Authors: Schober S, Döring M, Lang P, Schulte J, Olivieri M, Icheva V.
DOI: 10.1016/j.rpth.2025.102842
Abstract Summary
First reported case of a newborn with both severe combined immunodeficiency (SCID) and severe hemophilia A successfully treated. Doctors used prophylactic factor VIII therapy with extended half-life product during stem cell transplantation, adjusting doses based on platelet counts. No bleeding complications occurred.
Why Brain? 🧠
First case of newborn with both severe combined immunodeficiency and severe hemophilia A successfully treated with stem cell transplantation using individualized factor VIII prophylaxis, proving feasibility without bleeding.
License: cc by.
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