Details

Autor(en) / Beteiligte

• Grottke, Oliver
• Moser, Olga
• Farrag, Ahmed
• Elbracht, Miriam
• Orlikowsky, Thorsten
• Sonja Trepels‐Kottek

Titel

Plasma‐derived FactorX therapy for treatment of intracranial bleeding in a patient with FactorX deficiency: a case report

Ist Teil von

• Transfusion (Philadelphia, Pa.), 2019-07, Vol.59 (7), p.2228-2233

Ort / Verlag

Bethesda: Wiley Subscription Services, Inc

Erscheinungsjahr

2019

Quelle

Wiley-Blackwell Journals

Beschreibungen/Notizen

• BACKGROUNDFactor X (FX) deficiency (FXD) is an extremely rare autosomal recessive hereditary hematologic disorder, affecting approximately one in 1,000,000 of the general population.CASE REPORTThis case report describes an infant with hereditary severe FXD who presented with a spontaneous, life‐threatening intracranial hemorrhage and was treated with the first licensed plasma‐derived FX (pdFX) concentrate. On admission, laboratory assays showed severe coagulopathy of unknown cause; the patient was empirically treated using a multimodal hemostatic approach with prothrombin complex concentrate, fresh‐frozen plasma, and tranexamic acid. Subsequent single‐factor coagulation and genetic analyses confirmed the hereditary FXD diagnosis, and the therapeutic regimen was changed to a targeted regimen of 250 IU pdFX daily. Based on careful monitoring of the coagulation profile, pdFX administration frequency was increased to twice daily, followed by a reduction to once every 18 hours. The patient was discharged after 7 weeks of hospitalization in good clinical condition and now receives prophylactic pdFX three times weekly.