize treatment for patients with refractory bleeding, improving outcomes.
Area of Science:
- Hematology
- Pharmacology
Background:
- Congenital Factor XIII (FXIII) A-subunit deficiency is a rare bleeding disorder affecting 1-3 million individuals.
- Treatment involves recombinant FXIII (rFXIII), but optimal dosing is challenging due to individual variations and refractory bleeding.
Observation:
- This report describes a novel approach to rFXIII dosing in two patients with FXIII deficiency.
- Standard dosing protocols were deviated from to maintain therapeutic FXIII levels, addressing refractory bleeding.
Findings:
- Individualized rFXIII dosing, guided by pharmacokinetic data, can effectively manage complex cases of FXIII deficiency.
- This approach helped maintain therapeutic FXIII troughs and improve patient outcomes.
Implications:
- Highlights the need for personalized medicine in rare bleeding disorders.
- Emphasizes the importance of pharmacokinetic data in optimizing rFXIII therapy.
- Underscores the value of patient-centered, multidisciplinary care for complex hematological conditions.
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