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Background  Current treatment for severe hemophilia A is replacement of deficient factor. Although replacement therapy has improved life expectancy and quality, limitations include frequent infusions and high costs. Gene therapy is a potential alternative that utilizes an adeno‐associated virus ( AAV ) vector containing the human genetic code for factor 8 ( FVIII ) that transduces the liver, enabling endogenous production of  FVIII . Individuals with preexisting immunity to  AAV  serotypes may be less likely to benefit from this treatment.    Objectives  This study measured seroprevalence of antibodies to  AAV 5 and 8 in an  UK  adult hemophilia A cohort.    Patients/Methods  Patients were recruited from seven hemophilia centres in the  UK . Citrated plasma samples from 100 patients were tested for preexisting activities against  AAV 5 and 8 using  AAV  transduction inhibition and total antibodies assays.    Results  Twent‐one percent of patients had antibodies against  AAV 5 and 23% had antibodies against  AAV 8. Twenty‐five percent and 38% of patients exhibited inhibitors of  AAV 5 or  AAV 8 cellular transduction respectively. Overall seroprevalence using either assay against  AAV 5 was 30% and against  AAV 8 was 40% in this cohort of hemophilia A patients. Seropositivity for both  AAV 5 and  AAV 8 was seen in 24% of participants.    Conclusions  Screening for preexisting immunity may be important in identifying patients most likely to benefit from gene therapy. Clinical studies may be needed to evaluate the impact of preexisting immunity on the safety and efficacy of  AAV  mediated gene therapy.

research and practice in thrombosis and haemostasis

Adenovirus‐associated antibodies in  UK  cohort of hemophilia patients: A seroprevalence study of the presence of adenovirus‐associated virus vector–serotypes  AAV 5 and  AAV 8 neutralizing activity and antibodies in patients with hemophilia A